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An Emerging Era of Clinical Benefit From Gene Therapy

An Emerging Era of Clinical Benefit From Gene Therapy Opinion Editorials represent the opinions of the authors and JAMA EDITORIAL and not those of the American Medical Association. Harry L. Malech, MD; Hans D. Ochs, MD The report in this issue of JAMA by Hacein-Bey Abina and in those studies because selective outgrowth and persistence of colleagues provides strong evidence that gene therapy T-cell gene marking was enough to achieve substantial durable using myeloid/lymphoid conditioning combined with subse- clinical benefit even though there was no persistent gene mark- quent infusion of lentivector- ing correction of B cells or natural killer cells. Many of the authors + 2,3 transduced autologous CD34 of those pioneering studies are also the authors of the current Related article page 1550 hematopoietic stem cells gene therapy trial for WAS. (HSCs) achieves substantial In contrast to X-linked SCID, initial attempts to use a simi- restoration of immune function associated with prolonged lar approach to gene therapy for ADA-SCID by infusion of au- clinical benefit to patients with severe phenotype Wiskott- tologous HSCs transduced with a murine retrovirus vector with- Aldrich syndrome (WAS). Prior to treatment, 6 of the 7 pa- out prior myeloid conditioning did not result in the desired levels tients included in the http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png JAMA American Medical Association

An Emerging Era of Clinical Benefit From Gene Therapy

JAMA , Volume 313 (15) – Apr 21, 2015

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Publisher
American Medical Association
Copyright
Copyright 2015 American Medical Association. All Rights Reserved. Applicable FARS/DFARS Restrictions Apply to Government Use.
ISSN
0098-7484
eISSN
1538-3598
DOI
10.1001/jama.2015.2055
pmid
25898049
Publisher site
See Article on Publisher Site

Abstract

Opinion Editorials represent the opinions of the authors and JAMA EDITORIAL and not those of the American Medical Association. Harry L. Malech, MD; Hans D. Ochs, MD The report in this issue of JAMA by Hacein-Bey Abina and in those studies because selective outgrowth and persistence of colleagues provides strong evidence that gene therapy T-cell gene marking was enough to achieve substantial durable using myeloid/lymphoid conditioning combined with subse- clinical benefit even though there was no persistent gene mark- quent infusion of lentivector- ing correction of B cells or natural killer cells. Many of the authors + 2,3 transduced autologous CD34 of those pioneering studies are also the authors of the current Related article page 1550 hematopoietic stem cells gene therapy trial for WAS. (HSCs) achieves substantial In contrast to X-linked SCID, initial attempts to use a simi- restoration of immune function associated with prolonged lar approach to gene therapy for ADA-SCID by infusion of au- clinical benefit to patients with severe phenotype Wiskott- tologous HSCs transduced with a murine retrovirus vector with- Aldrich syndrome (WAS). Prior to treatment, 6 of the 7 pa- out prior myeloid conditioning did not result in the desired levels tients included in the

Journal

JAMAAmerican Medical Association

Published: Apr 21, 2015

References