Enhanced fetal hemoglobin production via dual-beneficial mutation editing of the HBG promoter in hematopoietic stem and progenitor cells for β-hemoglobinopathies

Enhanced fetal hemoglobin production via dual-beneficial mutation editing of the HBG promoter in hematopoietic stem and progenitor cells for β-hemoglobinopathies

Abstract Background Sickle cell disease (SCD) and β-thalassemia patients with elevated gamma globin (HBG1/G2) levels exhibit mild or no symptoms. To recapitulate this natural phenomenon, the most coveted gene therapy approach is to edit the regulatory sequences of HBG1/G2 to reactivate them. By edit...

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Main Authors: Prathibha Babu Chandraprabha, Manoj Kumar K. Azhagiri, Vigneshwaran Venkatesan, Wendy Magis, Kirti Prasad, Sevanthy Suresh, Aswin Anand Pai, Srujan Marepally, Alok Srivastava, Kumarasamypet Murugesan Mohankumar, David I. K. Martin, Saravanabhavan Thangavel
Format: Article
Language:English
Published: BMC 2024-12-01
Series:Stem Cell Research & Therapy
Subjects:
β-hemoglobinopathies
Gene therapy
Hematopoietic stem cells
Homology-directed gene editing
Single-stranded oligonucleotides
Online Access:https://doi.org/10.1186/s13287-024-04117-0
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