Sickle cell disease: progress towards combination drug therapy

BS Pace, A Starlard‐Davenport… - British journal of …, 2021 - Wiley Online Library
Summary Dr. John Herrick described the first clinical case of sickle cell anaemia (SCA) in
the United States in 1910. Subsequently, four decades later, Ingram and colleagues …

[HTML][HTML] Role of B-Cell Lymphoma/Leukemia 11A in Normal and Malignant Hematopoiesis

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 …

Reactivation of γ-globin expression using a minicircle DNA system to treat β-thalassemia

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 …

[图书][B] Large-scale Investigation of Fetal Hemoglobin Modulators and Inflammation Biomarkers in Sickle Cell Disease

M Cannon - 2021 - search.proquest.com
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 …

Nucleic acid therapy for β-thalassemia

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 …

[HTML][HTML] Transcriptional regulators of fetal hemoglobin

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 …

[PDF][PDF] Emerging therapies and management approaches in sickle cell disease (SCD): A critical review

S Patel, R Patel, SR Mukkala… - Journal of …, 2023 - researchgate.net
This comprehensive review delves into the intricate landscape of sickle cell disease (SCD),
a global genetic disorder impacting individuals, healthcare systems, and communities. The …

[PDF][PDF] Khamisi Pour G, Tamaddon G

N Obidi, F Saberi, A Taghavi, R Ranjbaran - We are distributing the … - researchgate.net
ABSTRACT Background: Small interfering RNAs (siRNAs) specifically target mRNAs and
induce silencing of protein-coding genes. MicroRNAs are single-stranded RNA molecules …