CRISPR-Mediated Knockout of Cybb in NSG Mice Establishes a Model of Chronic Granulomatous Disease for Human Stem-Cell Gene Therapy Transplants.

Sweeney, Colin L; Choi, Uimook; Liu, Chengyu; Koontz, Sherry; Ha, Seung-Kwon; Malech, Harry L

Sweeney, Colin L; Choi, Uimook; Liu, Chengyu; Koontz, Sherry; Ha, Seung-Kwon; Malech, Harry L.

Affiliation

Sweeney CL; 1 Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases , National Institutes of Health, Bethesda, Maryland.
Choi U; 1 Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases , National Institutes of Health, Bethesda, Maryland.
Liu C; 2 Transgenic Core, National Heart, Lung, and Blood Institute, National Institutes of Health , Bethesda, Maryland.
Koontz S; 1 Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases , National Institutes of Health, Bethesda, Maryland.
Ha SK; 3 Neuroimmunology Branch, National Institute of Neurological Disorders and Stroke , National Institutes of Health, Bethesda, Maryland.
Malech HL; 1 Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases , National Institutes of Health, Bethesda, Maryland.

Hum Gene Ther ; 28(7): 565-575, 2017 07.

Article de En | MEDLINE | ID: mdl-28264583

Sujet(s)
Mots clés

CRISPR/Cas9; Cybb; NSG mouse; chronic granulomatous disease; knockout

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Texte intégral: 1 Collection: 01-internacional Base de données: MEDLINE Sujet principal: Thérapie génétique / Transplantation de cellules souches / Clustered regularly interspaced short palindromic repeats / NADPH Oxidase 2 / Granulomatose septique chronique Limites: Animals / Female / Humans Langue: En Journal: Hum Gene Ther Sujet du journal: GENETICA MEDICA / TERAPEUTICA Année: 2017 Type de document: Article

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