Enhanced fetal hemoglobin production via dual-beneficial mutation editing of the HBG promoter in hematopoietic stem and progenitor cells for β-hemoglobinopathies
Cas9 RNP-ssODN-based nucleotide conversion at the HBG promoter offers a promising gene therapy approach to ameliorate the phenotypes of β-thalassemia and SCD. The developed approach can simplify and broaden applications that require the cointroduction of multiple nucleotide modifications in HSPCs.
Authors
- Srujan Kumar Marepally (ORCID: https://orcid.org/0000-0002-2451-3359)
- Manoj Kumar K. Azhagiri
- Aswin Anand Pai (ORCID: https://orcid.org/0000-0001-9421-8585)
- Vigneshwaran Venkatesan (ORCID: https://orcid.org/0000-0003-3814-9292)
- David I. K. Martin
- Wendy Magis
- K. Manjunatha Prasad (ORCID: https://orcid.org/0000-0001-8743-4503)
- Saravanabhavan Thangavel (ORCID: https://orcid.org/0000-0001-6760-4106)
- Sevanthy Suresh
- Alok Srivastava (ORCID: https://orcid.org/0000-0002-2132-8123)
- Prathibha Babu Chandraprabha
- Kumarasamypet M. Mohankumar
Institutions
- Manipal Academy of Higher Education (IN)
- Christian Medical College, Vellore (IN)
- Christian Medical College (IN)
- UCSF Benioff Children's Hospital (US)
Publication Details
- Journal
- Stem Cell Research & Therapy
- Published
- 2024-12-31
- DOI
- https://doi.org/10.1186/s13287-024-04117-0
- Citations
- 6
- Primary Topic
- Hemoglobinopathies and Related Disorders
- Type
- article
- Field-Weighted Citation Impact
- 2.63
Funders
- Department of Biotechnology, Ministry of Science and Technology, India
- Indian Council of Medical Research