Correction of Murine Rag2 Severe Combined Immunodeficiency by Lentiviral Gene Therapy Using a Codon-optimized RAG2 Therapeutic Transgene

Recombination activating gene 2 (RAG2) deficiency results in severe combined immunodeficiency (SCID) with complete lack of T and B lymphocytes. Initial gammaretroviral gene therapy trials for other types of SCID proved effective, but also revealed the necessity of safe vector design. We report the d...

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Detalhes bibliográficos
Main Authors: van Til, Niek P, de Boer, Helen, Mashamba, Nomusa, Wabik, Agnieszka, Huston, Marshall, Visser, Trudi P, Fontana, Elena, Poliani, Pietro Luigi, Cassani, Barbara, Zhang, Fang, Thrasher, Adrian J, Villa, Anna, Wagemaker, Gerard
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2012
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3464632/
https://ncbi.nlm.nih.gov/pubmed/22692499
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2012.110
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