Výsledky vyhledávání

Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice

Autor: Joseph Muenzer, Haiyan Fu, Julianne DiRosario, D M McCarty, K Gulaid

Publikováno v: Gene therapy

The presence of the blood-brain barrier (BBB) presents the most critical challenge in therapeutic development for mucopolysaccharidosis (MPS) IIIB, a lysosomal storage disease with severe neurological manifestation, because of alpha-N-acetylglucosami

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c4982a0765449aa9ea578ddb07a3bdc3
https://doi.org/10.1038/gt.2009.85

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Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo

Autor: C E Toulson, P Naik, Paul E. Monahan, D M McCarty, Haiyan Fu, Richard Jude Samulski

Publikováno v: Gene Therapy. 10:2112-2118

An important limitation of recombinant adeno-associated virus (rAAV) vector efficiency is the requirement of hostcell-mediated synthesis of double-stranded DNA from the single-stranded genome. We have bypassed this step in a specialized self-compleme

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1cac6081a78c3e99e2381616358265f9
https://doi.org/10.1038/sj.gt.3302134

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The adeno-associated virus (AAV) Rep protein acts as both a repressor and an activator to regulate AAV transcription during a productive infection

Autor: Nicholas Muzyczka, D J Pereira, D M McCarty

Publikováno v: Journal of Virology. 71:1079-1088

Adeno-associated virus (AAV) uses three promoters, p5, p19, and p40, to regulate viral gene expression. The p5 and p19 promoters direct the synthesis of the viral regulatory proteins, Rep78 and -68 and Rep52 and -40, respectively. The p5 Rep proteins

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0e25eacacfbfa2b459f97c8a5632dd83
https://doi.org/10.1128/jvi.71.2.1079-1088.1997

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Identification of linear DNA sequences that specifically bind the adeno-associated virus Rep protein

Autor: J H Ryan, X Zhou, Nicholas Muzyczka, D M McCarty, I Zolotukhin, D J Pereira

Publikováno v: Journal of Virology. 68:4988-4997

We have used baculovirus-expressed Rep68 that has been purified to homogeneity to reexamine the binding properties of the Rep protein. We find that Rep68 is capable of binding to a linear DNA sequence that is contained within a 25-bp sequence of the

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2e0a3d392bc04626e0eb9e1a2c9450ac
https://doi.org/10.1128/jvi.68.8.4988-4997.1994

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Analysis of mutations in adeno-associated virus Rep protein in vivo and in vitro

Autor: D. M. Mccarty, Nicholas Muzyczka, Tie-Hua Ni

Publikováno v: Journal of Virology. 66:4050-4057

The adeno-associated virus (AAV) Rep protein is required for both viral DNA replication and transactivation of the AAV promoters. Here we report the effects of mutations in the rep gene on transcription and replication in vivo and terminal repeat bin

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::08220ec6ba6212f0382975e6e7aa747a
https://doi.org/10.1128/jvi.66.7.4050-4057.1992

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Sequences required for coordinate induction of adeno-associated virus p19 and p40 promoters by Rep protein

Autor: M Christensen, Nicholas Muzyczka, D. M. Mccarty

Publikováno v: Journal of Virology. 65:2936-2945

A series of contiguous 30-bp deletions were introduced into the regions upstream of the p19 and p40 promoters of adeno-associated virus (AAV), and the effects of these deletions on induction of AAV transcription by the rep gene products was evaluated

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::7b8bd830aea8b52f05d648af3fdc6035
https://doi.org/10.1128/jvi.65.6.2936-2945.1991

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Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis

Autor: D M McCarty, Richard Jude Samulski, Paul E. Monahan

Publikováno v: Gene therapy. 8(16)

Adeno-associated virus (AAV) vectors package single-stranded genomes and require host-cell synthesis of the complementary strand for transduction. However, when the genome is half wild-type size, AAV can package either two copies, or dimeric inverted

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::280a2bfed6513fbb171f5a20e396375b
https://pubmed.ncbi.nlm.nih.gov/11509958

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Oligodendrocyte-specific gene expression in mouse brain: use of a myelin-forming cell type-specific promoter in an adeno-associated virus

Autor: H, Chen, D M, McCarty, A T, Bruce, K, Suzuki

Publikováno v: Journal of neuroscience research. 55(4)

To explore the feasibility of cell type-specific gene expression in oligodendrocytes as a possible therapeutic approach for demyelinating diseases, the cell specificity, tissue specificity, and duration of gene expression were investigated using reco

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid________::9fd0529058be471c9bc4770e345ef024
https://pubmed.ncbi.nlm.nih.gov/10723060

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