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Sample search results for: aav vectors therapeutic

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1 Current Gene Therapy, 2003, 3, 281-304 281 1566-5232/03 $41.00+.00 2003 Bentham Science Publishers Ltd.
 

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 ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
2 Recombinant AAV Viral Vectors Pseudotyped with Viral Capsids from Serotypes 1, 2, and 5 Display
 

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 ...

  

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

 

Collection: Biology and Medicine

 
3 original article The American Society of Gene & Cell Therapy Molecular Therapy 1
 

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... neurodegenerative diseases or delivering therapeutic molecules. ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
4 Intracellular trafficking of adeno-associated viral and JF Engelhardt1,2,3
 

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 ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
5 SHORT COMMUNICATION Comparative biology of rAAV transduction in ferret,
 

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 ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
6 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
 

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 ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
7 Helper Virus-Free, Optically Controllable, and Two-Plasmid-Based Production of Adeno-associated
 

Summary:  therapeutically interesting targets, e.g., hepatocytes [3], and second, AAV-2 vector-mediated gene transfer might... strategy for producing high-titer adeno-associated virus (AAV) vectors derived from six different AAV... , and based on transfection of only two ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
8 HUMAN GENE THERAPY 15:405413 (April 2004) Mary Ann Liebert, Inc.
 

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 ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
9 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
 

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 ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
10 Robust Systemic Transduction with AAV9 Vectors in Mice: Efficient Global Cardiac Gene Transfer Superior
 

Summary:  Robust Systemic Transduction with AAV9 Vectors in Mice: Efficient Global Cardiac Gene Transfer... vector that overcomes many of the limitations of rAAV2 and transduces various tissues efficiently... and globally through systemic vector administration. AAV9 is a ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
11 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
 

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 ...

  

Source: Baker, Timothy S. - Department of Chemistry and Biochemistry, University of California at San Diego

 

Collection: Biology and Medicine

 
12 GENE THERAPY Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors
 

Summary:  therapeutic protein levels. Our findings that the AAV-6 vector expressed more strongly and with faster... ) vectors for the liver-specific expres- sion of human blood coagulation factor IX (hFIX). Therefore, an AAV... , the resultant vector particles expressed high ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
13 original article 2088 www.moleculartherapy.org vol. 17 no. 12, 20882095 dec. 2009
 

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, ...

  

Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley

 

Collection: Materials Science ; Chemistry

 
14 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
 

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

  

Source: Baker, Timothy S. - Department of Chemistry and Biochemistry, University of California at San Diego

 

Collection: Biology and Medicine

 
15 JOURNAL OF VIROLOGY, 0022-538X/99/$04.00 0
 

Summary:  -associated virus vectors (rAAV) have been developed by substituting the viral rep and cap genes with a therapeutic... . As a specific application of this finding, we generated adenovirus­adeno-associated virus (AAV) hybrid vectors... , first-generation Ad vectors ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
16 original article The American Society of Gene Therapy Molecular Therapy vol. 16 no. 10, 17031709 oct. 2008 1703
 

Summary:  ­1709 oct. 2008 1703 Adeno-associated virus (AAV) vectors are extremely effective gene-delivery vehicles... for a broad range of applications. However, the therapeutic efficacy of these and other vectors is currently... of gene-delivery properties, existing AAV serotypes transduce some ...

  

Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley

 

Collection: Materials Science ; Chemistry

 
17 Expert Review Designer Gene Delivery Vectors: Molecular Engineering and Evolution
 

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... . Molecular engineering and directed evolution of ...

  

Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley

 

Collection: Materials Science ; Chemistry

 
18 JOURNAL OF VIROLOGY, 0022-538X/00/$04.00 0
 

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 ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
19 Investor and Media Contact: Stacie D. Byars
 

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 ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
20 JOURNAL OF VIROLOGY, 0022-538X/01/$04.00 0 DOI: 10.1128/JVI.75.16.76627671.2001
 

Summary:  with this vector have also demonstrated great promise. However, to achieve therapeutic benefit in patients, large... by these two vectors in undifferentiated myoblasts, pseudotyped rAAV- 2cap5 demonstrated dramatically enhanced... the nonviral and viral vectors used in muscle gene ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine


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21 JOURNAL OF VIROLOGY, June 2008, p. 58875911 Vol. 82, No. 12 0022-538X/08/$08.00 0 doi:10.1128/JVI.00254-08
 

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 ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
22 Current Gene Therapy, 2001, 1, 137-147 137 1566-5232/00 $20.00+.00 2001 Bentham Science Publishers Ltd.
 

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... . transduction) is needed before AAV-mediated gene transfer becomes ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
23 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
 

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 ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
24 Capsid modifications overcome low heterogeneous expression of heparan sulfate proteoglycan that limits AAV2-mediated gene transfer and therapeutic
 

Summary:  expression. Here we sought to determine the therapeutic potential of a capsid-modified AAV vector for gene... gene transfer and AAV2HSVtk/GCV therapeutic efficacy. We thought it likely that vectors modified... that limits AAV2-mediated gene ...

  

Source: Hemminki, Akseli - Department of Biosciences, University of Helsinki

 

Collection: Biology and Medicine

 
25 NATURE BIOTECHNOLOGY VOL 18 MAY 2000 http://biotech.nature.com 527 Recombinant adeno-associated virus (rAAV) vectors based on AAV
 

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 ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
26 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
 

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 ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
27 Enhanced Preparation of Adeno-Associated Viral Vectors by Using High Hydrostatic Pressure to
 

Summary:  based on adeno-asso- ciated virus (AAV) have significant therapeutic potential, but much room... 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 ...

  

Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley

 

Collection: Materials Science ; Chemistry

 
28 [23] Adeno-Associated Virus Vectors for Short Hairpin RNA Expression
 

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 ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
29 JOURNAL OF VIROLOGY, 0022-538X/99/$04.00 0
 

Summary:  ). rAAV vectors have been developed by substituting a therapeutic minigene for all virus open reading... understood. To investigate these mechanisms, we have utilized a re- combinant AAV (rAAV) shuttle vector... increase the utility of rAAV ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
30 Directed evolution of adeno-associated virus to an infectious respiratory virus
 

Summary:  the human therapeutic potential of a customized AAV vector. CF is a complex and lethal genetic disease... efficient AAV vector for pulmonary gene delivery. Directed evolution platform in organotypic polarized, well... therapeutic applications of ...

  

Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley

 

Collection: Materials Science ; Chemistry

 
31 ORIGINAL ARTICLE Surface immobilization of hexa-histidine-tagged
 

Summary:  , Berkeley, CA, USA Adeno-associated viral (AAV) vectors, which are undergoing broad exploration in clinical... the potential of AAV in a variety of therapeutic applications by reducing spread outside a target region, which... . This immobilized AAV vector system ...

  

Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley

 

Collection: Materials Science ; Chemistry

 
32 JOURNAL OF VIROLOGY, 0022-538X/00/$04.00 0
 

Summary:  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. Recombinant adeno-associated virus vectors (rAAV... , the number of single-stranded rAAV vector ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
33 JOURNAL OF VIROLOGY, 0022-538X/00/$04.00 0
 

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. ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
34 Copyright Lippincott Williams & Wilkins. Unauthorized reproduction of this article is prohibited. Gene therapy for hemophilia
 

Summary:  higher expression than an AAV8 vector with a single-stranded DNA. This allows therapeutic levels of human... -associated virus vector (AAV vector), adenoviral vector, gene therapy, hemophilia, retroviral vector Curr Opin... . ...

  

Source: Ponder, Katherine P. - Departments of Medicine & Biochemistry and Molecular Biophysics, Washington University in St. Louis

 

Collection: Biology and Medicine

 
35 original article 2096 www.moleculartherapy.org vol. 17 no. 12, 20962102 dec. 2009
 

Summary:  -term transgene expression following a single treatment make the virus a very efficient gene delivery vector.4 AAV... genome are replaced by a promoter and therapeutic transgene cassette flanked by the AAV inverted ter... Society of Gene & Cell Therapy Intravitreal Retinal Transduction With ...

  

Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley

 

Collection: Materials Science ; Chemistry

 
36 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
 

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 ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
37 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 ...

  

Source: Baltimore, David - Division of Biology, California Institute of Technology

 

Collection: Biotechnology ; Biology and Medicine

 
38 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
 

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 ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
39 JOURNAL OF VIROLOGY, 0022-538X/99/$04.00 0
 

Summary:  expression of therapeutic gene products from the liver by rAAV vectors has not been fully understood... January 1999/Accepted 30 March 1999 Recombinant adeno-associated virus (rAAV) vectors allow for sustained... vector expressing human coagulation factor F.IX ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
40 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 ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine


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41 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
 

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, ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
42 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 ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
43 Proc. Natl. Acad. Sci. USA Vol. 94, pp. 1408314088, December 1997
 

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 ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
44 64 NATURE MEDICINE VOLUME 5 NUMBER 1 JANUARY 1999 Hemophilia is an X-linked recessive bleeding disorder that
 

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 ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
45 original article 524 www.moleculartherapy.org vol. 17 no. 3, 524537 mar. 2009
 

Summary:  , and liver, may require repeated administra- tion of vector to achieve the desired therapeutic level in vivo... . E-mail: rmandel@ufl.edu Striatal Readministration of rAAV Vectors Reveals an Immune Response Against... loss, we readministered rAAV2/2 and then measured striatal ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
46 HUMAN GENE THERAPY 16:781791 (July 2005) Mary Ann Liebert, Inc.
 

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 ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
47 original article The American Society of Gene & Cell Therapy Molecular Therapy vol. 18 no. 5, 873880 may 2010 873
 

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... as a surrogate for therapeutic response.29,30 0 * ** M PS IIIB no txM PS IIIB AAVM PS ...

  

Source: Orrock, John - Department of Zoology, University of Wisconsin at Madison

 

Collection: Environmental Sciences and Ecology ; Biology and Medicine

 
48 ELSEVIER Virus Research 48 (1997) 41-56 Structural and functional heterogeneity of integrated
 

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 ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
49 A Novel Adeno-Associated Viral Variant for Efficient and Selective Intravitreal Transduction of Rat Muller Cells
 

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... changes. This isolated variant represents a new therapeutic ...

  

Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley

 

Collection: Materials Science ; Chemistry

 
50 Directed evolution of adeno-associated virus yields enhanced gene delivery vectors
 

Summary:  and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors... 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. ...

  

Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley

 

Collection: Materials Science ; Chemistry

 
51 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:  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 ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
52 Recombinant Adeno-associated Viral Vectors as Therapeutic Agents to Treat Neurological Disorders
 

Summary:  Recombinant Adeno-associated Viral Vectors as Therapeutic Agents to Treat Neurological Disorders... candidate vector for treating neurological disorders. Indeed, rAAV is currently being used in five early... . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 466 Progress in the ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
53 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
 

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). ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
54 Construction of diverse adeno-associated viral libraries for directed evolution of enhanced gene
 

Summary:  gene delivery vectors1,2. Many viruses have been used to deliver therapeutic genetic material... 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 ...

  

Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley

 

Collection: Materials Science ; Chemistry

 
55 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:  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... vectors that deliver genes of interest into a variety of ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
56 Species-Specific Differences in Mouse and Human Airway Epithelial Biology of Recombinant
 

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... when drawing conclusions about the validity of this model for predicting the ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
57 HUMAN GENE THERAPY 16:299306 (March 2005) Mary Ann Liebert, Inc.
 

Summary:  ) vectors have therapeutic potential for the treatment of several types of liver diseases including hepato... 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 ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
58 MOLECULAR THERAPY Vol. 6, No. 3, September 2002 Copyright The American Society of Gene Therapy
 

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 ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
59 JOURNAL OF VIROLOGY, 0022-538X/98/$04.00 0
 

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 ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
60 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 ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine


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61 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) ...

  

Source: Gray, Jeffrey J. - Department of Biomolecular and Chemical Engineering, Johns Hopkins University; Ostermeier, Marc - Department of Biomolecular and Chemical Engineering, Johns Hopkins University

 

Collection: Biology and Medicine ; Biotechnology ; Chemistry

 
62 Adeno-associated viral vectors and stereotaxic
 

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 ...

  

Source: Chapman, Michael S. - Department of Biochemistry and Molecular Biology, Oregon Health and Science University

 

Collection: Biology and Medicine

 
63 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 (rAAV) by ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
64 HUMAN GENE THERAPY 18:344354 (April 2007) Mary Ann Liebert, Inc.
 

Summary:  -CFTR vectors, other lung diseases such as 1-antitrypsin can be a therapeutic goal for AAV6 gene therapy... -associated virus (AAV) vectors in various somatic tissues has been shown to depend heavily on the AAV type from... which the vector capsid ...

  

Source: Miller, Dusty - Division of Human Biology, Fred Hutchinson Cancer Research Center

 

Collection: Biology and Medicine

 
65 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... ) vectors have been considered safe and are widely used for delivering therapeutic genes into experimental... animals and human subjects (14). rAAV2 vectors have the ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
66 A Two-hybrid Screen Identifies Cathepsins B and L as Uncoating Factors for Adeno-associated Virus 2
 

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 ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
67 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... (CED) method. We showed that adeno-associated viral vectors (AAV) can be distrib- uted in the brain... of the brain (contralateral side) (1, 2, 12, 13). CED of either AAV with reporter (LacZ) ...

  

Source: California at Davis, University of - Department of Neurology, Center for Neuroscience

 

Collection: Computer Technologies and Information Sciences ; Biology and Medicine

 
68 REVIEW ARTICLE Adeno-Associated Virus Vectors and Hematology
 

Summary:  - associated virus (AAV) vectors were among the first alterna- tives to be investigated. Although a substantial... amount of work with AAV vectors has occurred over the last several years, the bulk of evidence suggests... that now appear especially promising for AAV ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
69 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... of mutations (insertions, deletions, substitutions) by vector-mediated homologous recombi- nation.16,17 AAV... for in vivo selection of corrected hepatocytes following gene repair. In this system, when ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
70 Unique Biologic Properties of Recombinant AAV1 Transduction in Polarized Human Airway Epithelia*
 

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 ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
71 doi:10.1182/blood-2010-08-302729 Prepublished online November 24, 2010;
 

Summary:  . In addition, many therapeutic strategies that use AAV vectors in the liver are in preclinical development. Our... . Assessing the potential for AAV vector genotoxicity in a murine model http... THERAPY Assessing the potential forAAV vector ...

  

Source: Bushman, Frederic - Department of Microbiology, University of Pennsylvania

 

Collection: Biology and Medicine

 
72 Recombinant Adeno-associated Viral Vector-Mediated Glial Cell Line-Derived Neurotrophic Factor Gene Transfer Protects
 

Summary:  was undertaken to deter- mine if GDNF delivered via a recombinant adeno- associated viral vector (rAAV) could... ) or recombi- nant adeno-associated virus (rAAV) vectors (32). Recent reports using rAd delivered GDNF after... are protected from degeneration. METHODS Recombinant AAV ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
73 JOURNAL OF VIROLOGY, Oct. 2006, p. 98319836 Vol. 80, No. 19 0022-538X/06/$08.00 0 doi:10.1128/JVI.00878-06
 

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 ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
74 original article The American Society of Gene & Cell Therapy Molecular Therapy 1
 

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 ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
75 Unexpected off-targeting effects of anti-huntingtin ribozymes and siRNA in vivo
 

Summary:  therapeutic promise in vivo. Previously, we have reported that a specific, adeno- associated viral vector (rAAV... -Htt short-hairpin RNA molecules delivered via recombinant adeno-associated viral vectors (rAAV... access their target in a biologically relevant RNA transcript. Generation ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
76 Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host
 

Summary:  that rAAV-2 vectors can transduce human hepatocytes in vivo to result in therapeutically relevant levels... infusion of a recombinant adeno-associated viral vector (rAAV) expressing canine Factor IX (F.IX) resulted... of hemophilia B by a single infusion of ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
77 Systemic Mannitol-Induced Hyperosmolality Amplifies rAAV2-Mediated Striatal Transduction to a
 

Summary:  system (CNS). Transduction with rAAV2 vectors results in long-term transgene expression in CNS neurons... transfer Recombinant adeno-associated viral vector serotype 2 (rAAV2) has proven to be an attractive gene... with 2 Al of vector in a 6.7% mannitol solution and the right striatum ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
78 Characterization of the Relationship of AAV Capsid Domain Swapping to Liver
 

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 ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
79 Nerve Growth Factor Expressed in the Medial Septum Following in Vivo Gene Delivery Using a Recombinant Adeno-Associated Viral Vector
 

Summary:  to the medial septum via a recombinant adeno-associated viral vector (rAAV) encoding the cDNA for human NGF... of a control rAAV vector. 1999 Academic Press Key Words: gene therapy;Alzheimer's disease; growth factors... Age/BamHI frag- ment containing human NGF (33). Recombinant AAV ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
80 HUMAN GENE THERAPY 12:563573 (March 20, 2001) Mary Ann Liebert, Inc.
 

Summary:  of therapeutic levels of hFX from an AAV vector with the CMV-b-actin promoter in mice The above-described data... ,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 ...

  

Source: Ponder, Katherine P. - Departments of Medicine & Biochemistry and Molecular Biophysics, Washington University in St. Louis

 

Collection: Biology and Medicine


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81 Free DNA Ends Are Essential for Concatemerization of Synthetic Double-Stranded Adeno-Associated
 

Summary:  , we analyzed the role of inverted terminal repeat (ITR) sequences of ds circular or linear rAAV vector... an important role in rAAV vector genome concatemerization. Key Words: adeno-associated virus, gene therapy... parvovirus with a single-stranded (ss) DNA genome of 4.7 kb. The recombinant ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
82 Characterization of Intrastriatal Recombinant Adeno-Associated Virus-Mediated Gene Transfer of Human Tyrosine Hydroxylase
 

Summary:  independent in vivo L-DOPA production (mean 4­7 ng/ml). Rats that received the hTH rAAV vector alone produced... -DOPA was observed in animals that received mixed hTH and hGTPCHI rAAV vectors, there was no reduction of apomorphine... -induced rotational behavior 3 weeks after intra- striatal ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
83 Zerit (Bristol-Myers) Emtriva (HIV/Gilead)
 

Summary:  TM (Achillion) TriapineTM (NanoTherapeutics) PHY906 (Phytoceutica) CERE-110 (Ceregene) CERE-120 (Ceregene) NLX... -P101 (Neurologix) AAV hAADC2G ) The Yale Pharmaceutical Pipeline Preclinical Phase I Phase II Phase III... NDA/BLA Market AAV-hAADC2Genzyme) FESTINAVIR® (Oncolys/Bristol Myers Squibb) Radezolid (RX-1741) (Rib

  

Source: Wang, Xiao-Jing - Kavli Institute for Neuroscience & Department of Neurobiology, Yale University

 

Collection: Biology and Medicine

 
84 Progress in Direct Striatal Delivery of L-Dopa via Gene Therapy for Treatment of Parkinson's Disease Using Recombinant
 

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. ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
85 HUMAN GENE THERAPY 14:871881 (June 10, 2003) Mary Ann Liebert, Inc.
 

Summary:  DNA Vector Ends in Normal and DNA-PKcs­Deficient SCID Mouse Hepatocytes Transduced with rAAV Vectors... of transformation of single-stranded (ss) recombinant adeno-associated virus (rAAV) vector genomes into a variety... of stable double-stranded (ds) forms is key to a complete ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
86 MOLECULAR THERAPY Vol. 5, No. 1, January 2002 Copyright The American Society of Gene Therapy
 

Summary:  retrograde gene deliv- ery using AAV offers several advantages for therapeutic intervention in CNS disease... generated a 900-bp product (arrowhead) in a control reaction using a pAAV-GFP vector plasmid. #12;MOLECULAR... to efficiently target areas within the nervous system by a safe, long-term expressing ...

  

Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley

 

Collection: Materials Science ; Chemistry

 
87 MOLECULAR THERAPY Vol. 4, No. 4, October 2001 Copyright The American Society of Gene Therapy
 

Summary:  adeno-associated (rAAV) viral vectors hold great therapeutic potential for human diseases. However... Expanding AAV Packaging Capacity with Trans-splicing or Overlapping Vectors: A Quantitative Comparison... distinct approaches to overcome packaging restraints with ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
88 Oncoprotein Akt/PKB induces trophic effects in murine models of Parkinson's disease
 

Summary:  of the vector injection. (Right) Contraversive rotations are plotted as negative net rotations. AAV GFP... . An alternative approach is to directly activate, by viral vector transduction, intracellular signaling path- ways... pathways by vector transfer is a feasible approach to neuroprotection and ...

  

Source: Burke, Robert E - Departments of Neurology and Pathology, Columbia University

 

Collection: Biology and Medicine

 
89 LONG-TERM RESTORATION OF STRIATAL l-AROMATIC AMINO ACID DECARBOXYLASE ACTIVITY USING RECOMBINANT
 

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 ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
90 Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy
 

Summary:  therapeutic transgenes exceed the packaging limitation of present AAV vectors. Adeno-associated virus (AAV... ) vectors for muscle-directed thera- pies, a renewed interest in investigating the mechanisms of rAAV... of this vector in treating ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
91 Current Gene Therapy, 2007, 7, 347-360 347 1566-5232/07 $50.00+.00 2007 Bentham Science Publishers Ltd.
 

Summary:  and adaptive immune responses on therapeutic gene expression mediated by adenoviral, AAV, and lentiviral... gene therapy vectors and the resulting loss of therapeutic gene expression are examined in the context... 's immune response to the viral vector is a logical culprit for the lack of ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
92 Systemic but Not Intraocular Epo Gene Transfer Protects the Retina from Light-and
 

Summary:  of recombinant adeno-associated viral vectors (AAV) encoding the Prph2 transgene results in morpho- logical... models of retinal degeneration we delivered AAV vectors encoding Epo to the retinas and compared... injected AAV2 vectors encoding either Epo or EGFP ...

  

Source: Sandini, Giulio - Dipartimento di Informatica Sistemistica e Telematica, Università degli Studi di Genova

 

Collection: Engineering ; Computer Technologies and Information Sciences

 
93 RNA interference improves motor and neuropathological abnormalities in a Huntington's
 

Summary:  (23). Recombinant AAV serotype 1 vectors were generated as described in ref. 23. AAV titers were... . 2. AAV.shHD2.1 delivers widespread RNAi expression to mouse stria- tum. (A) AAV.shHD2.1 viral vector... exists for HD. RNA interference (RNAi) has emerged as a ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
94 original article The American Society of Gene Therapy Molecular Therapy vol. 17 no. 2, 285293 feb. 2009 285
 

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 ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
95 Dual Therapeutic Utility of Proteasome Modulating Agents for Pharmaco-Gene Therapy of the
 

Summary:  from the apical membrane [23,24]. Interestingly, studies comparing rAAV2 directly to rAAV2/5 vectors... circumvented, rAAV2 may emerge as a preferential vector for gene therapy of the CF lung. Additional limitations... to rAAV-mediated gene deliv- ery of CFTR include ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
96 The idea of gene-based therapeutics has been around for some time, but it only received serious attention
 

Summary:  , the therapeutic DNA either integrates into host chromosomal DNA or exists as an episomal vector. Gene addition... for therapeutic levels of transgene expression. The distribution of the vector after administration is influenced... with therapeutic sequences and they can be pseudotyped ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
97 NATURE MEDICINE VOLUME 6 NUMBER 5 MAY 2000 595 NEW TECHNOLOGY
 

Summary:  -associated virus (rAAV) has attracted tremendous interest as a promising vector for gene therapy. However... , a principal limitation of rAAV vectors is their re- stricted, 5-kilobase (kb) packaging capacity. This has hin... with this vector system. One well-known example is ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
98 HUMAN GENE THERAPY 16:11161123 (September 2005) Mary Ann Liebert, Inc.
 

Summary:  . Furthermore, because recombinant adeno-associated virus (rAAV) vectors have packing limitations with respect... transgene cassette. In the present study, we investi- gated whether rAAV vectors could effectively rescue... epithelia were infected from the apical mem- brane with rAAV2 or ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
99 Adenovirus Transduction is Required for the Correction of Diabetes Using Pdx-1 or
 

Summary:  - associated virus (AAV) vectors to deliver these pancreatic factors to the liver, as AAV8 vectors have been... in the livers of mice in vivo, we constructed AAV serotype 8 vectors expressing Pdx-1 and Ngn3 under... , such as the cytomegalovirus early promoter.19,20 We ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
100 Suppression of Insult-Induced Neurogenesis in Adult Rat Brain by Brain-Derived Neurotrophic Factor
 

Summary:  (data not shown). The rAAV vectors used in the present study contain an ires sequence which allows... . Transduction of the hippocampus with the BDNF- rAAV vector did not change the total number of new cells labeled... SE-221 84 Lund, Sweden; and Department of Neuroscience, Department of Pharmacology and ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine


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