A Small Key for a Heavy Door: Genetic Therapies for the Treatment of Hemoglobinopathies

A Small Key for a Heavy Door: Genetic Therapies for the Treatment of Hemoglobinopathies

Throughout the past decades, the search for a treatment for severe hemoglobinopathies has gained increased interest within the scientific community. The discovery that ɤ-globin expression from intact HBG alleles complements defective HBB alleles underlying β-thalassemia and sickle cell disease, has...

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Bibliographic Details
Main Authors: Hidde A. Zittersteijn, Cornelis L. Harteveld, Stefanie Klaver-Flores, Arjan C. Lankester, Rob C. Hoeben, Frank J. T. Staal, Manuel A. F. V. Gonçalves
Format: Article
Language:English
Published: Frontiers Media S.A. 2021-02-01
Series:Frontiers in Genome Editing
Subjects:
gene therapy
genome editing
hemoglobinopathies
fetal globin induction
thalasseamia
sickle-cell disease (SCD)
Online Access:https://www.frontiersin.org/articles/10.3389/fgeed.2020.617780/full

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https://www.frontiersin.org/articles/10.3389/fgeed.2020.617780/full

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