Publications:

Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47phox-Deficient Chronic Granulomatous Disease.

Human Gene Therapy

Schejtman, Andrea A; Vetharoy, Winston W; Choi, Uimook U; Rivat, Christine C; Theobald, Narda N; Piras, Giuseppa G; Leon-Rico, Diego D; Buckland, Karen K; Armenteros-Monterroso, Elena E; Benedetti, Sara S; Ashworth, Michael T MT; Rothe, Michael M; Schambach, Axel A; Gaspar, Hubert Bobby HB; Kang, Elizabeth M EM; Malech, Harry L HL; Thrasher, Adrian J AJ; Santilli, Giorgia G

Publication Date: 2021-09

Variant appearance in text: CGD: A84F

PubMed Link: 33740872

Variant Present in the following documents:

• Main text

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