Proteasome Inhibitors Enhance Gene Delivery by AAV Virus Vectors Expressing Large Genomes in Hemophilia Mouse and Dog Models: A Strategy for Broad Clinical Application

Delivery of genes that are larger than the wild-type adeno-associated virus (AAV) 4,681 nucleotide genome is inefficient using AAV vectors. We previously demonstrated in vitro that concurrent proteasome inhibitor (PI) treatment improves transduction by AAV vectors encoding oversized transgenes. In t...

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Bibliografiske detaljer
Main Authors: Monahan, Paul E, Lothrop, Clinton D, Sun, Junjiang, Hirsch, Matthew L, Kafri, Tal, Kantor, Boris, Sarkar, Rita, Tillson, D Michael, Elia, Joseph R, Samulski, R Jude
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group 2010
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Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2990516/
https://ncbi.nlm.nih.gov/pubmed/20700109
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2010.170
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