Work

Preliminary 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: 2019

Type: conference-abstract

Source: Blood

DOI: 10.1182/blood-2019-125743

Authors: Angela R. Smith, Gary J. Schiller, Gregory M Vercellotti, Janet L. Kwiatkowski, Lakshmanan Krishnamurti, Erica B. Esrick, David A. Williams, Weston P. Miller, Adrian Woolfson, Mark C. Walters

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

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: W2988018983

Cites:

Cited by: 50

Related to: 10