| Sample search results for: aav based gene |
| 1 | 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... , while AAV2/1 predominantly transduced the RPE. To more thoroughly compare the efficiencies of gene... that varying genome length and AAV capsids may allow for ... |
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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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| 2 | A Two-hybrid Screen Identifies Cathepsins B and L as Uncoating Factors for Adeno-associated Virus 2 | ||
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Summary: gene activation with the AAV5-based capsid bait was not significantly different from the empty plasmid... for human gene therapy, based on their unique tissue tropisms and distinct immuno- logical profiles... of a Gal4p-dependent lacZ reporter gene in yeast ... |
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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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| 3 | Current Gene Therapy, 2003, 3, 281-304 281 1566-5232/03 $41.00+.00 2003 Bentham Science Publishers Ltd. | ||
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Summary: Abstract: Gene transfer vectors based on the human adeno-associated virus serotype 2 (AAV-2) have been... - clinical testing of novel gene transfer vectors based on AAV serotypes (Part II). PART I: FROM VIRUS... -associated Virus (AAV) as Novel ... |
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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 | Recombinant AAV Viral Vectors Pseudotyped with Viral Capsids from Serotypes 1, 2, and 5 Display | ||
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Summary: @ufl.edu. Available online 1 July 2004 Recombinant adeno-associated virus 2 (rAAV2) has been shown to deliver genes... suggest that vectors based on distinct AAV serotypes can be chosen for specific applications... in the nervous system. Key Words: AAV, capsid, serotype, ... |
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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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| 5 | original article The American Society of Gene & Cell Therapy Molecular Therapy 1 | ||
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Summary: a promising candidate vector for the gene therapy. Recombinant vectors based on AAV2 capsid proteins have been... -specific nuclear protein (NeuN)]. The current data suggest that AAV2/5 and AAV2/1 are supe- rior to AAV2/8 for gene... , the ... |
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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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| 6 | SHORT COMMUNICATION Comparative biology of rAAV transduction in ferret, | ||
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Summary: - associated virus (rAAV)-mediated gene therapies to the lung for cystic fibrosis (CF). As new ferret and pig... (CF) lung disease, rAAV- based vectors have been tested successfully for the functional correction... to demonstrate detectable rAAV2 transduction (based ... |
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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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| 7 | 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... viral genes are the inverted terminal repeats (ITRs, 145 bp in length for AAV2) at either end... foreign genes in mammalian cells.13,14 Currently, eight serotypes 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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| 8 | 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... the processes behind AAV8 transduction is important for its application and optimal use in human gene therapy... with its robust transduction effi- ciency, our findings support the continued ... |
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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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| 9 | 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: augmented rAAV- 5-mediated gene transfer. Increased transgene expression was independent of viral genome... to be determined. AAV is currently considered an ideal vehicle for human gene therapy, as it is a small, defective... was the first primate AAV to be cloned and has been under ... |
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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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| 10 | Helper Virus-Free, Optically Controllable, and Two-Plasmid-Based Production of Adeno-associated | ||
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Summary: , and based on transfection of only two plasmids, i.e., an AAV vector construct and one of six novel AAV... helper plasmids. The latter were engineered to carry AAV serotype rep and cap genes together... -term gene expression in a large variety of tissues [1]. This ... |
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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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| 11 | Characterization of the Relationship of AAV Capsid Domain Swapping to Liver | ||
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Summary: based on other AAV serotypes are being studied for bet- ter gene transfer performance. In a scenario... Recombinant adeno-associated virus (AAV) vectors show promise for use in gene therapy. For liver-targeted gene... genes contain- ing exchanged ... |
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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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| 12 | HUMAN GENE THERAPY 15:405413 (April 2004) Mary Ann Liebert, Inc. | ||
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Summary: will be useful for studying novel gene delivery based treatments in animal models for diabetes and other... , and lung, resulting in long-term gene expressionfrom these tissues(Carter and Samulski, 2000). Ex vivo, AAV... - tion of the pAAV plasmid with DF5 (containing ... |
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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 | 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: , 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 is uncoating of vector genomes in the nucleus. The majority... before maximal gene expression. Vector genomes packaged inside ... |
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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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| 14 | Expert Review Designer Gene Delivery Vectors: Molecular Engineering and Evolution | ||
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Summary: desirable gene delivery targets, including stem cells, are refractory to vectors based on AAV2 (40... ; accepted August 3, 2007; published online 1 September 2007 Abstract. Gene delivery vectors based on adeno... vectors, particularly those based on ... |
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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, 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... are similar. A pseudo-atomic model generated for AAV5 based on the crystal structure of AAV2 and constrained... interactions among parvoviruses. Recombinant ... |
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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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| 16 | original article 2088 www.moleculartherapy.org vol. 17 no. 12, 20882095 dec. 2009 | ||
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Summary: creation of AAV vec- tors with valuable new gene delivery properties. We have engineered novel AAV variants... for the structural proteins VP1-3 that assemble into the icosahedral viral capsid.15 AAV vec- tors based... Engineering the AAV capsid for targeted delivery ... |
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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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| 17 | 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... potentially yield unlimited numbers of new AAV variants with novel gene-delivery properties, and subsequent... of gene-delivery properties, existing 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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| 18 | 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: of AAV are in development for use in human gene therapy applications due to their ability to transduce... reconstruction. A pseudoatomic model was built for the AAV4 capsid by use of a structure-based sequence alignment... reconstruction. Using this reconstruction and a structure-based ... |
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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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| 19 | JOURNAL OF VIROLOGY, 0022-538X/99/$04.00 0 | ||
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Summary: of Ad.AAV1. In contrast to our study, these Ad-based hybrid vectors contained viral genes that ex- press... gene cassette inserted into the E1 region. We hypothe- sized that the AAV ITRs present within... -associated virus vectors (rAAV) have been developed by ... |
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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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| 20 | Robust Systemic Transduction with AAV9 Vectors in Mice: Efficient Global Cardiac Gene Transfer Superior | ||
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Summary: other than AAV type 2. J. Virol. 72: 309 319. 8. Xiao, W., et al. (1999). Gene therapy vectors based... Robust Systemic Transduction with AAV9 Vectors in Mice: Efficient Global Cardiac Gene Transfer... as rAAV8, is a robust vector for gene ... |
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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 | JOURNAL OF VIROLOGY, 0022-538X/00/$04.00 0 | ||
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Summary: strategies for improving rAAV-mediated gene transfer in vivo. Recombinant adeno-associated virus (rAAV... -stranded (ss) rAAV genomes. Following intraportal administration of vector into animals, rAAV-mediated gene... -galactosidase driven by the human elongation ... |
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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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| 22 | original article The American Society of Gene Therapy Molecular Therapy vol. 17 no. 2, 285293 feb. 2009 285 | ||
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Summary: . 2009 285 Although recombinant adeno-associated virus (rAAV) has been widely used in lung gene therapy... suggest that rAAV may be a useful vector for gene targeting of airway stem/progenitor cells. Received 8... promise for efficient gene delivery to both dividing and nondividing cells ... |
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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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| 23 | GENE THERAPY Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors | ||
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Summary: the development of AAV pseudotype-based gene therapies for he- mophilia B and other liver-related dis- eases... that vectors based on AAV serotypes might offer 3 important benefits for clinical liver-directed gene transfer... -2 genome encoding the hfIX ... |
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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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| 24 | JOURNAL OF VIROLOGY, 0022-538X/01/$04.00 0 DOI: 10.1128/JVI.75.16.76627671.2001 | ||
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Summary: 52242 Received 22 March 2001/Accepted 11 May 2001 Adeno-associated virus (AAV)-based muscle gene therapy... a significant impact on improving rAAV-mediated gene therapy in muscle. Muscle-based gene therapy protocols have... . For these reasons, efforts aimed at ... |
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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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| 25 | Current Gene Therapy, 2001, 1, 137-147 137 1566-5232/00 $20.00+.00 2001 Bentham Science Publishers Ltd. | ||
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Summary: Publishers Ltd. Rate Limiting Steps of AAV Transduction and Implications for Human Gene Therapy S. Sanlioglu1... : Despite the fact that adeno-associated virus type 2 (AAV2) is an extremely attractive gene therapy vector... ). Despite its many attractive features, further optimization 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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| 26 | 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: -type AAV-2, AAV-8, or AAV-9 cap together with AAV-2 rep genes, as well as AAV-2-based vector plasmids... as two powerful and compatible approaches to the molecular evolution of novel AAV vectors for human ... |
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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 | 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 | ||
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Summary: that infection with recombinant adeno-associated virus (rAAV) vectors can mediate gene targeting in somatic cells... , but the mechanism is unclear. This paper explores the balance between random integration and gene targeting with rAAV... stimulates the frequency of rAAV-mediated ... |
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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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| 28 | original article 594 www.moleculartherapy.org vol. 18 no. 3, 594600 mar. 2010 | ||
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Summary: - tages, they would both operate in favor of improved efficacy of rAAV1-based vectors for gene therapy... Society of Gene & Cell Therapy Selecting the most efficient recombinant adeno- associated virus (rAAV... serotypes for clinical gene therapy and suggests ... |
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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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| 29 | Capsid modifications overcome low heterogeneous expression of heparan sulfate proteoglycan that limits AAV2-mediated gene transfer and therapeutic | ||
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Summary: as gene transfer vectors. However, vectors based on AAV type-2 (AAV2) are the most widely used and best... utilized these vectors in an assay based on competitive inhibition of AAV2-mediated gene delivery... that limits ... |
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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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| 30 | JOURNAL OF VIROLOGY, 0022-538X/00/$04.00 0 | ||
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Summary: for gene transfer to the airway epithelia in vivo. Adeno-associated viruses (AAVs) show promise as gene... not encode viral genes, they do not elicit a cell-mediated immune response (47, 12, 17). Thus, AAV has some... -coxsackie adenovirus receptor and penton ... |
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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 | 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: . 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 most commonly studied AAV vector for gene... of infection with rAAV-2 and ... |
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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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| 32 | ORIGINAL ARTICLE Surface immobilization of hexa-histidine-tagged | ||
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Summary: have promise for localized gene delivery, which may aid numerous applications of AAV delivery to gene... therapy. Gene Therapy advance online publication, 27 May 2010; doi:10.1038/gt.2010.81 Keywords: AAV... that used AAV2-mediated gene delivery ... |
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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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| 33 | JOURNAL OF VIROLOGY, 0022-538X/99/$04.00 0 | ||
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Summary: frame 6 gene product decreased the abundance of AAV circular intermediates, favoring instead the linear... increase the utility of rAAV vectors for gene therapy. Adeno-associated virus (AAV) is a nonpathogenic... ). Recombinant AAV (rAAV) has ... |
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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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| 34 | 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: -stranded linear rAAV genomes capped with covalently closed hairpins at termini. The 5 end of 3-base hairpin loops... of DNA-PKcs and Artemis in AAV-ITR hairpin metabolism in mice. Based on the previous observa- tions... . No-end double- stranded linear monomer rAAV genome capped with ... |
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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 | 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: muscles, and brain. Thus, rAAV8 is a robust vector for gene transfer to the liver and provides a promising... research tool for delivering genes to various target organs. In addition, the rAAV8 vector may offer... ). Among the vectors used to deliver genes to hepatocytes 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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| 36 | [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... , the field of nucleic acid-based inhibitors of gene expression has seen an unprecedented wave of interest... this hurdle and put such a delivery tool in place is central to establishing ... |
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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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| 37 | HUMAN GENE THERAPY 10:591 602 (March 1, 1999) Mary Ann Liebert, Inc. | ||
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Summary: are the foundation of developing new and innovative strategies to im- prove the efficiency of rAAV as a gene therapy... SUMMARY Adenovirus E4orf6 gene expression has been demonstrated to enhance rAAV transduction, leading... , hydroxyurea, and UV have also been shown to increase gene ... |
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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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| 38 | 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... , com- plete rAAV expression cassette. The mechanism of the enhanced gene expression from two vec- tors... expression was higher than expected based on ... |
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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 | Directed evolution of adeno-associated virus to an infectious respiratory virus | ||
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Summary: end points, of gene transfer vectors based on respiratory viruses for respiratory diseases (3... advanced methods to successfully engineer novel virus-based gene therapeutics. Directed evolution... for replication and gene expression. Wild-type AAV is a 4.7 kb ... |
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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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| 40 | Enhanced Preparation of Adeno-Associated Viral Vectors by Using High Hydrostatic Pressure to | ||
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Summary: capsid shell that encloses a single-stranded 4.7 kb DNA genome. Gene delivery vectors based on AAV... based on adeno-asso- ciated virus (AAV) have significant therapeutic potential, but much room... with either helper virus, such as adenovirus, or plasmids containing helper ... |
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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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| 41 | Virus Research 104 (2004) 5159 Novel approaches to augment adeno-associated virus | ||
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Summary: of rAAV transduction as assessed by gene expression. Based on the hypothesis that Rac1 was a central... ; Endocytosis; Rac 1; Gene therapy; PDTC 1. Introduction Adeno-associated virus (AAV) is a non-pathogenic hu... why AAV is favored as a vector for ... |
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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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| 42 | Investor and Media Contact: Stacie D. Byars | ||
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Summary: with which adeno-associated viral (AAV) vectors deliver genes to target cells. This approach covers small... . "As a company focused on developing AAV-based products for important therapeutic indications, we... to increase the therapeutic effect of AAV-based products and also may allow for smaller doses, ... |
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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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| 43 | 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: rAAV transduction in airway epithelia for gene therapy of cystic fibrosis. Adeno-associated virus... target has been an area of intense interest to gene therapy. Inefficiencies in re- combinant AAV (rAAV... for the clinical testing of rAAV-mediated ... |
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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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| 44 | Introduction Cystic fibrosis (CF) is the most common inherited dis- | ||
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Summary: . The develop- ment of AAV as a gene therapy vehicle for treating CF has several unique advantages based on its... by proteasome or ubiq- uitin ligase inhibitors. Tripeptide proteasome inhibitors increased persistent rAAV gene... infection. In vivo application of proteasome inhibitor in ... |
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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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| 45 | 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... that does not rely on marker gene expression, selection, or cell division, and therefore it can identify rAAV... -stranded DNA genome. Recombinant AAV (rAAV) is ... |
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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 | Loss of ATM Function Enhances Recombinant Adeno-Associated Virus Transduction and Integration through Pathways Similar to UV Irradiation | ||
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Summary: deficiency, and cancer. In this report we demonstrate that recombinant adeno-associated virus (rAAV) gene... understanding of genes involved in rAAV transduction. © 2000 Academic Press INTRODUCTION DNA damage manifested... features have made rAAV the vector of choice for many applica- ... |
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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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| 47 | JOURNAL OF VIROLOGY, 0022-538X/99/$04.00 0 | ||
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Summary: -associated virus (rAAV) vectors for gene therapy of inherited disorders have demon- strated considerable potential... I significantly inhibited AAV-2 internalization and gene delivery, but not viral binding... . Recombinant adeno-associated virus (rAAV) has gained in- creasing popularity ... |
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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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| 48 | HUMAN GENE THERAPY 18:367378 (April 2007) Mary Ann Liebert, Inc. | ||
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Summary: in genetic engineer- ing permit the customized design of AAV vectors for improved gene delivery, often based... -associated virus (AAV) is a promising vehicle for gene therapy, which will rely on the generation of high... SUMMARY Adeno-associated virus (AAV) is ... |
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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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| 49 | HUMAN GENE THERAPY 16:781791 (July 2005) Mary Ann Liebert, Inc. | ||
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Summary: IN THE NERVOUS SYSTEM 787 #12;bone regeneration using an exogenously regulated, AAV-2-based gene expression... Recombinant adeno-associated virus 2 (rAAV2) has been extensively used as a gene delivery vector... INTRODUCTION RECOMBINANT ADENO-ASSOCIATED VIRUS (rAAV) ... |
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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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| 50 | 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 provides useful information for the future design and application of recombinant AAV. Gene transfer vectors... based on the single-stranded DNA parvovirus 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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| 51 | 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... )-Mediated, but Not rAAV5-Mediated, Gene Transfer in the Brain Carmen S. Peden,1,2,3 Corinna Burger,2... makes it attractive as a gene therapy vector; ... |
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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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| 52 | Directed evolution of adeno-associated virus yields enhanced gene delivery vectors | ||
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Summary: `designer' rAAV vectors. The entire cap gene was subjected to (1) mutagenesis and recombination using PCR-based... neutralization and deliver genes more efficiently than wild-type capsid in the presence of anti-AAV serum... evolution to generate `designer' gene ... |
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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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| 53 | 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 this virus is based on studies evaluating wild type AAV or recombinant AAV which was unknowingly contaminated... differs from wild type AAV. © 1997 Elsevier ... |
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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 | 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 | ||
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Summary: or duplications. We assessed transcriptional activity of each rAAV2-targeted gene using a publicly available web-based... 2004 Recombinant adeno-associated virus (rAAV) vector holds promise for gene therapy. Despite a low... of rAAV integration because integration ... |
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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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| 55 | 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... . Based on the morphologic and molecular findings that AAV-2 endocytosis required functional Rac1, we next... . Inhibitors of AAV-2 ... |
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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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| 56 | JOURNAL OF VIROLOGY, 0022-538X/01/$04.00 0 DOI: 10.1128/JVI.75.15.69696976.2001 | ||
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Summary: ). Nonetheless, these re- sults suggest that most of the rAAV genomes and vector-based gene expression were... and extrachromosomal rAAV genomes in mouse livers and their relative contribution to stable gene expression in vivo... uncertainties as to whether ... |
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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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| 57 | NATURE BIOTECHNOLOGY VOLUME 24 NUMBER 8 AUGUST 2006 949 AAV hits the genomic bull's-eye | ||
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Summary: (AAV) vec- tors, which have been shown to correct faulty genes at reasonable efficiency in cultured... cells. In this issue, Miller et al.1 extend this work by demonstrating hepatic gene correction by AAV... VII.These studies provide a foundation for the development of AAV vector systems ... |
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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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| 58 | Construction of diverse adeno-associated viral libraries for directed evolution of enhanced gene | ||
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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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| 59 | original article 524 www.moleculartherapy.org vol. 17 no. 3, 524537 mar. 2009 | ||
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Summary: Society of Gene Therapy Recombinant adeno-associated virus (rAAV) expresses no viral genes after... and transgene expression would be unaffected. To further study whether trans- gene or rAAV capsid... © The American Society of Gene Therapy Immune Response to ... |
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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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| 60 | original article 2114 www.moleculartherapy.org vol.15no.12,21142123dec.2007 | ||
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Summary: -fold higher levels of apical gene transfer seen with rAAV2/1-based vectors in ALI cultures of human... Society of Gene Therapy Biological Differences in rAAV Transduction of Airway Epithelia in Humans... reflect the effectiveness of recombinant adeno-associated virus ... |
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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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| 61 | original article The American Society of Gene & Cell Therapy Molecular Therapy vol. 18 no. 5, 873880 may 2010 873 | ||
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Summary: , Intracranial AAV-mediated Gene Therapy, or Both in the Mouse Model of MPS IIIB Coy D Heldermon1,2 , Kevin K... , combining CNS-directed, AAV-mediated gene therapy with BMT resulted in dramatic synergy and signifi- cant... of Gene & Cell Therapy Combination Therapy for MPS IIIB (P = ... |
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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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| 62 | JOURNAL OF VIROLOGY, 0022-538X/99/$04.00 0 | ||
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Summary: extensively ex- plored as a potential vector for gene therapy (15). The advan- tages of the use of AAV-based... of transferring the human coagulation factor IX (hF.IX) gene into mouse liver by recombinant AAV (rAAV) vectors... expression of therapeutic gene products ... |
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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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| 63 | Recombinant Adeno-associated Viral Vectors as Therapeutic Agents to Treat Neurological Disorders | ||
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Summary: , potential caveats for rAAV-based gene therapy in the CNS are also presented. Key Words: Parkinson disease... would be to utilize AAV-based gene therapy to affect those genes. To attempt to target dominant disease... to survey the rAAV-based preclinical research in ... |
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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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| 64 | 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: (AAV)-based gene transfer techniques has proven to be an extremely pow- erful tool for treating many... genetic diseases (1, 12). Due to the relatively small packaging size of this single-stranded virus, AAV-based... offered tremendous potential for ex- panding AAV gene therapy to larger ... |
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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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| 65 | Gene Therapy (1999) 6, 14271437 1999 Stockton Press All rights reserved 0969-7128/99 $12.00 | ||
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Summary: AAV (rAAV) has demonstrated consider- able promise as a gene therapy vector for certain organ systems... by these environmental stimuli. The activation of rAAV transduction, as detected with a EGFP reporter gene, was evaluated... pathway which might alter the expression of cellular ... |
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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 | Sites in the AAV5 capsid tolerant to deletions and tandem duplications Kaoru Hida a | ||
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Summary: s t r a c t Gene therapy vectors based on adeno-associated virus (AAV) have shown much promise... January 2010 Available online 25 January 2010 Keywords: Directed evolution AAV Viral gene delivery a b... in the AAV2 capsid gene by site directed ... |
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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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| 67 | 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 | ||
|
Summary: without the trans- gene cassettes being altered, generating the AAV-5-based trans-splicing proviral... of helper viruses, such as adenovirus or herpes- virus (1, 2). AAV-based vector systems have attracted great... attention in the field of human gene therapy for more than 10 years. ... |
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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 | MOLECULAR THERAPY Vol. 4, No. 6, December 2001 Copyright The American Society of Gene Therapy | ||
|
Summary: cells. To evaluate the safety of in vivo recombinant adeno-asso- ciated virus (rAAV) mediated gene... transmission, AAV vectors, gene therapy, hemophilia INTRODUCTION Reproductive toxicology in the setting of gene... early disease targets for AAV- mediated ... |
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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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| 69 | NATURE BIOTECHNOLOGY VOLUME 25 NUMBER 10 OCTOBER 2007 1111 and drugs that act directly on disease genes. | ||
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Summary: - nancy. rAAV vector fragments were not detected in surrounding normal liver tissue. Genes close to the rAAV... in the future. What are the implications of the study of Donsante et al. for current rAAV gene ther- apy efforts... genes. Interestingly, the network ... |
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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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| 70 | 64 NATURE MEDICINE VOLUME 5 NUMBER 1 JANUARY 1999 Hemophilia is an X-linked recessive bleeding disorder that | ||
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Summary: 1999 ARTICLES Fig. 3 Gene therapy in hemophilia B mice. Recombinant rAAV-MFG-hFIX or rAAV-MFG-TH was de... . 5 Gene therapy in hemophilia B dogs. Recombinant AAV-MFG-canine Factor IX was delivered to the liver... procedures. The studies here demonstrate 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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| 71 | Species-Specific Differences in Mouse and Human Airway Epithelial Biology of Recombinant | ||
|
Summary: as surrogate models for rAAV transduction and gene therapy for CF. Keywords: recombinant adeno-associated virus... recombinant genes (19). How- ever, rAAV serotypes 2 and 5 are the most extensively studied vectors for gene... epithelia reproduce biologic features 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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| 72 | Adeno-associated viral vectors and stereotaxic | ||
|
Summary: : Inverted Terminal Repeats (ITR) flanking gene of interest 2. AAV helper plasmid expressing rep and cap... types · ~4.7 kb wild type genome #12;AAV Life Cycle #12;Packaging Components of rAAV 1. Transfer vector... · Not ideal for dividing cells, DNA is lost through cell division · Small genome allowing ... |
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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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| 73 | A Novel Adeno-Associated Viral Variant for Efficient and Selective Intravitreal Transduction of Rat Muller Cells | ||
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Summary: (AAV) mediated ocular gene therapy holds tremendous promise for treating and potentially curing... 11-cis retinal [13]. Gene-replacement therapy employing an AAV vector (rAAV) bearing a functional... for AAV in retinal gene therapy, providing ... |
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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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| 74 | JOURNAL OF VIROLOGY, 0022-538X/98/$04.00 0 | ||
|
Summary: Adeno-associated viral (AAV) vectors have demonstrated great utility for long-term gene expression... integration as the predom- inant mechanism of rAAV persistence in vivo. Muscle-mediated gene transfer... confirmed previous successes in rAAV-mediated gene transfer ... |
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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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| 75 | Adeno-Associated Virus Gene Repair Corrects a Mouse Model of Hereditary Tyrosinemia In Vivo | ||
|
Summary: Grompe1,5 Adeno-associated virus (AAV) vectors are ideal for performing gene repair due to their ability... -mediated gene repair is feasible in vivo and can functionally correct an appropriate selection-based metabolic... protein-coding genes have open reading frames small enough to fit ... |
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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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| 76 | JOURNAL OF VIROLOGY, 0022-538X/99/$04.00 0 | ||
|
Summary: large gene inserts or large promoter-gene combinations carried by two or more independent rAAV vectors... protein (16). In studies crucial to the development of AAV as a gene therapy vector, methods were... developed for propagating re- combinant AAV type 2 ... |
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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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| 77 | MOLECULAR THERAPY Vol. 6, No. 3, September 2002 Copyright The American Society of Gene Therapy | ||
|
Summary: the basis for future efforts to develop a recombinant AAV (rAAV)-based gene therapy approach... , such as neurons, cardiac and skeletal muscle, and hepatocytes [310]. Gene transfer with rAAV has been shown... to be relatively safe and long-lasting [11]. We recently investigated the ... |
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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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| 78 | Unique Biologic Properties of Recombinant AAV1 Transduction in Polarized Human Airway Epithelia* | ||
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Summary: for a given tissue target has been an area of intense interest in gene therapy research (4, 5). AAV type 2... the lung and leads to progressive and life-threatening bacterial lung infection. rAAV2-mediated CFTR gene... inhibitor treatment may eventually be an effective adjunct method to enhance ... |
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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 | Proc. Natl. Acad. Sci. USA Vol. 94, pp. 1408314088, December 1997 | ||
|
Summary: of the complete absence of wild-type viral genes in the vector (24, 26). These safety features of rAAV render... on rAAV to be used for gene therapy of neurodegenerative disorders may be presently more clinically... relevant than first-generation E1 E3 Ad vectors. The data regarding a ... |
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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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| 80 | Heparin binding induces conformational changes in Adeno-associated virus Hazel C. Levy a | ||
|
Summary: and E322 in AAV2 form a ring of five pairs of alternating basic/acidic residues at the base of the b... infected with an ade- novirus vector that expressed only the AAV capsid gene and no rep gene. Thus... Lafayette, IN 47907, USA c Department of Molecular Genetics and Microbiology and ... |
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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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| 81 | RAPID COMMUNICATION Structural Analysis of Adeno-Associated Virus Transduction Circular Intermediates | ||
|
Summary: -associated virus (rAAV) has emerged as a very promising gene delivery vehicle in several organs including muscle... viruses, such as adenovirus or herpes virus, and the wild-type AAV viral gene products Cap and Rep (1... . Regions flanking the D sequence of the 3 ITR included 40 bases ... |
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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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| 82 | HUMAN GENE THERAPY 16:299306 (March 2005) Mary Ann Liebert, Inc. | ||
|
Summary: the relative level of transduction. First, we delivered an AAV vector expressing the human factor IX gene from... infusion of rAAV is effective for liver-directed gene therapy and that other parameters related to blood... for adeno-associated vector serotype 2 (AAV-2) vectors show ... |
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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 | doi:10.1182/blood-2010-08-302729 Prepublished online November 24, 2010; | ||
|
Summary: Howard Hughes Medical Institute, Philadelphia, PA Gene transfer using adeno-associated vi- rus (AAV... tumor formation. This study addresses these questions with high-dose liver-directed AAV-mediated gene... onco- gene expression. Thus, we did not find evidence that ... |
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|
Source: Bushman, Frederic - Department of Microbiology, University of Pennsylvania |
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|
Collection: Biology and Medicine |
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| 84 | 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 | ||
|
Summary: genome of approximately 4.7 kb. Recombinant vi- ruses based on AAV serotype 2 are promising gene therapy... Z-expressing AAV serotype 2-based vectors at doses ranging between 4.0 108 and 1.1 1013 vector genomes (vg... -based vectors. Construction and production 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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| 85 | Transposon-Based Mutagenesis Generates Diverse Adeno-Associated Viral Libraries with Novel Gene | ||
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Summary: 10 Transposon-Based Mutagenesis Generates Diverse Adeno-Associated Viral Libraries with Novel Gene... . Briefly, a selectable marker is randomly inserted throughout the AAV2 cap gene and the resulting... "bookmarked" AAV cap gene is cloned into an ... |
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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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| 86 | HUMAN GENE THERAPY 18:344354 (April 2007) Mary Ann Liebert, Inc. | ||
|
Summary: deficiency. INTRODUCTION VECTORS BASED ON ADENO-ASSOCIATED VIRUS (AAV), a sin- gle-stranded DNA parvovirus... ;LUNG GENE TRANSFER USING AAV6 VECTORS 345 airway expression from an AAV2 vector is consistent... that is exposed to the virus. Sialic acid is required for ... |
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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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| 87 | JOURNAL OF VIROLOGY, Apr. 2004, p. 41654175 Vol. 78, No. 8 0022-538X/04/$08.00 0 DOI: 10.1128/JVI.78.8.41654175.2004 | ||
|
Summary: GFP target gene and rAAV vectors. The generation of a single base mutation in eGFP that destroys fluorescence... according to the base location of truncation in the eGFP gene relative to the starting codon at 1. rAAV2... findings demonstrating the efficacy 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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| 88 | Systemic Mannitol-Induced Hyperosmolality Amplifies rAAV2-Mediated Striatal Transduction to a | ||
|
Summary: (rAAV2) have been investigated as highly effective vehicles for gene transfer to the central nervous... degree than local coadministration. Key Words: rAAV, hyperosmolality, mannitol, CNS, gene therapy, gene... transfer Recombinant adeno-associated viral vector serotype 2 ... |
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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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| 89 | Convection-Enhanced Delivery of AAV Vector in Parkinsonian Monkeys; In Vivo Detection of Gene Expression and Restoration | ||
|
Summary: Convection-Enhanced Delivery of AAV Vector in Parkinsonian Monkeys; In Vivo Detection of Gene... An emerging new technology based on genetically engineering viral vectors that can insert genes into the cells... (pHLP19, containing the AAV rep and cap genes), and the ... |
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|
Source: California at Davis, University of - Department of Neurology, Center for Neuroscience |
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|
Collection: Computer Technologies and Information Sciences ; Biology and Medicine |
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| 90 | REVIEW ARTICLE Adeno-Associated Virus Vectors and Hematology | ||
|
Summary: .12-14 Virtually all the AAV vectors developed to date were based on AAV type 2, which has... AAV vectors do not include the rep gene (which would significantly limit the packaging capacity... sites. (B) Map of a typical AAV vector, showing replacement of the viral ... |
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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 | Unexpected off-targeting effects of anti-huntingtin ribozymes and siRNA in vivo | ||
|
Summary: ; Rodriguez-Lebron et al., 2005; Wang et al., 2005). Because AAV-based gene therapies for various... the expression of a subset of genes in the striatum. This effect was independent of rAAV transduction... : rAAV; Striatum; Huntington's disease; Mouse; Gene transfer; ... |
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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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| 92 | Recombinant Adeno-associated Viral Vector-Mediated Glial Cell Line-Derived Neurotrophic Factor Gene Transfer Protects | ||
|
Summary: , 47). Thus, rAAV-MD-GDNF was injected near the substan- tia nigra and gene expression was examined... available gene transfer systems, rAAV- delivered GDNF should be considered as a promising strategy... expression patterns of CNS gene transfer by an adeno-associated virus ... |
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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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| 93 | Nerve Growth Factor Expressed in the Medial Septum Following in Vivo Gene Delivery Using a Recombinant Adeno-Associated Viral Vector | ||
|
Summary: of a control rAAV vector. 1999 Academic Press Key Words: gene therapy;Alzheimer's disease; growth factors... -mediated gene delivery using the fim- bria-fornix (FF) model of cholinergic neuronal degenera- tion. An rAAV... to an identical control rAAV transduction (see Fig. 1B). Similar ... |
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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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| 94 | original article The American Society of Gene & Cell Therapy Molecular Therapy 1 | ||
|
Summary: to the efficacy of GDNF gene transfer. Intrastriatal injection of rAAV-GDNF yields neuroprotection of THir... by cotransfec- tion of a plasmid containing the rAAV rep and cap genes as well as adenovi- rus helper functions... original article© The American Society of Gene & Cell ... |
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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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| 95 | Characterization of Intrastriatal Recombinant Adeno-Associated Virus-Mediated Gene Transfer of Human Tyrosine Hydroxylase | ||
|
Summary: -dihydroxyphenylalanine The concept of gene therapy in the CNS for the treatment of neurological disorders is based on the fact... ). The applicability and safety of rAAV vectors for use in human gene therapy stems from several features associated... genes are re- moved in the case of ... |
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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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| 96 | Systemic but Not Intraocular Epo Gene Transfer Protects the Retina from Light-and | ||
|
Summary: and Characterization of Epo Expression Following AAV-Mediated Gene Transfer in Albino Rats and rds and rd10 Mice We... -Mediated Epo Gene Transfer We administered AAV vectors to Albino Lewis rats and rds and rd10 mice, light... morphological rescue after AAV-mediated Epo ... |
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|
Source: Sandini, Giulio - Dipartimento di Informatica Sistemistica e Telematica, Università degli Studi di Genova |
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|
Collection: Engineering ; Computer Technologies and Information Sciences |
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| 97 | MOLECULAR THERAPY Vol. 5, No. 1, January 2002 Copyright The American Society of Gene Therapy | ||
|
Summary: circuits. Key words: central nervous system, adeno-associated virus (AAV), gene transfer, gene therapy... retrograde gene deliv- ery using AAV offers several advantages for therapeutic intervention in CNS disease... from infection by AAV-GFP alone. (B) Prior ... |
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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 | Progress in Direct Striatal Delivery of L-Dopa via Gene Therapy for Treatment of Parkinson's Disease Using Recombinant | ||
|
Summary: are not practical. More- 49rAAV-MEDIATED L-DOPA GENE THERAPY FOR PD #12;over, peripheral continuous administration... -Dopa Delivery Continuous delivery of L-dopa directly to the stria- tum via rAAV gene therapy might... .e., the gene therapy procedure and whether the protein functions ... |
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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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| 99 | MOLECULAR THERAPY Vol. 4, No. 4, October 2001 Copyright The American Society of Gene Therapy | ||
|
Summary: for delivering genes larger than 5 kb. Based on the fact that independent AAV genomes can form large, circular... reconstitutes gene expression from two independent rAAV vectors, each encoding unique, nonoverlapping halves... -associated virus, rAAV, homologous ... |
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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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| 100 | Free DNA Ends Are Essential for Concatemerization of Synthetic Double-Stranded Adeno-Associated | ||
|
Summary: an important role in rAAV vector genome concatemerization. Key Words: adeno-associated virus, gene therapy... 3], modification of capsid proteins for retargeting [4], development of rAAV vectors based on sero- types other... expression. All of these developments have the potential to expand the utility 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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