Work

Updated Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia

Year: 2021

Type: conference-abstract

Source: Blood

DOI: 10.1182/blood-2021-147907

Authors: Mark C. Walters, Angela R. Smith, Gary J. Schiller, Erica B. Esrick, David A. Williams, Tatiana Gogoleva, Didier Rouy, Bettina M. Cockroft, Gregory M. Vercellotti

Institutions: UCSF Benioff Children's Hospital, University of Minnesota, University of California, Los Angeles, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston Children's Hospital, Sangamo BioSciences (United States)

Language: English

Topic: Hemoglobinopathies and Related Disorders

Subfield: Genetics

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W4200522731

Cites:

Cited by: 24

Related to: 10