Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

Abroad (travel likely)RecruitingPHASE1

Conditions

Sickle Cell Disease · Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0) · Beta-Thalassemia · Transfusion Dependent Beta-Thalassaemia

Simple summaryAI-assisted

This summary is produced by a language model and is experimental. It may be incomplete or inaccurate and is not a substitute for professional medical advice.

Locations

Boston Children's Hospital, Boston, Massachusetts, United States
Contact: Study coordinator (Emily Morris) · gene.therapy@childrens.harvard.edu · 617-355-8724

View source on ClinicalTrials.gov

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