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 …
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 …
from a multipotent stem cell and terminating in a mature, enucleated erythrocyte. Altered red …
Gene therapy of hemoglobinopathies: progress and future challenges
Recently, gene therapy clinical trials have been successfully applied to
hemoglobinopathies, such as sickle cell disease (SCD) and β-thalassemia. Among the great …
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
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 …
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 …
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
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 …
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 …
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 …
encodes β-globin), mainly sickle cell disease (SCD) and β-thalassemia, become …
Potent and uniform fetal hemoglobin induction via base editing
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 …
cell disease. We compared five strategies in CD34+ hematopoietic stem and progenitor …
[图书][B] Hematology E-Book: Basic Principles and Practice
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 …
Hematology, 8th Edition, remains your# 1 choice for expert guidance in all areas of this …