| Sample search results for: aav vectors avoid |
| 1 | Current Gene Therapy, 2003, 3, 281-304 281 1566-5232/03 $41.00+.00 2003 Bentham Science Publishers Ltd. | ||
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Summary: -associated Virus (AAV) as Novel Vectors for Human Gene Therapy D. Grimm and M.A. Kay* Stanford University, School... Abstract: Gene transfer vectors based on the human adeno-associated virus serotype 2 (AAV-2) have been... in clinical trials. So far, all these studies have provided evidence that ... |
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Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 2 | Unique Biologic Properties of Recombinant AAV1 Transduction in Polarized Human Airway Epithelia* | ||
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Summary: of these vectors to mouse lung (rAAV2/5 > rAAV2/1 rAAV2/2), emphasizing differences in transduction of these sero... eight serotypes of the AAV family have been identified and evaluated as recombinant vectors (2... that rAAV type 1, 5, and 6 ... |
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Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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Collection: Biology and Medicine |
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| 3 | Helper Virus-Free, Optically Controllable, and Two-Plasmid-Based Production of Adeno-associated | ||
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Summary: strategy for producing high-titer adeno-associated virus (AAV) vectors derived from six different AAV... , and based on transfection of only two plasmids, i.e., an AAV vector construct and one of six novel AAV... -packaging of vector DNA derived ... |
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Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 4 | Robust Systemic Transduction with AAV9 Vectors in Mice: Efficient Global Cardiac Gene Transfer Superior | ||
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Summary: in tissue A)/ (average ds rAAV8 vector copy number in tissue A). To avoid bias caused by a lot... Robust Systemic Transduction with AAV9 Vectors in Mice: Efficient Global Cardiac Gene Transfer... vector that overcomes many of the limitations of ... |
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Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 5 | Expert Review Designer Gene Delivery Vectors: Molecular Engineering and Evolution | ||
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Summary: vectors, particularly those based on AAV2, the best characterized AAV serotype. First, altering viral... of AAV vectors. BShielding^ polymers, site-directed mutagenesis, and alternative AAV serotypes have shown... success in avoiding immune ... |
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Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley |
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Collection: Materials Science ; Chemistry |
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| 6 | original article 2088 www.moleculartherapy.org vol. 17 no. 12, 20882095 dec. 2009 | ||
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Summary: (AAV) vectors, leads to predomi- nant transduction of neurons and epithelia within the central nervous... and retinal neovascular disease. To date, the use of viral vectors including adeno-associated virus (AAV... in vector production and promoter usage.19,23 Recently, ... |
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Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley |
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Collection: Materials Science ; Chemistry |
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| 7 | JOURNAL OF VIROLOGY, June 2008, p. 58875911 Vol. 82, No. 12 0022-538X/08/$08.00 0 doi:10.1128/JVI.00254-08 | ||
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Summary: its closest natural relative (AAV-2) by 60 capsid amino acids. Recombinant AAV-DJ vectors outperformed... as two powerful and compatible approaches to the molecular evolution of novel AAV vectors for human gene... in their properties (24). We and others have shown previously that the ... |
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Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 8 | Current Gene Therapy, 2001, 1, 137-147 137 1566-5232/00 $20.00+.00 2001 Bentham Science Publishers Ltd. | ||
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Summary: : Despite the fact that adeno-associated virus type 2 (AAV2) is an extremely attractive gene therapy vector... to broaden the array of target organs for this vector, molecular studies on the mechanism(s) of AAV... DNA sequences are substituted for all viral open reading frames, generating ... |
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Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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Collection: Biology and Medicine |
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| 9 | [23] Adeno-Associated Virus Vectors for Short Hairpin RNA Expression | ||
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Summary: of gene transfer vectors based on adeno-associated virus (AAV) for expressing short hairpin RNA (sh... approach, to trigger RNA interference in vitro and in vivo by shRNA expressed from AAV vectors... of novel AAV vector plasmids that were specifically designed for ... |
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Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 10 | Enhanced Preparation of Adeno-Associated Viral Vectors by Using High Hydrostatic Pressure to | ||
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Summary: for improvement remains in the areas of vector engineering and production. AAV production requires complementation... -based approaches have distinct advantages in the use of bioreactors to produce large quantities of AAV vectors... ) to selectively and completely inactivate helper adenovirus without any ... |
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Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley |
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Collection: Materials Science ; Chemistry |
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| 11 | Capsid modifications overcome low heterogeneous expression of heparan sulfate proteoglycan that limits AAV2-mediated gene transfer and therapeutic | ||
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Summary: -modified AAV vectors can mediate enhanced gene transfer to neoplasms characterized by low AAV receptor... expression. Here we sought to determine the therapeutic potential of a capsid-modified AAV vector for gene... of a modified vector with an integrin-binding RGD ... |
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Source: Hemminki, Akseli - Department of Biosciences, University of Helsinki |
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Collection: Biology and Medicine |
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| 12 | JOURNAL OF VIROLOGY, 0022-538X/00/$04.00 0 | ||
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Summary: to the vector and avoid interference VOL. 74, 2000 MECHANISM OF rAAV TRANSDUCTION IN HEPATOCYTES IN VIVO 3795... California Received 3 August 1999/Accepted 5 January 2000 Recombinant adeno-associated virus vectors (rAAV... and may be useful for improving liver gene transfer in vivo. ... |
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Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 13 | original article 2096 www.moleculartherapy.org vol. 17 no. 12, 20962102 dec. 2009 | ||
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Summary: -term transgene expression following a single treatment make the virus a very efficient gene delivery vector.4 AAV... Society of Gene & Cell Therapy Intravitreal Retinal Transduction With AAV Vectors cells (RGCs).15... Society of Gene & Cell Therapy Intravitreal Retinal Transduction With ... |
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Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley |
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Collection: Materials Science ; Chemistry |
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| 14 | Directed evolution of adeno-associated virus to an infectious respiratory virus | ||
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Summary: efficient AAV vector for pulmonary gene delivery. Directed evolution platform in organotypic polarized, well... attempts to produce AAV vectors in insect cells have shown that swapping the AAV2 VP1 region... process for AAV may avoid high levels ... |
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Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley |
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Collection: Materials Science ; Chemistry |
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| 15 | JOURNAL OF VIROLOGY, Aug. 2002, p. 76517660 Vol. 76, No. 15 0022-538X/02/$04.00 0 DOI: 10.1128/JVI.76.15.76517660.2002 | ||
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Summary: Gene therapy vectors based on adeno-associated viruses (AAVs) show promise for the treatment of retinal... vectors (2, 14, 25, 27). Recombinant adeno-asso- ciated viruses (AAVs) have been shown to direct long... -term expression in multiple tissues, including the central nervous system (2, 9, 11). ... |
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Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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Collection: Biology and Medicine |
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| 16 | JOURNAL OF VIROLOGY, June 2004, p. 63446359 Vol. 78, No. 12 0022-538X/04/$08.00 0 DOI: 10.1128/JVI.78.12.63446359.2004 | ||
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Summary: as a vector for human gene therapy applications. The most com- monly employed rAAV vector is based on the wild... makes it attractive as a gene therapy vector; however, natural exposure to wt AAV2 is already quite... vector after purification; right lane, ... |
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Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
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Collection: Biology and Medicine |
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| 17 | original article 524 www.moleculartherapy.org vol. 17 no. 3, 524537 mar. 2009 | ||
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Summary: . E-mail: rmandel@ufl.edu Striatal Readministration of rAAV Vectors Reveals an Immune Response Against... loss, we readministered rAAV2/2 and then measured striatal vector genomes using quantitative PCR... of transduced striatal neurons. Therefore, we decided to determine whether striatal ... |
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Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
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Collection: Biology and Medicine |
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| 18 | JOURNAL OF VIROLOGY, Jan. 2005, p. 364379 Vol. 79, No. 1 0022-538X/05/$08.00 0 doi:10.1128/JVI.79.1.364379.2005 | ||
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Summary: capacity (less than 5 kb) of adeno-associated virus (AAV) vectors has been effectively doubled... vectors for dual-vector trans-splicing approaches. To this end, we generated a novel AAV hybrid-ITR vector... and intramolecular homologous recombination of ... |
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Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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Collection: Biology and Medicine |
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| 19 | JOURNAL OF VIROLOGY, Apr. 2003, p. 47514759 Vol. 77, No. 8 0022-538X/03/$08.00 0 DOI: 10.1128/JVI.77.8.47514759.2003 | ||
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Summary: -PKcs regulates the removal of free rAAV vector ends in muscle tissue. In the present study, we have sought... , linear rAAV genomes and free vector ends were detected only in DNA-PKcs-deficient muscle by Southern... in hemo- philia B. The development of novel recombinant AAV ... |
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Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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Collection: Biology and Medicine |
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| 20 | MOLECULAR THERAPY Vol. 4, No. 6, December 2001 Copyright The American Society of Gene Therapy | ||
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Summary: of Germline Transmission of Vector Sequences Following Systemic Administration of Recombinant AAV-2 Vector... . These studies showed that no AAV vector sequences were detected in DNA extracted from rabbit semen samples... collected at time points ranging from 7 to 90 days following IM ... |
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Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 21 | HUMAN GENE THERAPY 16:781791 (July 2005) Mary Ann Liebert, Inc. | ||
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Summary: Recombinant adeno-associated virus 2 (rAAV2) has been extensively used as a gene delivery vector... , rela- tively new purification methods have resulted in almost com- pletely pure rAAV vector stocks... serotypes and their de- velopment into recombinant AAV vectors ... |
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Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
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Collection: Biology and Medicine |
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| 22 | Directed evolution of adeno-associated virus yields enhanced gene delivery vectors | ||
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Summary: evolution to generate `designer' gene delivery vectors with specified, enhanced properties. AAV is a 4... and the only one that has been used in clinical trials68. Recombinant gene delivery vector systems (rAAV... advantages. First, rAAV vectors are safe, as ... |
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Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley |
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Collection: Materials Science ; Chemistry |
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| 23 | Adeno-associated viral vectors and stereotaxic | ||
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Summary: types · ~4.7 kb wild type genome #12;AAV Life Cycle #12;Packaging Components of rAAV 1. Transfer vector... .4kb · Serotypes differentially infect cell types AAV as a Viral Vector #12;AAV Serotypes · AAV... Adeno-associated viral ... |
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Source: Chapman, Michael S. - Department of Biochemistry and Molecular Biology, Oregon Health and Science University |
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Collection: Biology and Medicine |
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| 24 | 64 NATURE MEDICINE VOLUME 5 NUMBER 1 JANUARY 1999 Hemophilia is an X-linked recessive bleeding disorder that | ||
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Summary: 1999 65 ARTICLES Fig. 1 Structures of the rAAV vectors. ITR, AAV inverted terminal repeat; MFG promoter... independently confirmed using a recombi- nant AAV vector that expresses factor IX from a cellular pro- moter25... hemophlilia B using a single intrahepatic administration of ... |
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Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 25 | Proc. Natl. Acad. Sci. USA Vol. 94, pp. 1408314088, December 1997 | ||
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Summary: A recombinant adeno-associated virus (rAAV) vector capable of infecting cells and expressing rat glial cell line... and to test the vector in an animal model of Parkinson's disease. To evaluate the ability of rAAV... study uses a viral vector derived from the nonpathogenic human ... |
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Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
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Collection: Biology and Medicine |
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| 26 | A Two-hybrid Screen Identifies Cathepsins B and L as Uncoating Factors for Adeno-associated Virus 2 | ||
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Summary: at a 1:1:10 ratio to form the intact viral capsid.2 Vectors derived from AAV are particularly powerful... in various cell types. Recently, the usefulness of AAV vectors has been further enhanced with the isolation... of reports, vectors pseudotyped with AAV8 can ... |
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Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 27 | A Novel Adeno-Associated Viral Variant for Efficient and Selective Intravitreal Transduction of Rat Muller Cells | ||
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Summary: , or intravitreal, injection of an efficient vehicle such as an adeno- associated viral vector (AAV). Although... demonstrate that AAV is a highly versatile vector capable of powerful shifts in tropism from minor sequence... 11-cis retinal [13]. Gene-replacement therapy employing an AAV ... |
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Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley |
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Collection: Materials Science ; Chemistry |
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| 28 | Recombinant AAV Viral Vectors Pseudotyped with Viral Capsids from Serotypes 1, 2, and 5 Display | ||
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Summary: Recombinant AAV Viral Vectors Pseudotyped with Viral Capsids from Serotypes 1, 2, and 5 Display... the tropism and transduction frequency in the central nervous system (CNS) of three different rAAV vector... serotypes. The vectors contained AAV2 terminal repeats ... |
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Source: Kalil, Ronald E. - Neuroscience Training Program & W.M. Keck Laboratory for Biological Imaging, University of Wisconsin at Madison; Mandel, Ronald J. - Department of Neuroscience, University of Florida |
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Collection: Biology and Medicine |
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| 29 | original article The American Society of Gene & Cell Therapy Molecular Therapy 1 | ||
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Summary: , or AAV2/8-GFP pseudotyped vectors into the caudate nucleus and puta- men bilaterally in a pattern... , the adeno-associated virus (AAV) has become a common vector of choice to deliver genes aimed at modeling... for specific receptors that may confer tissue specificity. AAV ... |
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Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
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Collection: Biology and Medicine |
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| 30 | Intracellular trafficking of adeno-associated viral and JF Engelhardt1,2,3 | ||
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Summary: Adeno-associated virus (AAV) has attracted considerable interest as a gene therapy vector over the past... on the successful cloning of AAV established the foundation of recombi- nant AAV vectors capable of expressing... as recombinant vectors ... |
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Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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Collection: Biology and Medicine |
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| 31 | Recombinant Adeno-associated Viral Vectors as Therapeutic Agents to Treat Neurological Disorders | ||
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Summary: candidate vector for treating neurological disorders. Indeed, rAAV is currently being used in five early... viral vectors (rAAV) [4]. rAAV is one logical choice for early stage clinical develop- ment due to its... in the development of rAAV as a viral ... |
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Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
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Collection: Biology and Medicine |
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| 32 | JOURNAL OF VIROLOGY, Mar. 2002, p. 20432053 Vol. 76, No. 5 0022-538X/02/$04.00 0 DOI: 10.1128/JVI.76.5.20432053.2002 | ||
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Summary: was the first primate AAV to be cloned and has been under inten- sive development as a gene therapy vector... regulatory elements by using dual-vector heterodimerization approaches. Compared to the other serotypes, AAV... for development as a gene delivery vector (2, 5). Although less is ... |
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Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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Collection: Biology and Medicine |
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| 33 | JOURNAL OF VIROLOGY, Oct. 2006, p. 98319836 Vol. 80, No. 19 0022-538X/06/$08.00 0 doi:10.1128/JVI.00878-06 | ||
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Summary: Adeno-associated virus serotype 8 (AAV8) is currently emerging as a powerful gene transfer vector, owing... (AAV) is an increasingly popular gene transfer vector with a number of inherent advantages over other... contributes to the ver- satility of the AAV vector ... |
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Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 34 | HUMAN GENE THERAPY 16:299306 (March 2005) Mary Ann Liebert, Inc. | ||
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Summary: to avoid any loss of rAAV-vector transduced cells by cytotoxic T lymphocyte (CTL)-mediated immune response... serotype 2 (AAV-2). However, when this vector is delivered at high doses into the portal vein or hepatic... the relative level of transduction. First, we delivered an AAV ... |
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Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 35 | MOLECULAR THERAPY Vol. 6, No. 3, September 2002 Copyright The American Society of Gene Therapy | ||
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Summary: (rAAV2) Vectors Renius Owen IV, Ronald J. Mandel, Chandramohan V. Ammini, Thomas J. Conlon, Douglas S... defects in the E1 subunit of the pyru- vate dehydrogenase (PDH) complex (PDC), we constructed rAAV vectors... and episodic lactic acido- sis [2]. Recently, recombinant adeno-associated viral ... |
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Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
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Collection: Biology and Medicine |
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| 36 | SHORT COMMUNICATION Comparative biology of rAAV transduction in ferret, | ||
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Summary: (CF) lung disease, rAAV- based vectors have been tested successfully for the functional correction... vectors demonstrated considerable differences in the tropisms of these three serotypes.8 For example, rAAV... gene therapy in CF pig and ferret models. Using an AAV2 proviral ... |
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Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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Collection: Biology and Medicine |
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| 37 | HUMAN GENE THERAPY 18:344354 (April 2007) Mary Ann Liebert, Inc. | ||
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Summary: -associated virus (AAV) vectors in various somatic tissues has been shown to depend heavily on the AAV type from... which the vector capsid proteins are derived. Among the AAV types studied, AAV6 efficiently transduces... SUMMARY We have previously shown that ... |
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Source: Miller, Dusty - Division of Human Biology, Fred Hutchinson Cancer Research Center |
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Collection: Biology and Medicine |
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| 38 | Characterization of the Relationship of AAV Capsid Domain Swapping to Liver | ||
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Summary: Recombinant adeno-associated virus (AAV) vectors show promise for use in gene therapy. For liver-targeted gene... transfer in animals, AAV vectors pseudotyped with the AAV serotype 8 (AAV8) capsid have definite advantages... cassette. By comparing the transduction ... |
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Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 39 | JOURNAL OF VIROLOGY, Mar. 2004, p. 31103122 Vol. 78, No. 6 0022-538X/04/$08.00 0 DOI: 10.1128/JVI.78.6.31103122.2004 | ||
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Summary: (AAV2) vectors is ineffi- cient; less than 10% of hepatocytes are permissive for stable transduction... , and transgene expression is characterized by a lag phase of up to 6 weeks. AAV2-based vector genomes packaged... of the liver with vectors based on AAV2 capsids ... |
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Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 40 | correspondence NATURE|VOL424|17JULY2003|www.nature.com/nature 251 | ||
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Summary: 423, 573574: 2003) suggests that there is a reasonable probability that recombinant AAV vectors may... demonstrate that AAV vector DNA will preferentially integrate into active genes when delivered into the livers... suggests that recombinant AAV vectors may pose ... |
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Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 41 | HUMAN GENE THERAPY 15:405413 (April 2004) Mary Ann Liebert, Inc. | ||
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Summary: efficiency of these four vectors in the pancreas of mice in vivo. We showed that adenovirus, AAV2, and AAV8... leukocyte infiltration early after delivery into the pancreas, whereas none of the AAV vectors elicited... -associated virus (AAV) is a single-stranded DNA ... |
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Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 42 | JOURNAL OF VIROLOGY, Apr. 2005, p. 50475058 Vol. 79, No. 8 0022-538X/05/$08.00 0 doi:10.1128/JVI.79.8.50475058.2005 | ||
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Summary: , Bethesda, Maryland3 Received 14 July 2004/Accepted 17 November 2004 Adeno-associated virus (AAV... of AAV are in development for use in human gene therapy applications due to their ability to transduce... different target cells. The need to manipulate AAV capsids for specific tissue delivery has generated |
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Source: Baker, Timothy S. - Department of Chemistry and Biochemistry, University of California at San Diego |
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Collection: Biology and Medicine |
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| 43 | JOURNAL OF VIROLOGY, Apr. 2004, p. 33613371 Vol. 78, No. 7 0022-538X/04/$08.00 0 DOI: 10.1128/JVI.78.7.33613371.2004 | ||
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Summary: , and it has been studied extensively (37). AAV2-based vectors have been used to mediate factor IX gene... , in a similar vein, understand- ing the capsid structure of AAV viral vectors might facilitate the development... studies of additional AAV serotypes have the potential to broaden ... |
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Source: Baker, Timothy S. - Department of Chemistry and Biochemistry, University of California at San Diego |
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Collection: Biology and Medicine |
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| 44 | JOURNAL OF VIROLOGY, 0022-538X/99/$04.00 0 | ||
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Summary: . As a specific application of this finding, we generated adenovirusadeno-associated virus (AAV) hybrid vectors... , first-generation Ad vectors containing AAV inverted terminal repeat sequences (ITRs) flanking a reporter... the hybrid vector genome could mediate the formation of ... |
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Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 45 | JOURNAL OF VIROLOGY, 0022-538X/00/$04.00 0 | ||
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Summary: 2000/Accepted 28 July 2000 Recombinant adeno-associated virus (rAAV) vectors stably transduce... hepatocytes in experimental animals. Following portal-vein administration of rAAV vectors in vivo, single... vectors in the presence of bacterial PaeR7 or Dam methyltransferase or constructed ... |
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Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 46 | original article The American Society of Gene & Cell Therapy Molecular Therapy 1 | ||
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Summary: to adult striatal PTN expression and approximated peak endogenous developmental levels (P1). rAAV2/1 vector... striatum using recombinant adeno-associated virus 2/1 (rAAV2/1) vector pro- tects both SNpc DA neurons... that a single injec- tion of either rAAV2/1-PTN/green fluorescent ... |
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Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
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Collection: Biology and Medicine |
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| 47 | original article The American Society of Gene Therapy Molecular Therapy vol. 16 no. 10, 17031709 oct. 2008 1703 | ||
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Summary: 1709 oct. 2008 1703 Adeno-associated virus (AAV) vectors are extremely effective gene-delivery vehicles... mutagen- esis and selection, that successfully generated AAV2 vectors with novel gene-delivery properties... to differences in viral function, recombinant AAV ... |
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Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley |
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Collection: Materials Science ; Chemistry |
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| 48 | GENE THERAPY Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors | ||
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Summary: ) vectors for the liver-specific expres- sion of human blood coagulation factor IX (hFIX). Therefore, an AAV... , the resultant vector particles expressed high hFIX lev- els ranging from 36% (AAV-4) to more than 2000... at all doses. Interest- ingly, systemic administration of 2 1012 ... |
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Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 49 | Transposon-Based Mutagenesis Generates Diverse Adeno-Associated Viral Libraries with Novel Gene | ||
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Summary: "bookmarked" AAV cap gene is cloned into an AAV packaging vector. The selectable marker is then replaced... therapy; protein engineering. 1. Introduction Adeno-associated viral (AAV) vectors show tremendous... coupled with the high efficiency of AAV ... |
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Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley |
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Collection: Materials Science ; Chemistry |
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| 50 | original article The American Society of Gene Therapy Molecular Therapy vol. 17 no. 2, 285293 feb. 2009 285 | ||
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Summary: label. Both rAAV5 and rAAV1 vectors were capable of transducing a subset of long-lived Clara cells... suggest that rAAV may be a useful vector for gene targeting of airway stem/progenitor cells. Received 8... are diluted following cell division. However, some studies have demonstrated ... |
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Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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Collection: Biology and Medicine |
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| 51 | HUMAN GENE THERAPY 12:563573 (March 20, 2001) Mary Ann Liebert, Inc. | ||
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Summary: ,4 and KATHERINE PARKER PONDER1,5 ABSTRACT Although AAV vectors show promise for hepatic gene therapy, the optimal... transcriptional regulatory ele- ments have not yet been identified. In this study, we show that an AAV vector... injection than an AAV vector with the ... |
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Source: Ponder, Katherine P. - Departments of Medicine & Biochemistry and Molecular Biophysics, Washington University in St. Louis |
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Collection: Biology and Medicine |
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| 52 | JOURNAL OF VIROLOGY, 0022-538X/01/$04.00 0 DOI: 10.1128/JVI.75.16.76627671.2001 | ||
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Summary: by these two vectors in undifferentiated myoblasts, pseudotyped rAAV- 2cap5 demonstrated dramatically enhanced... the nonviral and viral vectors used in muscle gene transfer, recombinant adeno-associated virus type 2 (rAAV-2... , various strategies have been under development to enhance the potency of ... |
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Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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Collection: Biology and Medicine |
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| 53 | original article 594 www.moleculartherapy.org vol. 18 no. 3, 594600 mar. 2010 | ||
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Summary: stronger than AAV1 capsid. This dual vector indexing approach may be useful in selecting lead vector... ,differentiatedairwayepithelialcellcultureshavebeenused to evaluate the relative efficiency of recombinant adeno-associated viral (rAAV) vectors across a range... species, whereas ... |
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Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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Collection: Biology and Medicine |
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| 54 | Progress in Direct Striatal Delivery of L-Dopa via Gene Therapy for Treatment of Parkinson's Disease Using Recombinant | ||
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Summary: -dihydroxyphenylalanine (L-dopa) in PD utilizing the recombinant adeno-associated viral vector (rAAV) system. The concept... -MEDIATED GENE EXPRESSION Description of the rAAV Vector Wild-type AAV (wtAAV) is a single-stranded DNA human... as a gene transfer vehicle. ... |
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Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
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Collection: Biology and Medicine |
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| 55 | JOURNAL OF VIROLOGY, Jan. 2005, p. 214224 Vol. 79, No. 1 0022-538X/05/$08.00 0 doi:10.1128/JVI.79.1.214224.2005 | ||
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Summary: adeno-associated virus (rAAV) vectors can mediate long-term stable transduction in various target... tissues. However, with rAAV serotype 2 (rAAV2) vectors, liver transduction is confined to only a small... whether rAAV vectors of other serotypes ... |
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Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 56 | JOURNAL OF VIROLOGY, July 2003, p. 73617366 Vol. 77, No. 13 0022-538X/03/$08.00 0 DOI: 10.1128/JVI.77.13.73617366.2003 | ||
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Summary: -length conventional AAV EGFP vectors suggested that second-strand synthesis of rAAV genomes was not rate limiting... . Recombinant adeno-associated virus (rAAV) is one of the most promising vectors for gene delivery to airway... epithelia for the gene therapy of cystic fibrosis (8). The ... |
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Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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Collection: Biology and Medicine |
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| 57 | LONG-TERM RESTORATION OF STRIATAL l-AROMATIC AMINO ACID DECARBOXYLASE ACTIVITY USING RECOMBINANT | ||
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Summary: EXPERIMENTAL PROCEDURES Recombinant vector production The cytomegalovirus (CMV)LacZ rAAV vector (pdx- 31 Lac... of the human TH (hTH) vector (rAAV MDhTH) has been described previously.30 The human AADC cDNA was generated... cassette was inserted between the AAV ... |
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Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
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Collection: Biology and Medicine |
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| 58 | JOURNAL OF VIROLOGY, Oct. 2007, p. 1130411321 Vol. 81, No. 20 0022-538X/07/$08.00 0 doi:10.1128/JVI.01225-07 | ||
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Summary: will not only enhance our understanding of the biology of rAAV vectors, which is essential for successful human... . The plasmid pVme DhF.IX carried the AAV-EF1 -hF.IX (human coagulation fac- tor IX-expressing AAV vector under... that there was approximately 0.1 rAAV ... |
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Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 59 | NATURE BIOTECHNOLOGY VOL 18 MAY 2000 http://biotech.nature.com 527 Recombinant adeno-associated virus (rAAV) vectors based on AAV | ||
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Summary: -associated virus (rAAV) vectors based on AAV type 2 can safely transduce various tissues and result in persistent... into the portal vasculature, rAAV vector genomes enter almost all the hepatocytes, yet only about 5% become stably... , fluorescent in situ hybridization (FISH), and ... |
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Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 60 | PEG Conjugation Moderately Protects Adeno-Associated Viral Vectors Against | ||
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Summary: : AAV gene therapy vectors have significant clinical promise, but serum neutralization poses a chal... con- trolled. ß 2005 Wiley Periodicals, Inc. Keywords: AAV; viral vector; polyethylene glycol; PEG... the preclinical and clinical level as deli- very agents for human gene transfer. Recombinant ... |
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Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley |
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Collection: Materials Science ; Chemistry |
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| 61 | JOURNAL OF VIROLOGY, 0022-538X/99/$04.00 0 | ||
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Summary: understood. To investigate these mechanisms, we have utilized a re- combinant AAV (rAAV) shuttle vector... increase the utility of rAAV vectors for gene therapy. Adeno-associated virus (AAV) is a nonpathogenic... ). rAAV ... |
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Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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Collection: Biology and Medicine |
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| 62 | HUMAN GENE THERAPY 10:591 602 (March 1, 1999) Mary Ann Liebert, Inc. | ||
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Summary: , and nonpatho- genic characteristics are several reasons why AAV is favored as a vector for gene therapy... has be- come a salient feature promoting the use of rAAV vector in gene threapy (Alexander et al... , an rAAV shuttle vector con- taining the ampicillin ... |
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Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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Collection: Biology and Medicine |
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| 63 | JOURNAL OF VIROLOGY, Oct. 2007, p. 1129011303 Vol. 81, No. 20 0022-538X/07/$08.00 0 doi:10.1128/JVI.00963-07 | ||
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Summary: shown that recombinant adeno-associated virus (rAAV) vector integrates preferen- tially in genes, near... with rAAV2 or rAAV8 vector. The results support our previous observations, but notably they have revealed... -stranded DNA genome. Recombinant AAV ... |
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Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 64 | ORIGINAL ARTICLE Surface immobilization of hexa-histidine-tagged | ||
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Summary: , Berkeley, CA, USA Adeno-associated viral (AAV) vectors, which are undergoing broad exploration in clinical... . This immobilized AAV vector system mediates high-efficiency delivery to cells that contact the surface and thus may... , respectively. Recombinant AAV ... |
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Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley |
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Collection: Materials Science ; Chemistry |
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| 65 | Investor and Media Contact: Stacie D. Byars | ||
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Summary: OF RECOMBINANT AAV VECTORS Seattle, WA October 18, 2006 Targeted Genetics Corporation (Nasdaq: TGEN) today... with which adeno-associated viral (AAV) vectors deliver genes to target cells. This approach covers small... molecule compounds and methods that enhance the trafficking of ... |
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Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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Collection: Biology and Medicine |
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| 66 | HUMAN GENE THERAPY 19:6169 (January 2008) Mary Ann Liebert, Inc. | ||
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Summary: of im- mune response against the vector (Flotte et al., 2007). New AAV serotypes have demonstrated... intravenous or intraperitoneal in- jection in wild-type neonatal mice. MATERIALS AND METHODS rAAV vector... signal. Both expression cassettes were contained within one AAV virion. For ... |
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|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
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Collection: Biology and Medicine |
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| 67 | JOURNAL OF VIROLOGY, Mar. 2004, p. 28632874 Vol. 78, No. 6 0022-538X/04/$08.00 0 DOI: 10.1128/JVI.78.6.28632874.2004 | ||
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Summary: . Recently, it has been reported that rAAV-5 vectors more effectively transduce murine lungs. Intranasal... ) and differentiated at an air-liquid interface. Recombinant AAV vectors and viral infections. Viral stocks of rAAV... - alovirus (CMV) immediate-early promoter. Full-length ... |
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|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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Collection: Biology and Medicine |
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| 68 | JOURNAL OF VIROLOGY, 0022-538X/00/$04.00 0 | ||
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Summary: advantages over first-generation adenovirus vectors. However, AAV infection from the apical surfaces of well... of vector delivered was also low. Thus, delivery of AAV as a CaPi coprecipitate mark- edly enhanced AAV... of recombinant AAV vectors. ... |
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|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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Collection: Biology and Medicine |
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| 69 | Virus Research 104 (2004) 5159 Novel approaches to augment adeno-associated virus | ||
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Summary: therapy vector. To this end, the current study has investigated novel methods for augmenting rAAV... why AAV is favored as a vector for gene therapy (Alexander et al., 1994; Kaplitt et al., 1994; Lai et... Available online 27 April 2004 Abstract Recombinant adeno-associated virus (rAAV) ... |
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|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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Collection: Biology and Medicine |
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| 70 | Current Gene Therapy, 2007, 7, 347-360 347 1566-5232/07 $50.00+.00 2007 Bentham Science Publishers Ltd. | ||
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Summary: Responses Against AAV Vectors Injected into the Brain Adeno-associated virus (AAV) is a small single... response when injecting lower titers of rAAV2 vectors (2 x 108 particles [Mandel et al., 1998], 4 x 108... to single injections of rAAV2 into naïve rats, ... |
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|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
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|
Collection: Biology and Medicine |
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| 71 | Long-Term rAAV-Mediated Gene Transfer of GDNF in the Rat Parkinson's Model: Intrastriatal But Not Intranigral Transduction | ||
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Summary: used recombinant adeno-associated vi- ral (rAAV) vectors to deliver glial cell line... . Here, we have used an rAAV- GDNF vector to express GDNF long-term (6 months) in either the nigral DA... levels throughout this pe- riod. These results provide evidence that rAAV is a highly efficient ... |
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|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
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|
Collection: Biology and Medicine |
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| 72 | JOURNAL OF VIROLOGY, Jan. 2006, p. 426439 Vol. 80, No. 1 0022-538X/06/$08.00 0 doi:10.1128/JVI.80.1.426439.2006 | ||
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Summary: IX) gene flanked by ITRs from AAV genotypes 1 through 6. Initial in vitro analyses of gfp vector DNA... and can be freely substituted. This corroborates our current model for AAV vector persistence in the liver... and provides useful information for the future design and application of recombinant ... |
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|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 73 | Loss of ATM Function Enhances Recombinant Adeno-Associated Virus Transduction and Integration through Pathways Similar to UV Irradiation | ||
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Summary: features have made rAAV the vector of choice for many applica- tions in gene therapy (Flotte et al., 1994... , we tested whether rAAV vectors have an altered transduction pro- file in ATM-deficient cell lines... compared with their wild- type counterparts. To test this hypothesis, we used an ... |
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|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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|
Collection: Biology and Medicine |
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| 74 | HUMAN GENE THERAPY 18:871880 (October 2007) Mary Ann Liebert, Inc. | ||
|
Summary: in the rat retina. We used two viral vectors, based on lentivirus and adeno-associated virus (AAV... (AAV) vectors can be used to deliver shRNAs to the rat retinal pigment epithelium (RPE) in vivo... fragment of the mouse opsin promoter driving GFP, and a "stabilized double-stranded" AAV ... |
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|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
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|
Collection: Biology and Medicine |
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| 75 | MOLECULAR AND CELLULAR BIOLOGY, May 2003, p. 35583565 Vol. 23, No. 10 0270-7306/03/$08.00 0 DOI: 10.1128/MCB.23.10.35583565.2003 | ||
|
Summary: that infection with recombinant adeno-associated virus (rAAV) vectors can mediate gene targeting in somatic cells... gene targeting frequencies reach 0.8% with a dual vector system in which one rAAV vector provides... that combining rAAV vectors ... |
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|
Source: Baltimore, David - Division of Biology, California Institute of Technology |
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Collection: Biotechnology ; Biology and Medicine |
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| 76 | Long-term Skeletal Muscle Protection After Gene Transfer in a Mouse Model of LGMD-2D | ||
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Summary: × 1011 vector genomes (vg) of rAAV2/1-tMCK-sgca in adult sgca-/- tibialis anterior and extensor... Society of Gene Therapy AAV1 Correction of LGMD-2A Muscle achieves wide vector distribution and its effect... -/- mouse at 4, 7, 10, and 13 weeks after administration of the ... |
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|
Source: Campbell, Kevin P. - Department of Physiology and Biophysics, University of Iowa |
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Collection: Biology and Medicine |
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| 77 | JOURNAL OF VIROLOGY, 0022-538X/99/$04.00 0 | ||
|
Summary: -associated virus (rAAV) vectors for gene therapy of inherited disorders have demon- strated considerable potential... with this vector. However, the mechanisms by which AAV-2 enters cells remain unknown. In the present report, we... with either Ad.LacZ or Ad.K44Adynamin for 48 h prior to infection with an ... |
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|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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|
Collection: Biology and Medicine |
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| 78 | Introduction Cystic fibrosis (CF) is the most common inherited dis- | ||
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Summary: , not only with rAAV-2, but also with all other types of viral vectors currently in use (1, 2). Several... will target the lung airway epithelium. Sev- eral viral vectors, including adenovirus, lentivirus, retro... - virus, and adeno-associated virus (AAV), have been used in strategies conceived for functional |
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|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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|
Collection: Biology and Medicine |
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| 79 | HUMAN GENE THERAPY 18:367378 (April 2007) Mary Ann Liebert, Inc. | ||
|
Summary: , recombinant (r)AAV2 vectors have been explored in numerous clinical trials (Kay et al., 2000; Manno et al... challenge with rAAV vectors involves the development of a scalable, high-throughput purification protocol... insertion, that generate high-titer rAAV vector ... |
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|
Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley |
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|
Collection: Materials Science ; Chemistry |
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| 80 | HUMAN GENE THERAPY 18:8187 (January 2007) Mary Ann Liebert, Inc. | ||
|
Summary: . ENGELHARDT13 ABSTRACT We have previously demonstrated that hybrid adeno-associated viral (AAV) vectors... and the efficiency of dual- AAV vector trans-splicing in cultured cells. Using hybrid-ITR vectors carrying two exons... -ITR AAV vectors ... |
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|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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|
Collection: Biology and Medicine |
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| 81 | Behavioral/Systems/Cognitive The Role of CA3 Hippocampal NMDA Receptors in Paired | ||
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Summary: . This has limited AAVCre expression to 1030% of total CA3 in dorsal hip- pocampus and avoided the spread... subfields of the hippocampus. Here, using a viral vector to temporally control a focal deletion of the NR1... contingencies. We used a viral vector technique (Scammell et al., 2003) to induce a ... |
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|
Source: Wagner, Anthony - Department of Psychology, Stanford University |
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Collection: Biology and Medicine |
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| 82 | JOURNAL OF VIROLOGY, 0022-538X/99/$04.00 0 | ||
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Summary: January 1999/Accepted 30 March 1999 Recombinant adeno-associated virus (rAAV) vectors allow for sustained... vector expressing human coagulation factor F.IX (hF.IX), AAV-EF1 -F.IX (hF.IX expression was controlled... vein, or directly into the liver parenchyma, and the forms of rAAV ... |
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|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 83 | ELSEVIER Virus Research 48 (1997) 41-56 Structural and functional heterogeneity of integrated | ||
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Summary: November 1996 Abstract Adeno-associated Virus (AAV) has emerged as a promising vector for gene therapy... of transcomplementing methods of virus propagation has allowed for the generation of recombinant AAV vectors for which... ). These methods have set the stage for the use of AAV as a ... |
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|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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Collection: Biology and Medicine |
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| 84 | JOURNAL OF VIROLOGY, 0022-538X/00/$04.00 0 | ||
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Summary: as a gene therapy vector (1, 22, 29, 63). Recombinant AAV-2 (rAAV) carrying reporter genes has been shown... Analysis of purity of rAAV stocks. The tgAAVCF vector, an AAV-2 vector encoding the cystic fibrosis... /Accepted 14 July 2000 ... |
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|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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|
Collection: Biology and Medicine |
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| 85 | JOURNAL OF VIROLOGY, 0022-538X/01/$04.00 0 DOI: 10.1128/JVI.75.15.69696976.2001 | ||
|
Summary: of a series of hF.IX-expressing rAAV vectors. At doses of 2.4 1011 to 3.0 1011 vector genomes per mouse (n 12... uncertainties as to whether rAAV-mediated hepatic gene expression can persist lifelong after a single vector... -stranded (ss) DNA genome of approximately 4.7 kb. Recombinant ... |
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|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
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|
Collection: Biology and Medicine |
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| 86 | 2003 NaturePublishing Group 346 | MAY 2003 | VOLUME 4 www.nature.com/reviews/genetics | ||
|
Summary: -associated viruses(AAV) Most rAAV vectors have been derived fromAAV2,but,so far,a total of eight distinctAAV... 49 .Pseudotyping the rAAV2 vector genome with capsids from alternative serotypes to achieve more efficient gene... to efficient ... |
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|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
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|
Collection: Biology and Medicine |
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| 87 | The idea of gene-based therapeutics has been around for some time, but it only received serious attention | ||
|
Summary: that contain AAV8-specific epitopes. Non-viral vectors may induce humoral responses directed against a protein... of efficacy in several different cancer trials117,118 . AAV vectors The properties of adeno-associated virus... (AAV) vectors have made them less ... |
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|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 88 | Gene Therapy (1999) 6, 14271437 1999 Stockton Press All rights reserved 0969-7128/99 $12.00 | ||
|
Summary: AAV (rAAV) has demonstrated consider- able promise as a gene therapy vector for certain organ systems... with this vector in vivo. Materials and methods Generation of recombinant AAV stocks The cis-acting plasmid (p... derived from the luciferase gene of the pGL3-basic ... |
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|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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|
Collection: Biology and Medicine |
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| 89 | NATURE BIOTECHNOLOGY VOLUME 25 NUMBER 10 OCTOBER 2007 1111 and drugs that act directly on disease genes. | ||
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Summary: and hubs. AAV vectors and tumorigenicity Mark A Kay Chromosomal integration of rAAV vectors may induce... therapy, recombinant adeno- associated viral (rAAV) vectors have shown special promise because... vectors2. The authors found that neonatal ... |
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|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
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Collection: Biology and Medicine |
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| 90 | GENE THERAPY AAV-mediated factor IX gene transfer to skeletal muscle in patients with | ||
|
Summary: of a recombinant adeno-associated viral (rAAV) vector ex- pressing F.IX. In this study we investi- gated the safety... administration of rAAV demon- strate that administration of AAV vector by the intramuscular route is safe... (AAV) vectors ... |
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|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
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|
Collection: Biology and Medicine |
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| 91 | Copyright Lippincott Williams & Wilkins. Unauthorized reproduction of this article is prohibited. Gene therapy for hemophilia | ||
|
Summary: -associated virus vector (AAV vector), adenoviral vector, gene therapy, hemophilia, retroviral vector Curr Opin... . The status of the liver-directed adeno-associated virus (AAV) vector-mediated gene therapy trial in humans... - peutic gene. ... |
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|
Source: Ponder, Katherine P. - Departments of Medicine & Biochemistry and Molecular Biophysics, Washington University in St. Louis |
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|
Collection: Biology and Medicine |
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| 92 | original article 2114 www.moleculartherapy.org vol.15no.12,21142123dec.2007 | ||
|
Summary: attracted considerable interest as a vector for treat- ing CF lung disease. Indeed, rAAV-based vectors have... 14 To date, a variety of rAAV vector serotypes have been tested as gene trans- fer vectors.15 However... the detection of ... |
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|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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|
Collection: Biology and Medicine |
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| 93 | original article The American Society of Gene & Cell Therapy Molecular Therapy vol. 18 no. 5, 873880 may 2010 873 | ||
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Summary: (AAV) vectors has resulted in sus- tained increased NAGLU activity, reduced glycosaminoglycan... - mal enzyme levels produced by the AAV vector in the brain and the contribution of normal donor marrow... reported studies using rAAV vectors in the MPS IIIB mouse.1315 ... |
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|
Source: Orrock, John - Department of Zoology, University of Wisconsin at Madison |
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|
Collection: Environmental Sciences and Ecology ; Biology and Medicine |
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| 94 | Species-Specific Differences in Mouse and Human Airway Epithelial Biology of Recombinant | ||
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Summary: immunogenicity. To date, at least nine different serotypes of rAAV vectors (types 19) have been used to express... recombinant genes (19). How- ever, rAAV serotypes 2 and 5 are the most extensively studied vectors for gene... infection with rAAV5 vectors were ... |
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|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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|
Collection: Biology and Medicine |
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| 95 | Vectors of Gene Therapy KATHERINE PARKER PONDER, M.D. | ||
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Summary: (AAV). Other viral vectors that have been less extensively used are derived from herpes simplex virus 1... CHAPTER 4 Vectors of Gene Therapy KATHERINE PARKER PONDER, M.D. INTRODUCTION Currently, gene... into a cell are vehicles called vectors. Vectors can be divided into viral and ... |
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|
Source: Ponder, Katherine P. - Departments of Medicine & Biochemistry and Molecular Biophysics, Washington University in St. Louis |
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|
Collection: Biology and Medicine |
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| 96 | JOURNAL OF VIROLOGY, 0022-538X/98/$04.00 0 | ||
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Summary: Adeno-associated viral (AAV) vectors have demonstrated great utility for long-term gene expression... remain unclear. Using a recombinant shuttle vector, we have demonstrated that circularized rAAV interme... for the gener- ation of recombinant vectors (28, 29). In contrast to ... |
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|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
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|
Collection: Biology and Medicine |
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| 97 | Construction of diverse adeno-associated viral libraries for directed evolution of enhanced gene | ||
|
Summary: transfer properties of these viruses are needed. AAV vectors have proven to be safe and efficient gene... utilized as a gene delivery vector79. In addition, the recent discovery of the crystal structure of AAV2... by crystal structure data, has led to the creation of recombinant AAV2 ... |
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|
Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley |
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|
Collection: Materials Science ; Chemistry |
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| 98 | Sites in the AAV5 capsid tolerant to deletions and tandem duplications Kaoru Hida a | ||
|
Summary: s t r a c t Gene therapy vectors based on adeno-associated virus (AAV) have shown much promise... reserved. 1. Introduction Adeno-associated virus (AAV)1 is considered one of the most promising vectors... in clinical trials with recombinant AAV serotype 2 (rAAV2) ... |
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|
Source: Gray, Jeffrey J. - Department of Biomolecular and Chemical Engineering, Johns Hopkins University; Ostermeier, Marc - Department of Biomolecular and Chemical Engineering, Johns Hopkins University |
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|
Collection: Biology and Medicine ; Biotechnology ; Chemistry |
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| 99 | JOURNAL OF VIROLOGY, Nov. 2002, p. 1134311349 Vol. 76, No. 22 0022-538X/02/$04.00 0 DOI: 10.1128/JVI.76.22.1134311349.2002 | ||
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Summary: with rAAV vectors, we injected mice with two different human factor IX or Escherichia coli lac... Z-expressing AAV serotype 2-based vectors at doses ranging between 4.0 108 and 1.1 1013 vector genomes (vg... administration into ex- perimental animals. Recombinant AAV ... |
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|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
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|
Collection: Biology and Medicine |
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| 100 | JOURNAL OF VIROLOGY, Mar. 2005, p. 36063614 Vol. 79, No. 6 0022-538X/05/$08.00 0 doi:10.1128/JVI.79.6.36063614.2005 | ||
|
Summary: 2004 Recombinant adeno-associated virus (rAAV) vector holds promise for gene therapy. Despite a low... animals and human subjects (14). rAAV2 vectors have the ability to inte- grate into host chromosomes... to carefully consider rAAV-mediated insertional mutagenesis because of the ... |
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|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
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|
Collection: Biology and Medicine |
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