Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult-Reference-Cited by-同舟云学术

Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult

Published:2017-09-14 Issue:11 Volume:130 Page:1327-1335
ISSN:0006-4971
Container-title:Blood
language:en
Short-container-title:

Author:

Morris Emma C.¹²,Fox Thomas¹,Chakraverty Ronjon³⁴,Tendeiro Rita¹,Snell Katie⁵⁶,Rivat Christine⁵,Grace Sarah³,Gilmour Kimberly⁶,Workman Sarita²,Buckland Karen⁵,Butler Katie⁵,Chee Ronnie²,Salama Alan D.⁷,Ibrahim Hazem⁸,Hara Havinder⁵,Duret Cecile⁵,Mavilio Fulvio⁹,Male Frances¹⁰,Bushman Frederick D.¹⁰,Galy Anne⁹,Burns Siobhan O.¹²,Gaspar H. Bobby⁵⁶,Thrasher Adrian J.⁵⁶

Affiliation:

1. Institute of Immunity and Transplantation, University College London, London, United Kingdom;

2. Department of Immunology and

3. Department of Haematology, Royal Free London NHS Foundation Trust, London, United Kingdom;

4. Cancer Institute, University College London, London, United Kingdom;

5. Institute of Child Health, University College London, London, United Kingdom;

6. Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom;

7. Centre for Nephrology, University College London, London, United Kingdom;

8. Department of Histopathology, Royal Free London NHS Foundation Trust, London, United Kingdom;

9. Genethon, Evry, France; and

10. Department of Microbiology, University of Pennsylvania School of Medicine, Philadelphia, PA

Abstract

Key Points We describe the first successful use of gene therapy in a severely affected adult with WAS. Gene therapy is a viable strategy for adult WAS patients with severe chronic disease complications where allogeneic transplantation presents.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Link

http://ashpublications.org/blood/article-pdf/130/11/1327/1402533/blood777136.pdf

Reference27 articles.

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2. Deletion of Wiskott-Aldrich syndrome protein triggers Rac2 activity and increased cross-presentation by dendritic cells;Baptista;Nat Commun,2016

3. Mechanisms of WASp-mediated hematologic and immunologic disease;Burns;Blood,2004

4. Wiskott-Aldrich syndrome protein deficiency in B cells results in impaired peripheral homeostasis;Meyer-Bahlburg;Blood,2008

5. Current and emerging treatment options for Wiskott-Aldrich syndrome;Worth;Expert Rev Clin Immunol,2015

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