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Article

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

Stem cell research & therapy - 31 Dec 2024

Chandraprabha Prathibha Babu, Azhagiri Manoj Kumar K, Venkatesan Vigneshwaran, Magis Wendy, Prasad Kirti, Suresh Sevanthy, Pai Aswin Anand, Marepally Srujan, Srivastava Alok, Mohankumar Kumarasamypet Murugesan, Martin David I K, Thangavel Saravanabhavan

Abstract excerpt

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 editing more than one regulatory sequence in the HBG promoter, the production of fetal hemoglobin (HbF) can be significantly...

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Share to Anemia, Sickle Cell
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Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 39736768
DOI: 10.1186/s13287-024-04117-0
PMCID: PMC11687217

Genes

HBG1
SCD
BCL11A
MSC

Provenance

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Enhanced fetal hemoglobin production via dual-beneficial mutation editing of the HBG promoter in hematopoietic stem and progenitor cells for β-hemoglobinopathies.PMID 39736768Read ↗

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