Manipulation of Developmental Gamma-Globin Gene Expression: an Approach for Healing Hemoglobinopathies

Overview

Journal Mol Cell Biol

Specialty Cell Biology

Date 2020 Oct 20

PMID 33077498

Citations 8

Authors

Vigneshwaran Venkatesan

Saranya Srinivasan

Prathibha Babu

Saravanabhavan Thangavel

Affiliations

Soon will be listed here.

Abstract

β-Hemoglobinopathies are the most common monogenic disorders, and a century of research has provided us with a better understanding of the attributes of these diseases. Allogenic stem cell transplantation was the only potentially curative option available for these diseases until the discovery of gene therapy. The findings on the protective nature of fetal hemoglobin in sickle cell disease (SCD) and thalassemia patients carrying hereditary persistence of fetal hemoglobin (HPFH) mutations has given us the best evidence that the cure for β-hemoglobinopathies remains hidden in the hemoglobin locus. The detailed understanding of the developmental gene regulation of gamma-globin (γ-globin) and the emergence of gene manipulation strategies offer us the opportunity for developing a γ-globin gene-modified autologous stem cell transplantation therapy. In this review, we summarize different therapeutic strategies that reactivate fetal hemoglobin for the gene therapy of β-hemoglobinopathies.

Citing Articles

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Zinc finger nuclease-mediated gene editing in hematopoietic stem cells results in reactivation of fetal hemoglobin in sickle cell disease.

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