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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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Autors principals: 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
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group 2012
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Accés en línia: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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