Enhanced fetal hemoglobin production via dual-beneficial mutation editing of the HBG promoter in hem...
Enhanced fetal hemoglobin production via dual-beneficial mutation editing of the HBG promoter in hematopoietic stem and progenitor cells for β-hemoglobinopathies
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Publisher
England: BioMed Central
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Language
English
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Publisher
England: BioMed Central
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Scope and Contents
Contents
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 product...
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Full title
Enhanced fetal hemoglobin production via dual-beneficial mutation editing of the HBG promoter in hematopoietic stem and progenitor cells for β-hemoglobinopathies
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TN_cdi_doaj_primary_oai_doaj_org_article_b32aa20255e7453e8753d8e3cc21811a
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_b32aa20255e7453e8753d8e3cc21811a
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ISSN
1757-6512
E-ISSN
1757-6512
DOI
10.1186/s13287-024-04117-0
