Effects of Vector Backbone and Pseudotype on Lentiviral Vector-mediated Gene Transfer: Studies in Infant ADA-Deficient Mice and Rhesus Monkeys

Systemic delivery of a lentiviral vector carrying a therapeutic gene represents a new treatment for monogenic disease. Previously, we have shown that transfer of the adenosine deaminase (ADA) cDNA in vivo rescues the lethal phenotype and reconstitutes immune function in ADA-deficient mice. In order...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Carbonaro Sarracino, Denise, Tarantal, Alice F, Lee, C Chang I., Martinez, Michele, Jin, Xiangyang, Wang, Xiaoyan, Hardee, Cinnamon L, Geiger, Sabine, Kahl, Christoph A, Kohn, Donald B
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2014
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4428412/
https://ncbi.nlm.nih.gov/pubmed/24925206
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2014.88
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