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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Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group 2010
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Original Article
On-line přístup: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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