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Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?

More than 20 years ago, X-linked severe combined immunodeficiency (SCID-X1) appeared to be the best condition to test the feasibility of hematopoietic stem cell gene therapy. The seminal SCID-X1 clinical studies, based on first-generation gammaretroviral vectors, demonstrated good long-term immune r...

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Shranjeno v:
Bibliografske podrobnosti
izdano v:Hum Gene Ther
Main Authors: Cavazzana, Marina, Six, Emmanuelle, Lagresle-Peyrou, Chantal, André-Schmutz, Isabelle, Hacein-Bey-Abina, Salima
Format: Artigo
Jezik:Inglês
Izdano: Mary Ann Liebert, Inc. 2016
Teme:
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC4779287/
https://ncbi.nlm.nih.gov/pubmed/26790362
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2015.137
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