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...
Topics
- Fetal Hemoglobin
- Gene Editing
- Humans
- Animals
- Promoter Regions, Genetic
- Mice
- Hematopoietic Stem Cells
- gamma-Globins
- beta-Thalassemia
- Hemoglobinopathies
- Mutation
- Anemia, Sickle Cell