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In vivo HSC prime editing rescues sickle cell disease in a mouse model
- Source :
- Blood; 20230101, Issue: Preprints
- Publication Year :
- 2023
-
Abstract
- •Correction of the sickle-cell mutation and disease phenotypes is achieved by in vivo HSC transduction with vectorized prime editors.•Our approach for in vivo HSC prime editing that does not require HSC transplantation and myeloablation should simplify HSC gene therapy.
Details
- Language :
- English
- ISSN :
- 00064971 and 15280020
- Issue :
- Preprints
- Database :
- Supplemental Index
- Journal :
- Blood
- Publication Type :
- Periodical
- Accession number :
- ejs62303464
- Full Text :
- https://doi.org/10.1182/blood.2022018252