Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID

Genetic deficiency of the nuclease DCLRE1C/Artemis causes radiosensitive severe combined immunodeficiency (RS-SCID) with lack of peripheral T and B cells and increased sensitivity to ionizing radiations. Gene therapy based on transplanting autologous gene-modified hematopoietic stem cells could sign...

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Опубликовано в: :Mol Ther Methods Clin Dev
Главные авторы: Charrier, Sabine, Lagresle-Peyrou, Chantal, Poletti, Valentina, Rothe, Michael, Cédrone, Grégory, Gjata, Bernard, Mavilio, Fulvio, Fischer, Alain, Schambach, Axel, de Villartay, Jean-Pierre, Cavazzana, Marina, Hacein-Bey-Abina, Salima, Galy, Anne
Формат: Artigo
Язык:Inglês
Опубликовано: American Society of Gene & Cell Therapy 2019
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Article
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC6838972/
https://ncbi.nlm.nih.gov/pubmed/31720302
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.08.014
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