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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Bibliographische Detailangaben
Veröffentlicht in:Hum Gene Ther
Hauptverfasser: Sweeney, Colin L., Choi, Uimook, Liu, Chengyu, Koontz, Sherry, Ha, Seung-Kwon, Malech, Harry L.
Format: Artigo
Sprache:Inglês
Veröffentlicht: Mary Ann Liebert, Inc. 2017
Schlagworte:
Research Articles
Online Zugang: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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