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

Chronic granulomatous disease (CGD) is characterized by defects in the production of microbicidal reactive oxygen species (ROS) by phagocytes. Testing of gene and cell therapies for the treatment of CGD in human hematopoietic cells requires preclinical transplant models. The use of the lymphocyte-de...

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Detalhes bibliográficos
Publicado no:Hum Gene Ther
Main Authors: Sweeney, Colin L., Choi, Uimook, Liu, Chengyu, Koontz, Sherry, Ha, Seung-Kwon, Malech, Harry L.
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2017
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5549813/
https://ncbi.nlm.nih.gov/pubmed/28264583
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2017.005
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