CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease-Reference-Cited by-同舟云学术

CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease

Published:2017-01-11 Issue:372 Volume:9 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

De Ravin Suk See¹,Li Linhong²,Wu Xiaolin³,Choi Uimook¹,Allen Cornell²,Koontz Sherry¹,Lee Janet¹,Theobald-Whiting Narda¹,Chu Jessica¹,Garofalo Mary¹,Sweeney Colin¹,Kardava Lela⁴,Moir Susan⁴,Viley Angelia²,Natarajan Pachai²,Su Ling³,Kuhns Douglas¹,Zarember Kol A.¹,Peshwa Madhusudan V.²,Malech Harry L.¹

Affiliation:

1. Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA.

2. MaxCyte Inc., Gaithersburg, MD 20878, USA.

3. Cancer Research Technology Program, Leidos Biomedical Research Inc., Frederick, MD 21701, USA.

4. Laboratory of Immunoregulation, National Institutes of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA.

Abstract

CRISPR-mediated gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease resulted in functional human leukocytes in mice after transplantation.

Funder

Intramural Research Program of NIAID

NIH

Maryland Stem Cell Research Fund

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference31 articles.

1. Comparison of outcomes of hematopoietic stem cell transplantation without chemotherapy conditioning by using matched sibling and unrelated donors for treatment of severe combined immunodeficiency

2. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1

3. Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome