Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease-Reference-Cited by-同舟云学术

Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease

Published:2020-05-12 Issue:9 Volume:4 Page:2058-2063
ISSN:2473-9529
Container-title:Blood Advances
language:en
Short-container-title:

Author:

Hsieh Matthew M.¹^ORCID,Bonner Melissa²,Pierciey Francis John²,Uchida Naoya¹^ORCID,Rottman James²,Demopoulos Laura²,Schmidt Manfred³,Kanter Julie⁴^ORCID,Walters Mark C.⁵^ORCID,Thompson Alexis A.⁶⁷^ORCID,Asmal Mohammed²,Tisdale John F.¹^ORCID

Affiliation:

1. Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD;

2. bluebird bio, Inc, Cambridge, MA;

3. GeneWerk GmbH, Heidelberg, Germany;

4. Division of Hematology and Oncology, University of Alabama at Birmingham, Birmingham, AL;

5. University of California San Francisco Benioff Children’s Hospital, Oakland, CA;

6. Division of Hematology, Oncology and Stem Cell Transplant, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL; and

7. Department of Pediatrics, Feinberg School of Medicine, Northwestern University, Chicago, IL

Abstract

Key Points Ability to accurately attribute adverse events post–gene therapy is required to describe the benefit-risk of these novel treatments. A SCD patient developed myelodysplastic syndrome post-LentiGlobin treatment; we show how insertional oncogenesis was excluded as the cause.

Publisher

American Society of Hematology

Subject

Hematology

Link

http://ashpublications.org/bloodadvances/article-pdf/4/9/2058/1728701/advancesadv2019001330.pdf

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