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Sample search results for: aav gene transfer

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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
 

Summary:  that varying genome length and AAV capsids may allow for improved expression and/or gene transfer to specific... done and gene transfer was quantified. We showed that AAV2/3 does not transduce cells in the murine... capsid binding. Though it is possible that the lack ...

  

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

 

Collection: Biology and Medicine

 
2 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... : Inverted Terminal Repeats (ITR) flanking gene of interest 2. AAV helper plasmid expressing rep and cap... · Not ideal for dividing cells, DNA is lost through cell division · ...

  

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

 

Collection: Biology and Medicine

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

Summary:  Abstract: Gene transfer vectors based on the human adeno-associated virus serotype 2 (AAV-2) have been... . Second, gene transfer might be hampered by neutralizing anti-AAV-2 antibodies, which are highly prevalent... - clinical testing of novel ...

  

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

 

Collection: Biology and Medicine

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

Summary:  and promising tools for human gene transfer, owing to their high safety (the vectors are devoid of wild... of a Gal4p-dependent lacZ reporter gene in yeast transformed with the AAV8 bait and several clones... gene activation with the AAV5-based capsid bait was not ...

  

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

 

Collection: Biology and Medicine

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

Summary:  @ufl.edu. Available online 1 July 2004 Recombinant adeno-associated virus 2 (rAAV2) has been shown to deliver genes... in the nervous system. Key Words: AAV, capsid, serotype, gene delivery, nervous system, brain, spinal cord... been used successfully to transfer ...

  

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

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

Summary:  )-mediated, but not rAAV5-mediated, gene transfer in the brain. J Virol 78: 6344­6359. 28. Burger, C, Nash, K... , TE and Zweig, RM (2006). Efficient neuronal gene transfer with AAV8 leads to neurotoxic levels of tau... of neurturin by AAV2 ...

  

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

 

Collection: Biology and Medicine

 
7 SHORT COMMUNICATION Comparative biology of rAAV transduction in ferret,
 

Summary:  and transmembrane mucins inhibit gene transfer with AAV4 more efficiently than AAV5. J Biol Chem 2002; 277: 23709... - associated virus (rAAV)-mediated gene therapies to the lung for cystic fibrosis (CF). As new ferret and pig... appropriate animal model to test ...

  

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

 

Collection: Biology and Medicine

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

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 ...

  

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

 

Collection: Biology and Medicine

 
9 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... as a human gene ...

  

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

 

Collection: Biology and Medicine

 
10 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:  augmented rAAV- 5-mediated gene transfer. Increased transgene expression was independent of viral genome... proteasome inhibitors, such as LLnL or Z-LLL, can substantially augment rAAV-2-mediated gene transfer... (12). Hence, we examined whether the proteasome ...

  

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

 

Collection: Biology and Medicine

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

Summary:  for gene transfer. J Biol Chem 2001; 21: 21. Intracellular trafficking of AAV vectors W Ding et al 879 Gene... Adeno-associated virus (AAV) has attracted considerable interest as a gene therapy vector over the past... viral genes are the inverted ...

  

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

 

Collection: Biology and Medicine

 
12 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... helper plasmids. The latter were engineered to carry AAV serotype rep and cap genes together... with an AAV-2 vector plasmid and a helper construct ...

  

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

 

Collection: Biology and Medicine

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

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... transfer in animals, ...

  

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

 

Collection: Biology and Medicine

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

Summary:  of these. Viral-mediated gene delivery is currently the most efficient gene transfer method... , 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 ...

  

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

 

Collection: Biology and Medicine

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

Summary:  muscle compared to AAV2/2 (83). In addition, AAV2/5 mediated efficient gene transfer to murine cerebellar... that pseudotyped AAV2/7 exhibited gene transfer to skeletal muscle with efficiency comparable to that of AAV1... ). ...

  

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

 

Collection: Materials Science ; Chemistry

 
16 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:  interactions among parvoviruses. Recombinant adeno-associated viruses (AAV) hold promise for gene transfer... by gene transfer (27). AAV2 has also been investigated in vitro and in vivo for use in gene transfer... viruses for ...

  

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

 

Collection: Biology and Medicine

 
17 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:  on AAV2 are being tested in a clinical trial of liver-directed gene transfer for hemophilia B, but recent... before maximal gene expression. Vector genomes packaged inside AAV6 or AAV8 capsids do not persist... DNA) genome. Vectors derived from ...

  

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

 

Collection: Biology and Medicine

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

Summary:  ). AAV8, 9, Rh10, Rh43 vector gene transfer in the rat brain: effects of serotype, promoter... creation of AAV vec- tors with valuable new gene delivery properties. We have engineered novel AAV variants... evolution can successfully cre- ate AAV ...

  

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

 

Collection: Materials Science ; Chemistry

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

Summary:  cardiac gene transfer superior to that of AAV8. Mol Ther 14: 45­53. 13. Wu, Z, Miller, E, Agbandje... ­1709 oct. 2008 1703 Adeno-associated virus (AAV) vectors are extremely effective gene-delivery vehicles... potentially yield unlimited numbers of new AAV variants ...

  

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

 

Collection: Materials Science ; Chemistry

 
20 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:  of AAV are in development for use in human gene therapy applications due to their ability to transduce... of Molecular Genetics and Microbiology,4 and Powell Gene Therapy Center,5 College of Medicine, University... , Bethesda, Maryland3 Received 14 July 2004/Accepted 17 November 2004 Adeno-associated virus ...

  

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

 

Collection: Biology and Medicine


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

Summary:  for efficient gene transfer into target cells, and (iii) that the presence of AAV ITRs in these genomic... derivatives, which can be used as efficient gene transfer vehicles, we designed an adenovirus-AAV hybrid... gene cassette inserted into the ...

  

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

 

Collection: Biology and Medicine

 
22 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... (rAAV2) is a promising vector, in vivo gene transfer with rAAV2 is suboptimal in many instances... gene therapy. It should be ...

  

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

 

Collection: Biology and Medicine

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

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 ...

  

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

 

Collection: Biology and Medicine

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

Summary:  - or liver-directed gene transfer with hFIX-expressing AAV-2 vectors so far show that the recombinant... -directed gene transfer strate- gies that rely on the readministration of AAV serotype vectors. Discussion... that vectors based on AAV ...

  

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

 

Collection: Biology and Medicine

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

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 ...

  

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

 

Collection: Biology and Medicine

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

Summary:  the nonviral and viral vectors used in muscle gene transfer, recombinant adeno-associated virus type 2 (rAAV-2... has been examined in efforts to identify additional means of improving rAAV-2-mediated gene transfer... of gene ...

  

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

 

Collection: Biology and Medicine

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

Summary:  . transduction) is needed before AAV-mediated gene transfer becomes therapeutically useful. Although, AAV... AAV5 bound to airway epithelia and mediated gene transfer 50-fold more efficiently than AAV2 (Zabner... is essential ...

  

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

 

Collection: Biology and Medicine

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

Summary:  for gene transfer to the airway epithelia in vivo. Adeno-associated viruses (AAVs) show promise as gene... that is resistant to gene transfer with AAV (2). Differen- tiated epithelia were infected with recombinant AAV type... ...

  

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

 

Collection: Biology and Medicine

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

Summary:  that limits AAV2-mediated gene transfer and therapeutic efficacy in human ovarian carcinoma Wenfang Shi... -modified AAV vectors can mediate enhanced gene transfer to neoplasms characterized by low AAV receptor... of receptors in ...

  

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

 

Collection: Biology and Medicine

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

Summary:  as two powerful and compatible approaches to the molecular evolution of novel AAV vectors for human gene... capsid for efficient and safe gene transfer has been exemplified in many preclinical studies, as well... strategies rely on the direct physical modification of the AAV capsid ...

  

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

 

Collection: Biology and Medicine

 
31 original article 594 www.moleculartherapy.org vol. 18 no. 3, 594600 mar. 2010
 

Summary:  to the relative efficiency of gene transfer among the most newly available rAAV serotypes that might be used... advantages for rAAV1 over rAAV5 in the chimpanzee airway, both in terms of a superior level of gene transfer... ) but not ...

  

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

 

Collection: Biology and Medicine

 
32 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... , but the mechanism is unclear. This paper explores the balance between random integration and gene targeting with rAAV... stimulates the frequency of rAAV-mediated ...

  

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

 

Collection: Biotechnology ; Biology and Medicine

 
33 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:  . 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... transfer is derived from the type 2 serotype ...

  

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

 

Collection: Biology and Medicine

 
34 ORIGINAL ARTICLE Surface immobilization of hexa-histidine-tagged
 

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 ...

  

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

 

Collection: Materials Science ; Chemistry

 
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
 

Summary:  muscles, and brain. Thus, rAAV8 is a robust vector for gene transfer to the liver and provides a promising... , including hemo- philia B (22). Despite such recent advances, rAAV2-mediated hepatic gene transfer has still... research tool for delivering ...

  

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

 

Collection: Biology and Medicine

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

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 ...

  

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

 

Collection: Biology and Medicine

 
37 [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... ). rAAV has the specific ability to transduce quiescent cells, and its use for gene transfer provides... -stranded form from which AAV ...

  

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

 

Collection: Biology and Medicine

 
38 HUMAN GENE THERAPY 10:591 602 (March 1, 1999) Mary Ann Liebert, Inc.
 

Summary:  expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector. Brain Res. 713, 99... 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 ...

  

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

 

Collection: Biology and Medicine

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

Summary:  , com- plete rAAV expression cassette. The mechanism of the enhanced gene expression from two vec- tors... and incorporated into the 5 end of the cytoplasmic lacZ gene of pAAV-EF1-LacZ11. pAAV-Pless-nlsLacZ was produced... including intron 1 and the splice acceptor of exon 2 of the EF1 ...

  

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

 

Collection: Biology and Medicine

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

Summary:  of airway epithelia as well as improved gene transfer. Furthermore, the novel AAV virus mediates successful... ) but not AAV2 binds to the apical surfaces of airway epithelia and facilitates gene transfer. J Virol 74... gene ...

  

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

 

Collection: Materials Science ; Chemistry


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

Summary:  Ion Adeno-associated virus (AAV) has become the most promis- ing ocular gene delivery vehicle over the past... -term transgene expression following a single treatment make the virus a very efficient gene delivery vector.4 AAV... In recombinant vec- tors, genes encoding replication (rep) ...

  

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

 

Collection: Materials Science ; Chemistry

 
42 JOURNAL OF VIROLOGY, Oct. 2007, p. 1130411321 Vol. 81, No. 20 0022-538X/07/$08.00 0 doi:10.1128/JVI.01225-07
 

Summary:  of adeno-associ- ated virus (AAV) are processed by DNA repair machinery; however, how and what cellular... factors are involved in the process remain elusive. Here, we show that DNA-PKcs and Artemis open AAV... inverted terminal repeat (ITR) hairpin loops in a tissue-dependent manner. We investigated recombinant AAV

  

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

 

Collection: Biology and Medicine

 
43 Introduction Cystic fibrosis (CF) is the most common inherited dis-
 

Summary:  infection. In vivo application of proteasome inhibitor in mouse lung augmented rAAV gene transfer from unde... inhibitors also increased rAAV-2­mediated gene transfer to the liver tenfold, but they did not affect... ligase inhibitors increased the extent of ...

  

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

 

Collection: Biology and Medicine

 
44 Virus Research 104 (2004) 5159 Novel approaches to augment adeno-associated virus
 

Summary:  ; 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 gene therapy (Alexander et al., 1994; Kaplitt et al., 1994; Lai et... al., 2002; Xiao et al., 1996). Recombinant AAV ...

  

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

 

Collection: Biology and Medicine

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

Summary:  in the laboratory- and clinical- scale production of AAV gene delivery vectors. Moreover, HHP provides a tool... capsid shell that encloses a single-stranded 4.7 kb DNA genome. Gene delivery vectors based on AAV... of this important gene delivery vehicle. Prior to this study, the ...

  

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

 

Collection: Materials Science ; Chemistry

 
46 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:  effective at augmenting gene transfer to mouse lungs with rAAV than previously tested aldehyde proteasome... rAAV transduction in airway epithelia for gene therapy of cystic fibrosis. Adeno-associated virus... target has been an area of intense interest to gene ...

  

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

 

Collection: Biology and Medicine

 
47 Loss of ATM Function Enhances Recombinant Adeno-Associated Virus Transduction and Integration through Pathways Similar to UV Irradiation
 

Summary:  to augment gene transfer with rAAV vectors. MATERIALS AND METHODS Generation of recombinant AAV stocks... patterns of CNS gene transfer by an adeno-associated virus (AAV) vector. Brain Res. 713, 99­107. Miao, C. H... deficiency, and cancer. In ...

  

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

 

Collection: Biology and Medicine

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

Summary:  -stranded rAAV DNA. FIG. 2. Expression of K44A dynamin I mutant inhibits rAAV-2-mediated gene transfer in He... of rAAV-mediated gene transfer by the dynamin I mutant was not due to inhibition on viral at- tachment... -associated virus ...

  

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

 

Collection: Biology and Medicine

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

Summary:  and may be useful for improving liver gene transfer in vivo. Recombinant adeno-associated virus vectors (rAAV... concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nat. Genet. 16... -directed gene ...

  

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

 

Collection: Biology and Medicine

 
50 JOURNAL OF VIROLOGY, Oct. 2007, p. 1129011303 Vol. 81, No. 20 0022-538X/07/$08.00 0 doi:10.1128/JVI.00963-07
 

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 ...

  

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

 

Collection: Biology and Medicine

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

Summary:  , Hutchison S, et al. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe... -associated virus vector (AAV vector), adenoviral vector, gene therapy, hemophilia, retroviral vector Curr Opin... animals, a summary of progress in the use of gene ...

  

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

 

Collection: Biology and Medicine

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

Summary:  of adenovi- rus, AAV and lentivirus vectors for gene transfer of GDNF to the nigrostriatal system in the rat... coupled to a minimal bidirectional tet cassette for tight regulation of rAAV-mediated gene transfer... intrastriatal delivery of L-dopa using ...

  

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

 

Collection: Biology and Medicine

 
53 HUMAN GENE THERAPY 18:367378 (April 2007) Mary Ann Liebert, Inc.
 

Summary:  -associated virus (AAV) is a promising vehicle for gene therapy, which will rely on the generation of high... SUMMARY Adeno-associated virus (AAV) is promising a vehicle for gene therapy. However, numerous... 100 AAV serotypes, which exhibit a diverse array of gene ...

  

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

 

Collection: Materials Science ; Chemistry

 
54 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:  and provides useful information for the future design and application of recombinant AAV. Gene transfer vectors... to AAV gene transfer and relieved some of the concerns associ- ated with the abundance of neutralizing... by modifying the viral serotype, attempts to ...

  

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

 

Collection: Biology and Medicine

 
55 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:  )-Mediated, but Not rAAV5-Mediated, Gene Transfer in the Brain Carmen S. Peden,1,2,3 Corinna Burger,2... any immunologically relevant risk to rAAV gene transfer in the brain. On the contrary, our data... as a vector for human gene therapy ...

  

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

 

Collection: Biology and Medicine

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

Summary:  successfully applied to generate rAAV2 vectors with some cell- selective gene transfer capabilities28... blocks in the intracellular AAV gene delivery pathway47. Moreover, because the gene transfer... and therapeutic concentrations of human ...

  

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

 

Collection: Materials Science ; Chemistry

 
57 original article 2114 www.moleculartherapy.org vol.15no.12,21142123dec.2007
 

Summary:  -clinical testing of rAAV gene transfer to the lung, the results of the studies should be interpreted with caution... comparing rAAV gene transfer between airway epithelia in humans and monkeys, it was important to demonstrate... similar, thereby suggesting that their ...

  

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

 

Collection: Biology and Medicine

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

Summary:  of rAAV-mediated gene transfer in a CNL1-knockout mouse model [128] (see [119] for review) and one... , potential caveats for rAAV-based gene therapy in the CNS are also presented. Key Words: Parkinson disease... . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . ...

  

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

 

Collection: Biology and Medicine

 
59 JOURNAL OF VIROLOGY, 0022-538X/01/$04.00 0 DOI: 10.1128/JVI.75.15.69696976.2001
 

Summary:  of beta-glucuronidase from localized sites after AAV vector gene transfer results in widespread... and extrachromosomal rAAV genomes in mouse livers and their relative contribution to stable gene expression in vivo... uncertainties as to whether rAAV-mediated hepatic ...

  

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

 

Collection: Biology and Medicine

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

Summary:  as a gene therapy vector (1, 22, 29, 63). Recombinant AAV-2 (rAAV) carrying reporter genes has been shown... . Inhibitors of AAV-2 endocytosis and nuclear trafficking also reduce rAAV-mediated gene expression. HeLa cells... ,2 * Department of ...

  

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

 

Collection: Biology and Medicine


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

Summary:  transfer properties of these viruses are needed. AAV vectors have proven to be safe and efficient gene... with an AAV vector. Nat. Genet. 24, 257­261 (2000). 4. Manno, C.S. et al. AAV-mediated factor IX gene transfer... utilized as a ...

  

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

 

Collection: Materials Science ; Chemistry

 
62 HUMAN GENE THERAPY 18:8187 (January 2007) Mary Ann Liebert, Inc.
 

Summary:  to enhance in vivo gene transfer with AAV (Yan et al., 2004), we adminis- tered this drug systemically... product. Such a dual-AAV approach has been successfully tested with reporter genes in mouse muscle (Duan... the promising prospects of dual-rAAV vector ap- ...

  

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

 

Collection: Biology and Medicine

 
63 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... differs from wild type AAV. © 1997 Elsevier Science B.V. Keywords: Adeno-associated virus; Gene therapy... genes synthesize structural components of the virus. ...

  

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

 

Collection: Biology and Medicine

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

Summary:  neurons as measured by rAAV vector genomes (Figure 9d). dIscussIon Recombinant AAV-mediated gene transfer... Society of Gene Therapy Recombinant adeno-associated virus (rAAV) expresses no viral genes after... response, are extremely important ...

  

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

 

Collection: Biology and Medicine

 
65 MOLECULAR THERAPY Vol. 4, No. 6, December 2001 Copyright The American Society of Gene Therapy
 

Summary:  early disease targets for AAV- mediated gene transfer, hemophilia and muscular dystro- phy, affect males... -mediated gene transfer is extremely low. FIG. 5. AAV does not transduce murine germ cells. Human 293T cells (A... cells. To evaluate the safety of in vivo ...

  

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

 

Collection: Biology and Medicine

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

Summary:  disease in MPS VII mice following AAV-mediated neonatal gene transfer. Gene Ther 8: 1291­1298. #12;... , Intracranial AAV-mediated Gene Therapy, or Both in the Mouse Model of MPS IIIB Coy D Heldermon1,2 , Kevin K... , combining CNS-directed, ...

  

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

 

Collection: Environmental Sciences and Ecology ; Biology and Medicine

 
67 64 NATURE MEDICINE VOLUME 5 NUMBER 1 JANUARY 1999 Hemophilia is an X-linked recessive bleeding disorder that
 

Summary:  procedures. The studies here demonstrate that rAAV-mediated gene transfer of the hFIX cDNA can correct... gene transfer of recombinant AAV vectors. Nature Genet. 16, 270-275 (1997). 24. Miao, C.H. et al... of long-term hepatic gene ...

  

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

 

Collection: Biology and Medicine

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

Summary:  of transferring the human coagulation factor IX (hF.IX) gene into mouse liver by recombinant AAV (rAAV) vectors... , 30, 31, 37). Gene transfer to the liver by rAAV vectors is a relatively recent observation... extensively ex- plored as a ...

  

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

 

Collection: Biology and Medicine

 
69 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... of rAAV integration because integration occurs preferentially in genes and accompanies chromosomal... mutagenesis. Possible cancer-related genes were hit by rAAV ...

  

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

 

Collection: Biology and Medicine

 
70 Gene Therapy (1999) 6, 14271437 1999 Stockton Press All rights reserved 0969-7128/99 $12.00
 

Summary:  AAV (rAAV) has demonstrated consider- able promise as a gene therapy vector for certain organ systems... 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 ...

  

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

 

Collection: Biology and Medicine

 
71 Sites in the AAV5 capsid tolerant to deletions and tandem duplications Kaoru Hida a
 

Summary:  January 2010 Available online 25 January 2010 Keywords: Directed evolution AAV Viral gene delivery a b... s t r a c t Gene therapy vectors based on adeno-associated virus (AAV) have shown much promise... in the AAV2 capsid gene by site directed mutations. More ...

  

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

 
72 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
 

Summary:  (AAV)-based gene transfer techniques has proven to be an extremely pow- erful tool for treating many... offered tremendous potential for ex- panding AAV gene therapy to larger disease genes (8, 9, 11, 19, 26... , which have large disease genes are ...

  

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

 

Collection: Biology and Medicine

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

Summary:  JA, Zabner J. Secreted and trans- membrane mucins inhibit gene transfer with AAV4 more efficiently... as surrogate models for rAAV transduction and gene therapy for CF. Keywords: recombinant adeno-associated virus... recombinant genes (1­9). How- ever, ...

  

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

 

Collection: Biology and Medicine

 
74 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:  attention in the field of human gene therapy for more than 10 years. Although recombinant AAV (rAAV) vectors... extensively studied as a gene transfer vector. This vector serotype has also been used in clinical trials... - mophilia A, dual-vector approaches may be useful in the ap- ...

  

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

 

Collection: Biology and Medicine

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

Summary:  integration as the predom- inant mechanism of rAAV persistence in vivo. Muscle-mediated gene transfer... confirmed previous successes in rAAV-mediated gene transfer to muscle tissue (5, 10, 15, 19, 34... Adeno-associated viral (AAV) vectors have ...

  

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

 

Collection: Biology and Medicine

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

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 ...

  

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

 

Collection: Materials Science ; Chemistry

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

Summary:  ­30). These tissue-specific differences in rAAV-mediated gene transfer may be due, in part, to variable levels... large gene inserts or large promoter-gene combinations carried by two or more independent rAAV vectors... protein (16). In studies crucial to the ...

  

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

 

Collection: Biology and Medicine

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

Summary:  , such as neurons, cardiac and skeletal muscle, and hepatocytes [3­10]. Gene transfer with rAAV has been shown... ). Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector... : (352) 846-2739. E-mail: ...

  

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

 

Collection: Biology and Medicine

 
79 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... protein-coding genes have open reading frames small enough to fit within the low coding capacity of AAV (4... human diseases.18 Single-stranded AAV genomes modulate ...

  

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

 

Collection: Biology and Medicine

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

Summary:  causing significant hepatocellular injury. Quantitative evaluation of AAV-mediated liver gene transfer... To provide a quantitative evaluation of gene transfer to the liver lobes, we determined AAV copy number... whether higher gene ...

  

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

 

Collection: Biology and Medicine


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81 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... -directed gene transfer using adeno-associated virus (AAV) vectors has the potential to serve as therapy... liver-directed AAV-mediated ...

  

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

 

Collection: Biology and Medicine

 
82 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... transfer Recombinant adeno-associated viral vector serotype 2 (rAAV2) has proven to be an attractive gene... is imperative to realize the full potential of ...

  

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

 

Collection: Biology and Medicine

 
83 Transposon-Based Mutagenesis Generates Diverse Adeno-Associated Viral Libraries with Novel Gene
 

Summary:  ) Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector... . Briefly, a selectable marker is randomly inserted throughout the AAV2 cap gene and the resulting... "bookmarked" AAV cap gene is ...

  

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

 

Collection: Materials Science ; Chemistry

 
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... clinical trials of gene transfer for the treatment of cystic fibrosis, hemophilia B, and limb girdle mus... a 2-log-range linear dose response of rAAV transduction evaluated by ...

  

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

 

Collection: Biology and Medicine

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

Summary:  ;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 AAV5 gene transfer (Wal- ters et al., 2001... airways. LUNG GENE ...

  

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

 

Collection: Biology and Medicine

 
86 Heparin binding induces conformational changes in Adeno-associated virus Hazel C. Levy a
 

Summary:  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 Powell Gene Therapy Center... Heparin sulfate a b s t r a c t Adeno-associated virus serotype 2 (AAV2) ...

  

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

 

Collection: Biology and Medicine

 
87 Convection-Enhanced Delivery of AAV Vector in Parkinsonian Monkeys; In Vivo Detection of Gene Expression and Restoration
 

Summary:  -dopa/carbidopa administration following AAV­AADC gene transfer. (A) L-dopa levels were measured in the tissue punches following... and simple injection of AAV in MPTP- treated monkeys we found a significant increase of gene transfer... Convection-Enhanced Delivery of ...

  

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

 

Collection: Computer Technologies and Information Sciences ; Biology and Medicine

 
88 correspondence NATURE|VOL424|17JULY2003|www.nature.com/nature 251
 

Summary:  demonstrate that AAV vector DNA will preferentially integrate into active genes when delivered into the livers... suggests that recombinant AAV vectors may pose similar risks in gene-therapy trials. There are substantial... elements that are more likely than AAV vectors to activate a ...

  

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

 

Collection: Biology and Medicine

 
89 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:  findings demonstrating the efficacy of rAAV for gene targeting. In an effort to improve gene... the level of gene repair and rAAV transduction. Interestingly, however, our results demonstrated... . These results suggest that the S phase of the cell cycle may more efficiently facilitate ...

  

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

 

Collection: Biology and Medicine

 
90 REVIEW ARTICLE Adeno-Associated Virus Vectors and Hematology
 

Summary:  have shown the therapeutic value of AAV-mediated gene transfer in murine and/or canine hemo- philia B... gene transfer applications. Instead, it is applications involving the production of secreted proteins... AAV vectors do not include the rep ...

  

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

 

Collection: Biology and Medicine

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

Summary:  of CNS gene transfer by an adeno-associated virus (AAV) vector. Brain Res. 713: 99­107. 31. Phelps, C. H... factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nat. Genet. 16: 270­276. 63rAAV... of a control ...

  

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

 

Collection: Biology and Medicine

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

Summary:  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 (AAV) vector. Brain Res. 713: 99... gene transfer of ...

  

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

 

Collection: Biology and Medicine

 
93 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 ...

  

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

 

Collection: Engineering ; Computer Technologies and Information Sciences

 
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... . experiment 3: neuroprotection from intrastriatal 6-oHdA We next examined the impact of PTN gene transfer (2l... . These results are depicted in Figure 4a­d and g. To determine ...

  

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

 

Collection: Biology and Medicine

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

Summary:  Characterization of Intrastriatal Recombinant Adeno-Associated Virus-Mediated Gene Transfer... gene transfer techniques, i.e., systemic L-DOPA. If a gene therapy for PD is to be developed... , intrastriatal gene transfer must be demonstrated to be ...

  

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

 

Collection: Biology and Medicine

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

Summary:  : rAAV; Striatum; Huntington's disease; Mouse; Gene transfer; Neurological disorder; Genetic disorder... , FL, USA Received 28 July 2007; revised 30 October 2007; accepted 5 November 2007 Gene transfer... the expression of a subset of genes in the striatum. This effect ...

  

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

 

Collection: Biology and Medicine

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

Summary:  as a gene transfer vehicle. Construc- tion of recombinant AAV vectors is carried out by removing the viral... -Mediated Striatal Transgene Expression The first study to report intrastriatal gene transfer using rAAV... the dose of a protein by ...

  

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

 

Collection: Biology and Medicine

 
98 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 ...

  

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

 

Collection: Materials Science ; Chemistry

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

Summary:  ). Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector... reconstitutes gene expression from two independent rAAV vectors, each encoding unique, nonoverlapping halves... -associated virus, rAAV, homologous ...

  

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

 

Collection: Biology and Medicine

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

Summary:  epithelia (Liu et al., 2002). However, adenoviral vec- tor-mediated gene transfer currently is unsuitable... adeno-associated virus (rAAV) has attracted considerable interest as a vector for gene therapy because... increased the effectiveness of rAAV vectors for gene ...

  

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

 

Collection: Biology and Medicine


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