H Zhang, J Zeng, F Zhang, J Liu, L Liang - Biology, 2025 - mdpi.com
B-cell lymphoma/leukemia 11A (BCL11A) is a crucial transcriptional regulator, widely recognized for its role in controlling fetal hemoglobin and its potential as a gene therapy …
SP Ma, XX Gao, GQ Zhou, HK Zhang, JM Yang… - Gene, 2022 - Elsevier
Reactivation of fetal hemoglobin by editing the B-cell lymphoma/leukemia 11A (BCL11A) erythroid enhancer is an effective gene therapy for β-thalassemia. Using the CRISPR/Cas9 …
Abstract Sickle Cell Disease (SCD) is a debilitating hematological disorder that affects 1 out of every 365 African-American births with an even greater prevalence worldwide. The …
A d'Arqom - Biologics: Targets and Therapy, 2020 - Taylor & Francis
Abstract β-thalassemia is caused by mutations in the β-globin gene which diminishes or abolishes β-globin chain production. This reduction causes an imbalance of the α/β-globin …
GP Dos Santos, LT Rabi, AA Bezerra… - … , Transfusion and Cell …, 2024 - Elsevier
Sickle cell anemia is a hereditary disease caused by sickle-shaped red blood cells that can lead to vaso-occlusive crises. Treatment options are currently limited, highlighting the need …
This comprehensive review delves into the intricate landscape of sickle cell disease (SCD), a global genetic disorder impacting individuals, healthcare systems, and communities. The …