CRISPR-Mediated Knockout ofCybbin NSG Mice Establishes a Model of Chronic Granulomatous Disease for Human Stem-Cell Gene Therapy Transplants-Reference-Cited by-同舟云学术

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

Published:2017-07 Issue:7 Volume:28 Page:565-575
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Sweeney Colin L.¹,Choi Uimook¹,Liu Chengyu²,Koontz Sherry¹,Ha Seung-Kwon³,Malech Harry L.¹

Affiliation:

1. Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland.

2. Transgenic Core, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland.

3. Neuroimmunology Branch, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2017.005

Reference29 articles.

1. Common Severe Infections in Chronic Granulomatous Disease

2. Concentrated RD114-pseudotyped MFGS-gp91phox vector achieves high levels of functional correction of the chronic granulomatous disease oxidase defect in NOD/SCID/ 2-microglobulin-/- repopulating mobilized human peripheral blood CD34+ cells

3. Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

4. Successful Combination of Sequential Gene Therapy and Rescue Allo-HSCT in Two Children with X-CGD - Importance of Timing

5. Simian immunodeficiency virus lentivector corrects human X-linked chronic granulomatous disease in the NOD/SCID mouse xenograft

Cited by 11 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Advances in CRISPR/Cas gene therapy for inborn errors of immunity;Frontiers in Immunology;2023-03-27

2. Lifelong Immune Modulation Versus Hematopoietic Cell Therapy for Inborn Errors of Immunity;The Journal of Allergy and Clinical Immunology: In Practice;2021-02

3. Progress in treating chronic granulomatous disease;British Journal of Haematology;2020-07-08

4. Humanized mice are precious tools for evaluation of hematopoietic gene therapies and preclinical modeling to move towards a clinical trial;Biochemical Pharmacology;2020-04

5. Using CRISPR/Cas9 for Gene Knockout in Immunodeficient NSG Mice;Methods in Molecular Biology;2018-10-24

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