Academic literature on the topic 'AAV2'

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Journal articles on the topic "AAV2"

1

Rutledge, Elizabeth A., Christine L. Halbert, and David W. Russell. "Infectious Clones and Vectors Derived from Adeno-Associated Virus (AAV) Serotypes Other Than AAV Type 2." Journal of Virology 72, no. 1 (1998): 309–19. http://dx.doi.org/10.1128/jvi.72.1.309-319.1998.

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ABSTRACT Adeno-associated viruses (AAVs) are single-stranded dependent parvoviruses being developed as transducing vectors. Although at least five serotypes exist (AAV types 1 to 5 [AAV1 to -5]), only AAV2, AAV3, and AAV4 have been sequenced, and the vectors in use were almost all derived from AAV2. Here we report the cloning and sequencing of a second AAV3 genome and a new AAV serotype designated AAV6 that is related to AAV1. AAV2, AAV3, and AAV6 were 82% identical at the nucleotide sequence level, and AAV4 was 75 to 78% identical to these AAVs. Significant sequence variation was noted in por
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2

Xin, Ke-Qin, Hiroaki Mizukami, Masashi Urabe, et al. "Induction of Robust Immune Responses against Human Immunodeficiency Virus Is Supported by the Inherent Tropism of Adeno-Associated Virus Type 5 forDendritic Cells." Journal of Virology 80, no. 24 (2006): 11899–910. http://dx.doi.org/10.1128/jvi.00890-06.

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ABSTRACT The ability of adeno-associated virus serotype 1 to 8 (AAV1 to AAV8) vectors expressing the human immunodeficiency virus type 1 (HIV-1) Env gp160 (AAV-HIV) to induce an immune response was evaluated in BALB/c mice. The AAV5 vector showed a higher tropism for both mouse and human dendritic cells (DCs) than did the AAV2 vector, whereas other AAV serotype vectors transduced DCs only poorly. AAV1, AAV5, AAV7, and AAV8 were more highly expressed in muscle cells than AAV2. An immunogenicity study of AAV serotypes indicates that AAV1, AAV5, AAV7, and AAV8 vectors expressing the Env gp160 gen
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3

Yang, Grace S., Michael Schmidt, Ziying Yan, et al. "Virus-Mediated Transduction of Murine Retina with Adeno-Associated Virus: Effects of Viral Capsid and Genome Size." Journal of Virology 76, no. 15 (2002): 7651–60. http://dx.doi.org/10.1128/jvi.76.15.7651-7660.2002.

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ABSTRACT 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 epithelium (RPE) and photoreceptor cell transduction, while AAV2/1 predominantly transduced the RPE. To more thoroughly compare the efficiencies of gene transfer of AAV2, AAV3, AAV5, and AAV6, we quantified, using stereological methods, the kinetics and efficiency of AAV transduction to mouse photoreceptor cells. We observed per
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4

Boye, Sanford L., Antonette Bennett, Miranda L. Scalabrino, et al. "Impact of Heparan Sulfate Binding on Transduction of Retina by Recombinant Adeno-Associated Virus Vectors." Journal of Virology 90, no. 8 (2016): 4215–31. http://dx.doi.org/10.1128/jvi.00200-16.

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ABSTRACTAdeno-associated viruses (AAVs) currently are being developed to efficiently transduce the retina following noninvasive, intravitreal (Ivt) injection. However, a major barrier encountered by intravitreally delivered AAVs is the inner limiting membrane (ILM), a basement membrane rich in heparan sulfate (HS) proteoglycan. The goal of this study was to determine the impact of HS binding on retinal transduction by Ivt-delivered AAVs. The heparin affinities of AAV2-based tyrosine-to-phenylalanine (Y-F) and threonine-to-valine (T-V) capsid mutants, designed to avoid proteasomal degradation d
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5

Lins-Austin, Bridget, Saajan Patel, Mario Mietzsch, et al. "Adeno-Associated Virus (AAV) Capsid Stability and Liposome Remodeling During Endo/Lysosomal pH Trafficking." Viruses 12, no. 6 (2020): 668. http://dx.doi.org/10.3390/v12060668.

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Adeno-associated viruses (AAVs) are small, non-pathogenic ssDNA viruses being used as therapeutic gene delivery vectors for the treatment of a variety of monogenic diseases. An obstacle to successful gene delivery is inefficient capsid trafficking through the endo/lysosomal pathway. This study aimed to characterize the AAV capsid stability and dynamics associated with this process for a select number of AAV serotypes, AAV1, AAV2, AAV5, and AAV8, at pHs representative of the early and late endosome, and the lysosome (6.0, 5.5, and 4.0, respectively). All AAV serotypes displayed thermal melt tem
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Chai, Zheng, Xintao Zhang, Amanda Lee Dobbins, Ellie Azure Frost, R. Jude Samulski, and Chengwen Li. "Chimeric Capsid Proteins Impact Transduction Efficiency of Haploid Adeno-Associated Virus Vectors." Viruses 11, no. 12 (2019): 1138. http://dx.doi.org/10.3390/v11121138.

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Our previous studies have demonstrated that haploid AAV vectors made from capsids of two different serotypes induced high transduction and prevented serotype-specific antibody binding. In this study, we explored the transduction efficiency of several haploid viruses, which were made from the VP1/VP2 of one serotype and VP3 of another compatible serotype. After systemic injection of 2 × 1010 vg of AAV vectors into mice, the haploid AAV vectors, composed of VP1/VP2 from serotypes 8 or 9, and VP3 from AAV2, displayed a two to seven-fold increase in liver transduction compared with those of parent
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7

Majowicz, Anna, Nolukholo Ncete, Floris van Waes, et al. "Seroprevalence of Pre-Existing Nabs Against AAV1, 2, 5, 6 and 8 in South African Hemophilia B Patient Population." Blood 134, Supplement_1 (2019): 3353. http://dx.doi.org/10.1182/blood-2019-128217.

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Introduction Several studies have shown that the induction of antibodies by natural exposure to various AAV serotypes can compromise the subsequent use of AAV as a gene therapy vector, limiting patient eligibility for AAV-delivered therapeutics. The implications of pre-existing antibodies to AAV serotypes are very different: Levels of anti-AAV2 or anti-AAV8 neutralizing antibodies (NABs) as low as 5 have been related to a decrease or even total impairment of AAV liver transduction after systemic delivery in humans. However, successful gene transfer has been reported in patients with anti-AAV5
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8

Nam, Hyun-Joo, Michael Douglas Lane, Eric Padron, et al. "Structure of Adeno-Associated Virus Serotype 8, a Gene Therapy Vector." Journal of Virology 81, no. 22 (2007): 12260–71. http://dx.doi.org/10.1128/jvi.01304-07.

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ABSTRACT Adeno-associated viruses (AAVs) are being developed as gene therapy vectors, and their efficacy could be improved by a detailed understanding of their viral capsid structures. AAV serotype 8 (AAV8) shows a significantly greater liver transduction efficiency than those of other serotypes, which has resulted in efforts to develop this virus as a gene therapy vector for hemophilia A and familial hypercholesterolemia. Pseudotyping studies show that the differential tissue tropism and transduction efficiencies exhibited by the AAVs result from differences in their capsid viral protein (VP)
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9

Jiang, Haiyan, David Lillicrap, Susannah Patarroyo-White, et al. "Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs." Blood 108, no. 1 (2006): 107–15. http://dx.doi.org/10.1182/blood-2005-12-5115.

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Hemophilia A, a deficiency of functional coagulation factor VIII (FVIII), is treated via protein replacement therapy. Restoring 1% to 5% of normal blood FVIII activity prevents spontaneous bleeding, making the disease an attractive gene therapy target. Previously, we have demonstrated short-term activity of a liver-specific AAV2 vector expressing canine B-domain-deleted FVIII (cFVIII) in a hemophilia canine model. Here, we report the long-term efficacy and safety of AAV-cFVIII vectors of serotypes 2, 5, 6, and 8 in both hemophilia A mice and dogs. AAV6-cFVIII and AAV8-cFVIII restored physiolog
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10

Silveria, Mark A., Edward E. Large, Grant M. Zane, Tommi A. White та Michael S. Chapman. "The Structure of an AAV5-AAVR Complex at 2.5 Å Resolution: Implications for Cellular Entry and Immune Neutralization of AAV Gene Therapy Vectors". Viruses 12, № 11 (2020): 1326. http://dx.doi.org/10.3390/v12111326.

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Adeno-Associated Virus is the leading vector for gene therapy. Although it is the vector for all in vivo gene therapies approved for clinical use by the US Food and Drug Administration, its biology is still not yet fully understood. It has been shown that different serotypes of AAV bind to their cellular receptor, AAVR, in different ways. Previously we have reported a 2.4Å structure of AAV2 bound to AAVR that shows ordered structure for only one of the two AAVR domains with which AAV2 interacts. In this study we present a 2.5Å resolution structure of AAV5 bound to AAVR. AAV5 binds to the first
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