Anemia: progress in molecular mechanisms and therapies

VG Sankaran, MJ Weiss - Nature medicine, 2015 - nature.com
Anemia is a major source of morbidity and mortality worldwide. Here we review recent
insights into how red blood cells (RBCs) are produced, the pathogenic mechanisms …

Erythropoiesis: insights into pathophysiology and treatments in 2017

A Zivot, JM Lipton, A Narla, L Blanc - Molecular Medicine, 2018 - Springer
Erythropoiesis is a tightly-regulated and complex process originating in the bone marrow
from a multipotent stem cell and terminating in a mature, enucleated erythrocyte. Altered red …

Gene therapy of hemoglobinopathies: progress and future challenges

Y Ikawa, A Miccio, E Magrin… - Human Molecular …, 2019 - academic.oup.com
Recently, gene therapy clinical trials have been successfully applied to
hemoglobinopathies, such as sickle cell disease (SCD) and β-thalassemia. Among the great …

Domain-focused CRISPR screen identifies HRI as a fetal hemoglobin regulator in human erythroid cells

JD Grevet, X Lan, N Hamagami, CR Edwards… - Science, 2018 - science.org
Increasing fetal hemoglobin (HbF) levels in adult red blood cells provides clinical benefit to
patients with sickle cell disease and some forms of β-thalassemia. To identify potentially …

[HTML][HTML] Gene therapy for β-hemoglobinopathies

M Cavazzana, C Antoniani, A Miccio - Molecular therapy, 2017 - cell.com
β-Thalassemia and sickle cell disease (SCD) are the world's two most widely disseminated
hereditary hemoglobinopathies. β-Thalassemia originated in the Mediterranean, Middle …

Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells

MK Cromer, J Camarena, RM Martin, BJ Lesch… - Nature medicine, 2021 - nature.com
Abstract β-Thalassemia pathology is due not only to loss of β-globin (HBB), but also to
erythrotoxic accumulation and aggregation of the β-globin-binding partner, α-globin …

Molecular and cellular mechanisms that regulate human erythropoiesis

AL Caulier, VG Sankaran - Blood, The Journal of the American …, 2022 - ashpublications.org
To enable effective oxygen transport,∼ 200 billion red blood cells (RBCs) need to be
produced every day in the bone marrow through the fine-tuned process of erythropoiesis …

A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition

EA Traxler, Y Yao, YD Wang, KJ Woodard, R Kurita… - Nature medicine, 2016 - nature.com
Disorders resulting from mutations in the hemoglobin subunit beta gene (HBB; which
encodes β-globin), mainly sickle cell disease (SCD) and β-thalassemia, become …

Potent and uniform fetal hemoglobin induction via base editing

T Mayuranathan, GA Newby, R Feng, Y Yao… - Nature …, 2023 - nature.com
Inducing fetal hemoglobin (HbF) in red blood cells can alleviate β-thalassemia and sickle
cell disease. We compared five strategies in CD34+ hematopoietic stem and progenitor …

[图书][B] Hematology E-Book: Basic Principles and Practice

R Hoffman, EJ Benz, LE Silberstein, H Heslop, J Weitz… - 2022 - books.google.com
Extensively revised, comprehensive content from leading global contributors ensures that
Hematology, 8th Edition, remains your# 1 choice for expert guidance in all areas of this …