Work

Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown, β-Globin Induction and Simultaneous Repression of β-Globin for the Potential Treatment of Sickle Cell Disease

Year: 2017

Type: article

Source: Blood

DOI: 10.1182/blood.v130.suppl_1.3557.3557

Authors: Olivier Nègre, Christian Brendel, Geoffrey Parsons, Min Luo, Myriam Armant, Swaroopa Guda, Lauryn Christiansen, Gretchen Lewis, Gábor Veres, Melissa Bonner, David A. Williams

Institutions: Bluebird Bio (United States), Boston Children's Hospital, Harvard University

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: closed

OpenAlex ID: W3136005036

FWCI:

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