Article

Safety and Efficacy of CTX001 in Patients with Transfusion-Dependent β-Thalassemia and Sickle Cell Disease: Early Results from the Climb THAL-111 and Climb SCD-121 Studies of Autologous CRISPR-CAS9-Modified CD34+ Hematopoietic Stem and Progenitor Cells

2020-11-04

Abstract excerpt

Background: BCL11A is a key transcription factor that suppresses the production of fetal hemoglobin (HbF) in red blood cells (RBCs), leading to the production of adult Hb (HbA). In diseases with hemoglobin production defects such as b-thalassemia, or in sickle cell disease (SCD), HbF upregulation could ameliorate anemia and reduce transfusion requirements, such as in β-thalassemia, or reduce clinical complications...

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Literature Corpus work: 4d899baa-8b3f-5475-b810-ba459181afee
DOI: 10.1182/blood-2020-139575

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