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Sample search results for: aav omar parve

Page:   2  3  4  5 
 
1 Suppression of Insult-Induced Neurogenesis in Adult Rat Brain by Brain-Derived Neurotrophic Factor
 

Summary:  transduction of recombinant adeno-associated virus (rAAV) carrying the BDNF gene, inhibits the brain insult... Breeding and Research Center, Ejby, Denmark) weighing 295­ 310 g received unilateral injections of rAAV... containing either the green fluorescent protein (GFP) gene (GFP- rAAV, n 10) or the genes for GFP and BDNF

  

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

 

Collection: Biology and Medicine

 
2 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... degenerative diseases. In prior work, subretinal injections of AAV2, AAV5, and AAV2 pseudotyped with AAV5... capsids (AAV2/5) showed variable retinal pigmented ...

  

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

 

Collection: Biology and Medicine

 
3 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... .4kb · Serotypes differentially infect cell types AAV as a Viral Vector ...

  

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

 

Collection: Biology and Medicine

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

Summary:  . A particularly interesting candidate is AAV8, which can efficiently and rapidly transduce a wide range of tissues... in vivo. To further unravel the mechan- isms behind AAV8 transduction, we used yeast two- hybrid analyses... with the corresponding portion of the AAV2 capsid in yeast, but not with AAV5. We ...

  

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

 

Collection: Biology and Medicine

 
5 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 AAV-2 vectors possess many

  

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

 

Collection: Biology and Medicine

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

Summary:  Unique Biologic Properties of Recombinant AAV1 Transduction in Polarized Human Airway Epithelia... . In the present study, we sought to compare the biologic properties of rAAV2/1, rAAV2/2, and rAAV2/5 transduction... method. Results demonstrated that apical transduction of human airway epithelia with ...

  

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

 

Collection: Biology and Medicine

 
7 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... @ufl.edu. Available online 1 July 2004 Recombinant adeno-associated virus 2 (rAAV2) has been shown to deliver genes... to neurons effectively in the brain, retina, and spinal cord. The characterization of new AAV serotypes has

  

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

 
8 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:  2001 In the presence of complementing adeno-associated virus type 2 (AAV-2) Rep proteins, AAV-2 genomes... can be pseudotyped with the AAV-5 capsid to assemble infectious virions. Using this pseudotyping... strategy, the involvement of the ubiquitin-proteasome system in AAV-5 and AAV-2 ...

  

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

 

Collection: Biology and Medicine

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

Summary:  the transduction efficiency of different adeno-associated virus (AAV) capsid serotypes encod- ing for green... fluorescent protein (GFP) flanked by AAV2 inverted terminal repeats in the nonhuman primate basal ganglia... ). Six intact young adult cynomolgus monkeys received a single 10µl injection of AAV2/1-GFP, ...

  

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

 

Collection: Biology and Medicine

 
10 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... decade. In all, 85% of the current 2052 PubMed references on AAV (as of December 2004) have been... delivery, an increased appreciation for the complexities of AAV biology has emerged. The biology

  

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

 

Collection: Biology and Medicine

 
11 SHORT COMMUNICATION Comparative biology of rAAV transduction in ferret,
 

Summary:  SHORT COMMUNICATION Comparative biology of rAAV transduction in ferret, pig and human airway... - associated virus (rAAV)-mediated gene therapies to the lung for cystic fibrosis (CF). As new ferret and pig... the transduction biology of three rAAV serotypes (AAV1, 2 and 5) in human, ferret, pig and ...

  

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

 

Collection: Biology and Medicine

 
12 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... to be in part due to its ability to uncoat more readily than other AAV serotypes such as AAV2, understanding all... the processes behind AAV8 transduction is important for its application and optimal ...

  

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

 

Collection: Biology and Medicine

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

Summary:  strategy for producing high-titer adeno-associated virus (AAV) vectors derived from six different AAV... serotypes (AAV-1 to AAV-6). The method, referred to as "HOT," is helper virus free, optically controllable... , and based on transfection of only two plasmids, i.e., an AAV vector ...

  

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

 

Collection: Biology and Medicine

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

Summary:  Characterization of the Relationship of AAV Capsid Domain Swapping to Liver Transduction Efficiency... 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 ...

  

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

 

Collection: Biology and Medicine

 
15 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... inside AAV6 or AAV8 capsids can transduce the liver with higher efficiency, but the ...

  

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

 

Collection: Biology and Medicine

 
16 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

 
17 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:  serotype 5 (AAV5) requires sialic acid on host cells to bind and infect. Other parvoviruses, including... . In contrast to AAV5, AAV2 does not bind sialic acid, but rather binds heparan sulfate proteo- glycans at its... to cell receptor attachment, we determined the structure of AAV5 by cryo-electron ...

  

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

 

Collection: Biology and Medicine

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

Summary:  -associated virus (AAV) are two of the most efficient at transducing nondividing cells. We have constructed... recombinant adenovirus (AdVLacZ), adeno-associated virus serotype 2 (AAV2LacZ), and pseudotyped adeno... -associated virus serotype 5 and 8 (AAV5LacZ, AAV8LacZ) carrying the LacZ reporter, and ...

  

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

 

Collection: Biology and Medicine

 
19 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... , and infection of off-target cells. Recently, we have implemented directed evolution of AAV, involving... -efficiency in vitro recombination to create a large (107 ), diverse library of random chimeras of numerous parent ...

  

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

 

Collection: Materials Science ; Chemistry

 
20 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... -stranded (ss) rAAV genomes become dou- ble stranded (ds), circularized, and/or concatemerized concomitant

  

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

 

Collection: Biology and Medicine


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21 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... creation of AAV vec- tors with valuable new gene delivery properties. We have engineered novel AAV variants... capable of efficient glia transduction by employing directed evolution with a panel of four distinct ...

  

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

 

Collection: Materials Science ; Chemistry

 
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... Superior to That of AAV8 Katsuya Inagaki,1 Sally Fuess,2 Theresa A. Storm,2 Gregory A. Gibson,3 Charles F... recently shown that recombinant adeno-associated virus serotype 8 (rAAV8) is a robust alternative serotype

  

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

 

Collection: Biology and Medicine

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

Summary:  -associated virus (AAV) are highly promising due to several desirable features of this parent virus, including... maintenance of the viral genome. However, several problems should be addressed to enhance the utility of AAV... vectors, particularly those based on AAV2, the best characterized AAV serotype. ...

  

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

 

Collection: Materials Science ; Chemistry

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

Summary:  . As a specific application of this finding, we generated adenovirus­adeno-associated virus (AAV) hybrid vectors... , first-generation Ad vectors containing AAV inverted terminal repeat sequences (ITRs) flanking a reporter... gene cassette inserted into the E1 region. We hypothe- sized that the AAV ITRs present within

  

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... approaches, it remains unclear to what extent commonly used AAV serotypes transduce adult progenitors... in the lung. In this study, we evaluated the life span and proliferative capac- ity of rAAV1-, 2-, and 5

  

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

 

Collection: Biology and Medicine

 
26 JOURNAL OF VIROLOGY, June 2010, p. 58805889 Vol. 84, No. 12 0022-538X/10/$12.00 doi:10.1128/JVI.02719-09
 

Summary:  -associated virus (AAV) serotypes AAV1 to AAV3, AAV5, and AAV9; the Erythrovirus B19; and the newly discovered human... parvovirus genotypes 4 (Parv4) and 5 (Parv5) (23, 27, 50). Of these, only B19 had been implicated in disease... until the ...

  

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

 

Collection: Biology and Medicine

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

Summary:  Colosi, and Mark A. Kay We report the generation and use of pseudotyped adeno-associated viral (AAV... ) vectors for the liver-specific expres- sion of human blood coagulation factor IX (hFIX). Therefore, an AAV... -2 genome encoding the hfIX gene was cross-pack- aged into capsids of AAV types 1 to 6 using

  

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

 

Collection: Biology and Medicine

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

Summary:  52242 Received 22 March 2001/Accepted 11 May 2001 Adeno-associated virus (AAV)-based muscle gene therapy... . For these reasons, efforts aimed at increasing the efficacy of AAV-mediated gene delivery to muscle have... compared the efficiency of gene delivery to mouse muscle cells for recombinant AAV type 2 ...

  

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

 

Collection: Biology and Medicine

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

Summary:  February 2008/Accepted 2 April 2008 Adeno-associated virus (AAV) serotypes differ broadly in transduction... , however, their use in patients is restricted by prevalent anti-AAV immunity or by their inadequate... performance in specific targets, exemplified by the AAV type 2 (AAV-2) prototype in the liver. ...

  

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

 

Collection: Biology and Medicine

 
30 Parvovirus 4 as Potential Cause
 

Summary:  but unexpected agents, and novel agents (2). Human parvovirus 4 (PARV4) is a single... -stranded DNAvirusfirstdetectedin2005(3).InfectionswithPARV4 are accompanied by acute viremia for several weeks (C. Sharp et al... and high PARV4 levels in their cerebrospinal fluid (CSF). The Study We obtained CSF from a cohort

  

Source: Wang, David - Department of Molecular Microbiology, Washington University in St. Louis

 

Collection: Biology and Medicine

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

Summary:  Society of Gene & Cell Therapy Selecting the most efficient recombinant adeno- associated virus (rAAV... promis- ing rAAV serotypes following lung coinfection. Analysis of differential luciferase activity... in chimpanzee airway brushings demonstrated a 20-fold higher efficiency for rAAV1 over rAAV5 at ...

  

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

 

Collection: Biology and Medicine

 
32 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

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

Summary:  Publishers Ltd. Rate Limiting Steps of AAV Transduction and Implications for Human Gene Therapy S. Sanlioglu1... : Despite the fact that adeno-associated virus type 2 (AAV2) is an extremely attractive gene therapy vector... to broaden the array of target organs for this vector, molecular studies on the mechanism(s) of AAV

  

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

 

Collection: Biology and Medicine

 
34 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:  -Strand Genome Conversion of Adeno-Associated Virus Type 2 (AAV-2) and AAV-5 Is Not Rate Limiting following... Recombinant adeno-associated virus type 5 (rAAV-5) is known to efficiently transduce airway epithelia via... apical infection. In contrast, rAAV-2 has been shown to be inherently ineffective at ...

  

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

 

Collection: Biology and Medicine

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

Summary:  -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... ) Curcumin (Allertein) GGTI 2418 (Tigris) hI-conI (Iconics) AAV/dXIAP(Nuerologix) AAV/NPY ...

  

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

 

Collection: Biology and Medicine

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

Summary:  , Louisiana2 Received 7 July 1998/Accepted 1 October 1998 A central feature of the adeno-associated virus (AAV... , the molecular processes associated with episomal long-term persis- tence of AAV genomes are only poorly... understood. To investigate these mechanisms, we have utilized a re- combinant AAV (rAAV) ...

  

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

 

Collection: Biology and Medicine

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

Summary:  have been demonstrated to enhance recombinant adeno-associated virus (rAAV) transduction. Such findings... are the foundation of developing new and innovative strategies to im- prove the efficiency of rAAV as a gene therapy... stimuli increase rAAV transduction are only partially understood but have been suggested to involve ...

  

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

 

Collection: Biology and Medicine

 
38 Neuropathological and Behavioral Consequences of Adeno-Associated Viral Vector-Mediated
 

Summary:  to the striatum at biologically active levels via recombinant adeno-associated viral (rAAV) gene transfer 4... , which agreed with the specificity for interneuron transduction by the rAAV vector. These data show... -associated virus (rAAV), can deliver enzymes or trophic factors to the brain at bioactive levels for one year

  

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

 

Collection: Biology and Medicine

 
39 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 exhibit similar restricted liver transduction, we performed a ...

  

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

 

Collection: Biology and Medicine

 
40 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... method relied on in vivo selection of rAAV integrants and could be employed for the liver... but not for other tissues. Here, we describe a novel method for high-throughput rAAV integration site analysis

  

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

 

Collection: Biology and Medicine


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41 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... expression is not completely understood, stable rAAV genomes exist as ...

  

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

 

Collection: Biology and Medicine

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

Summary:  1999 Adeno-associated virus (AAV) is inefficient at infecting differentiated airway epithelia because... of a lack of receptors at the apical surface. We hypothesized that incorporation of AAV in a calcium... phosphate coprecipi- tate would circumvent this barrier. Interestingly, coprecipitation of AAV type 2

  

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

 

Collection: Biology and Medicine

 
43 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 quantities of AAV vectors

  

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

 

Collection: Materials Science ; Chemistry

 
44 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:  - tion and transduction of recombinant adeno-associated virus type 2 (rAAV-2) and rAAV-5 serotypes... - tives) for their ability to induce rAAV transduction. The anthracycline derivatives doxorubicin... and aclarubicin also significantly augmented rAAV transduction in airway cell lines, polarized human airway

  

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

 

Collection: Biology and Medicine

 
45 [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 the easy and rapid cloning of sh

  

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

 

Collection: Biology and Medicine

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

Summary:  (AAV) serotypes can infect various cells of the retina when administered subretinally, but the retinal... and survival of retinal neurons. Intravitreal administration could cir- cumvent this problem, but only AAV2 can... several AAV serotype capsids and fol- lowed their retinal distribution after intravitreal injection. ...

  

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

 

Collection: Materials Science ; Chemistry

 
47 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... expression. Here we sought to determine the therapeutic potential of a capsid-modified AAV ...

  

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

 

Collection: Biology and Medicine

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

Summary:  by nature, could greatly enhance the respiratory infectivity of a virus. Adeno-associated viruses (AAV... for replication and gene expression. Wild-type AAV is a 4.7 kb single-stranded DNA virus that contains 2 ORFs: rep... ­3) that assemble to form the viral capsid (12). Although not necessarily considered a respiratory virus, ...

  

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

 

Collection: Materials Science ; Chemistry

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

Summary:  deficiency, and cancer. In this report we demonstrate that recombinant adeno-associated virus (rAAV) gene... . This enhancement of rAAV transduction in AT cells is correlated with an increased abundance of circular form rAAV... evaluating AAV trafficking using Cy3-labeled virus suggest that a nuclear ...

  

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

 

Collection: Biology and Medicine

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

Summary:  Available online 27 April 2004 Abstract Recombinant adeno-associated virus (rAAV) receptor binding... -limiting steps in rAAV type-2 (rAAV-2) transduction. Several strategies have been developed to enhance rAAV-2... therapy vector. To this end, the current study has investigated novel methods for augmenting ...

  

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

 

Collection: Biology and Medicine

 
51 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... a period of 5 weeks concomitant with the conversion of the single- stranded rAAV molecules into high... that the transgene product is made in about 5% of hepatocytes, suggesting that most rAAV-mediated gene expression

  

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

 

Collection: Biology and Medicine

 
52 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 AAV vectors to the nucleus of a host cell

  

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

 

Collection: Biology and Medicine

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

Summary:  - virus, and adeno-associated virus (AAV), have been used in strategies conceived for functional... . The develop- ment of AAV as a gene therapy vehicle for treating CF has several unique advantages based on its... viral biology. For example, wild-type AAV infections are known to occur in the respiratory epithelium

  

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

 

Collection: Biology and Medicine

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

Summary:  -associated virus (rAAV) vectors for gene therapy of inherited disorders have demon- strated considerable potential... for molecular medicine. Recent identification of the viral receptor and corecep- tors for AAV type 2 (AAV-2) has... with this vector. However, the mechanisms by which AAV-2 enters cells ...

  

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

 

Collection: Biology and Medicine

 
55 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... (MOI) of rAAV. It has previously been shown that the introduction of a DNA double-stranded ...

  

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

 

Collection: Biotechnology ; Biology and Medicine

 
56 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... may thereby reduce off-target side effects. We have genetically engineered an AAV variant capable

  

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

 

Collection: Materials Science ; Chemistry

 
57 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:  with adeno-associated virus 8 (AAV-8) pseudotypes, i.e., AAV-2 genomes packaged into AAV-8 capsids. Here we... (inverted terminal repeats [ITRs]) derived from AAV genotypes other than 2. To this end, we generated two... IX) gene flanked by ITRs from AAV genotypes 1 through 6. Initial in ...

  

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

 

Collection: Biology and Medicine

 
58 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... optimization or scalability issues, and most AAV serotypes do not bind heparin or sialic acid, used for AAV2... /3 or AAV4/5 purification, requiring the development of new chromatography strategies. ...

  

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

 

Collection: Materials Science ; Chemistry

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

Summary:  /Accepted 14 July 2000 Adeno-associated virus (AAV) is a single-stranded DNA parvovirus that causes... controlling infection. In this study, we have examined endocytic and intracellular trafficking of AAV-2 using... that internalization of heparan sulfate proteo- glycan-bound AAV-2 requires V 5 integrin and activation of the ...

  

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

 

Collection: Biology and Medicine

 
60 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:  . Circulating Anti-Wild-Type Adeno-Associated Virus Type 2 (AAV2) Antibodies Inhibit Recombinant AAV2 (rAAV2... )-Mediated, but Not rAAV5-Mediated, Gene Transfer in the Brain Carmen S. Peden,1,2,3 Corinna Burger,2... -associated virus type 2 (AAV2), with 30% expressing neutralizing Ab ...

  

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

 

Collection: Biology and Medicine


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

Summary:  Society of Gene Therapy Recombinant adeno-associated virus (rAAV) expresses no viral genes after... transduction. In addition, because the brain is relatively immunoprivileged, intracranial rAAV transduction may... be immunologically benign due to a lack of antigen presentation. However, pre- exposure to AAV allows neutralizing

  

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

 

Collection: Biology and Medicine

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

Summary:  of integrated recombinant AAV genomes Dongsheng Duan b, Krishna J. Fisher a,b, John F. Burda a,b, John F... November 1996 Abstract Adeno-associated Virus (AAV) has emerged as a promising vector for gene therapy... of this virus is based on studies evaluating wild type AAV or recombinant AAV which was ...

  

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

 

Collection: Biology and Medicine

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

Summary:  damage including UV, hyd- of rAAV transduction. Furthermore, using electron spin res- roxyurea... and ionizing irradiation have been shown to aug- onance spectroscopy (ESR), we demonstrated that both ment rAAV... /or activation of cellular factors important in the inhibits the augmentation of rAAV transduction following

  

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

 

Collection: Biology and Medicine

 
64 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... ) sequences mediate intermolecular recombination of AAV genomes, with the goal of engineering more efficient... vectors for dual-vector trans-splicing approaches. To this end, we generated a novel AAV hybrid-ITR vector

  

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

 

Collection: Biology and Medicine

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

Summary:  model of MPS IIIB, neo- natal mice were treated with intracranial AAV2/5-NAGLU (AAV), syngeneic bone... marrow transplant (BMT), or both (AAV/BMT). All treatments resulted in some improve- ment in clinical... phenotype. Adeno-associated viral (AAV) treatment resulted in improvements in lifespan, motor ...

  

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

 

Collection: Environmental Sciences and Ecology ; Biology and Medicine

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

Summary:  (AAV) 2 variants with enhanced properties. High-throughput selection processes were designed to isolate... neutralization and deliver genes more efficiently than wild-type capsid in the presence of anti-AAV serum... evolution to generate `designer' gene delivery vectors with specified, enhanced properties. AAV is a 4

  

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

 

Collection: Materials Science ; Chemistry

 
67 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 in combination with proteasome modulation enhanced dual-vector delivery of a ...

  

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

 

Collection: Biology and Medicine

 
68 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... - pression of transgenes. New rAAV serotypes have been characterized and demonstrated to have improved... - ties of rAAV that have been studied in the nervous system such as cell tropism, duration of transgene

  

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

 

Collection: Biology and Medicine

 
69 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:  concatemerization of the recombinant adeno-associated virus (rAAV) genome has been suggested as the predominant... process facilitating long-term rAAV transduction in muscle. A recent study (S. Song, P. J. Laipis, K. I... -PKcs regulates the removal of free rAAV vector ends in muscle tissue. In the present study, we have sought

  

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

 

Collection: Biology and Medicine

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

Summary:  January 1999/Accepted 30 March 1999 Recombinant adeno-associated virus (rAAV) vectors allow for sustained... expression of transgene products from mouse liver following a single portal vein administration. Here a rAAV... vector expressing human coagulation factor F.IX (hF.IX), AAV-EF1 -F.IX (hF.IX expression was controlled

  

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

 

Collection: Biology and Medicine

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

Summary:  Society of Gene Therapy Biological Differences in rAAV Transduction of Airway Epithelia in Humans... reflect the effectiveness of recombinant adeno-associated virus (rAAV)-mediated gene delivery... to assessing the biology of rAAV-mediated transduction for three serotypes, we characterized the bioelectric

  

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

 

Collection: Biology and Medicine

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

Summary:  fibrosis (CF) using murine models. In this context, recombinant adeno-associated virus (rAAV) type 2 and rAAV... epithelia (rAAV2 rAAV5) as compared with mouse lung (rAAV5 rAAV2). It is unclear if these differences... biology. To this end, we compared ...

  

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

 

Collection: Biology and Medicine

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

Summary:  , California 94305 Received 15 February 2001/Accepted 29 April 2001 Recombinant adeno-associated virus (rAAV... and extrachromosomal rAAV genomes in mouse livers and their relative contribution to stable gene expression in vivo... of a series of hF.IX-expressing rAAV vectors. At doses of 2.4 1011 to 3.0 1011 vector genomes per ...

  

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

 

Collection: Biology and Medicine

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

Summary:  Sites in the AAV5 capsid tolerant to deletions and tandem duplications Kaoru Hida a , Sang Y. Won b... 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

  

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

 
75 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... several naturally occurring AAV variants have been isolated with a variety of tropisms, or cellular... /Principal Findings: We have previously applied directed evolution to create several novel AAV variants capable

  

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

 

Collection: Materials Science ; Chemistry

 
76 NATURE BIOTECHNOLOGY VOLUME 25 NUMBER 10 OCTOBER 2007 1111 and drugs that act directly on disease genes.
 

Summary:  and hubs. AAV vectors and tumorigenicity Mark A Kay Chromosomal integration of rAAV vectors may induce... therapy, recombinant adeno- associated viral (rAAV) vectors have shown special promise because... , a recent study by Donsante et al.1, published in Science, has revived a debate on the safety of rAAV

  

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

 

Collection: Biology and Medicine

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

Summary:  Adeno-associated viral (AAV) vectors have demonstrated great utility for long-term gene expression... in muscle tissue. However, the mechanisms by which recombinant AAV (rAAV) genomes persist in muscle tissue... remain unclear. Using a recombinant shuttle vector, we have demonstrated that circularized rAAV ...

  

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

 

Collection: Biology and Medicine

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

Summary:  of Germline Transmission of Vector Sequences Following Systemic Administration of Recombinant AAV-2 Vector... cells. To evaluate the safety of in vivo recombinant adeno-asso- ciated virus (rAAV) mediated gene... following intramuscular (IM) injection of rAAV in four species of male animals (mouse, rat, rabbit and dog

  

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

 

Collection: Biology and Medicine

 
79 64 NATURE MEDICINE VOLUME 5 NUMBER 1 JANUARY 1999 Hemophilia is an X-linked recessive bleeding disorder that
 

Summary:  to have toxicity and immunogenicity associated with the therapy. Recombinant adeno-associated viral (rAAV... coagulation. In hemophilic dogs, a dose of rAAV that was approximately 1/10 per body weight that given to mice... 1999 65 ARTICLES Fig. 1 Structures of the rAAV vectors. ITR, AAV inverted terminal ...

  

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

 

Collection: Biology and Medicine

 
80 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... of a potent cytomegalovirus (CMV) immediate early promoter (AAV-MD-rGDNF) was constructed. Two ex- periments... were performed to evaluate the time course of expression of rAAV-mediated GDNF protein expression

  

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

 

Collection: Biology and Medicine


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

Summary:  , random adeno-associated viral (AAV) libraries that can be selected for a desired function. First, the AAV... extension process. The resulting PCR product is then cloned into pSub2 to generate a diverse (>106) AAV2... plasmid library. Finally, the AAV2 plasmid library is used to package a diverse pool of ...

  

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

 

Collection: Materials Science ; Chemistry

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

Summary:  Heparin sulfate a b s t r a c t Adeno-associated virus serotype 2 (AAV2) uses heparan sulfate proteoglycan... as a cell surface-attachment receptor. In this study the structures of AAV2 alone and complexed with heparin... density in the interior of the capsid in the AAV2­heparin complex was interpreted as nucleic acid

  

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

 

Collection: Biology and Medicine

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

Summary:  -associated virus vector (AAV vector), adenoviral vector, gene therapy, hemophilia, retroviral vector Curr Opin... -mail: kponder@im.wustl.edu Current Opinion in Hematology 2006, 13:301­307 Abbreviations AAV adeno... . The status of the liver-directed adeno-associated virus (AAV) vector-mediated gene therapy trial in humans

  

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

 

Collection: Biology and Medicine

 
84 RAPID COMMUNICATION Structural Analysis of Adeno-Associated Virus Transduction Circular Intermediates
 

Summary:  , 1999; accepted May 24, 1999 Recombinant adeno-associated virus (rAAV) has recently been demonstrated... of the ITR arrays. To gain further insight into potential mechanisms by which AAV circular genomes are formed... sequences. The most predominant form consisted of a structure similar to that of previously generated AAV

  

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

 

Collection: Biology and Medicine

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

Summary:  @ufl.edu. Available online 18 January 2006 Recombinant adeno-associated virus (rAAV) is derived from a small human... and supports long-term transgene expression in the nervous system. These properties make rAAV a reasonable... candidate vector for treating neurological disorders. Indeed, rAAV is currently being used in five ...

  

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

 

Collection: Biology and Medicine

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

Summary:  , Iowa Received 15 April 1999/Accepted 26 July 1999 Long-term recombinant AAV (rAAV) transgene expression... in muscle has been associated with the molecular conversion of single-stranded rAAV genomes to high... form remain to be defined. To this end, we tested whether concatamerization of rAAV circular

  

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

 

Collection: Biology and Medicine

 
87 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... deletions, which may lead to loss-of-function insertional mutagenesis. Here, by analyzing 347 rAAV

  

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

 

Collection: Biology and Medicine

 
88 Effect of hull gap on pressure distribution under PAR platform Randy Lee M. Mallari1 and Dr. Konstantin Matveev2
 

Summary:  Objectives · To study the effect of hull gap on the PARV pressure distribution. · To correlate idealized jet... augmented ram vehicle (PARV) is a new concept of amphibious ground-effect craft which has several advantages... over traditional air-cushion vehicles and wing-in-ground craft [1]. The PARV is supported by a high

  

Source: Collins, Gary S. - Department of Physics and Astronomy, Washington State University

 

Collection: Physics

 
89 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:  , California 945022 Received 23 May 2002/Accepted 7 August 2002 Recombinant adeno-associated virus (rAAV... of liver transduction are not fully understood, and furthermore, the relationships between rAAV dose... with rAAV vectors, we injected mice with two different human factor IX or Escherichia coli lac

  

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

 

Collection: Biology and Medicine

 
90 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... the basis for future efforts to develop a recombinant AAV (rAAV)-based gene therapy approach

  

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

 

Collection: Biology and Medicine

 
91 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... model of the metabolic disease hereditary tyrosinemia type I was used to test whether targeted AAV... and adult mice were treated with AAV serotypes 2 and 8 carrying a wild-type genomic sequence for repairing

  

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

 

Collection: Biology and Medicine

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

Summary:  ,4 HIROYUKI NAKAI,1 LINDA B. COUTO,3 and MARK A. KAY1,2 ABSTRACT Recombinant adeno-associated virus (rAAV... 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 vector expressing the human factor IX gene from

  

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

 

Collection: Biology and Medicine

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

Summary:  -associated virus (AAV) vectors in various somatic tissues has been shown to depend heavily on the AAV type from... which the vector capsid proteins are derived. Among the AAV types studied, AAV6 efficiently transduces... that with the proper AAV capsid proteins and promoter sequences, ...

  

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

 

Collection: Biology and Medicine

 
94 Iterated DFT Based Techniques for Join Size Estimation
 

Summary:  the phase information. The rst version of this method called Approximation by Absolute Value (AAV) gives... tree representation of the vectors obtained through the iterated application of the DFT. TAA uses AAV... DFT based Algorithms 4.1 Motivation: AAV and Self Join We start by motivating our approach by using

  

Source: Egecioglu, Ömer - Department of Computer Science, University of California at Santa Barbara

 

Collection: Computer Technologies and Information Sciences

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

Summary:  . Assessing the potential for AAV vector genotoxicity in a murine model http... THERAPY Assessing the potential forAAV vector genotoxicity in a murine model Hojun Li,1,2 Nirav Malani,3... Howard Hughes Medical Institute, Philadelphia, PA Gene transfer using adeno-associated vi- rus (AAV

  

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

 

Collection: Biology and Medicine

 
96 This article was originally published in a journal published by Elsevier, and the attached copy is provided by Elsevier for the
 

Summary:  of parvalbumin and greater levels of the Parv2 (relative to Parv1) isoform as compared to red muscle. Parvalbumin... antibodies were employed, Parv-19 (Sigma, P3088) and 11-M (Alpha Diagnostic). After 2 h of incubation... high parvalbumin content were probed with two anti-parvalbumin antibodies, Parv 19 and ...

  

Source: Coughlin, David J. - Science Division, Widener University

 

Collection: Environmental Sciences and Ecology

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

Summary:  recombinant adeno-associated virus type 2/1 (rAAV2/1), to provide neuroprotection and functional restoration... 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

  

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

 

Collection: Biology and Medicine

 
98 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... . The onset of rAAV-mediated GDNF transgene expression near the substantia nigra was determined to begin... survival data indicate that rAAV-GDNF delivery results in a highly significant sparing of nigral DA neurons

  

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

 

Collection: Biology and Medicine

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

Summary:  used high titer purified recombi- nant adeno-associated virus (rAAV) containing cDNAs encod- ing human... stain- ing was observed 3 weeks after rAAV transduction, with little detectable perturbation... of the tissue. Six months after intrastri- atal rAAV transduction, TH staining was present but apparently

  

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

 

Collection: Biology and Medicine

 
100 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... the expression of a subset of genes in the striatum. This effect was independent of rAAV transduction... reported herein. Thus, long-term rAAV-mediated RNAi in the brain does not, in and of itself, negatively

  

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

 

Collection: Biology and Medicine


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