Work

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

Year: 2020

Type: article

Study design: Case Report

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.

Source: Blood Advances

DOI: 10.1182/bloodadvances.2019001330

Authors: Matthew M. Hsieh, Melissa Bonner, Francis John Pierciey, Naoya Uchida, James Rottman, Laura Demopoulos, Manfred Schmidt, Julie Kanter, Mark C. Walters, Alexis A. Thompson, Mohammed Asmal, John F. Tisdale

Institutions: National Institutes of Health, National Heart, Lung, and Blood Institute, Bluebird Bio (United States), University of Alabama at Birmingham, UAB Medicine, University of California, San Francisco, UCSF Benioff Children's Hospital, Northwestern University, Lurie Children's Hospital

Language: English

Topic: Virus-based gene therapy research

Subfield: Genetics

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,800

Funder: bluebird bio

OpenAlex ID: W3024470836

FWCI: 11.62

Cites: 33

Cited by: 151

Related to: 10