Therapeutic base editing in the adult liver
- Resource Type
- Authors
- Piter J. Bosma; Coen C. Paulusma
- Source
- Nature Reviews Gastroenterology and Hepatology, 18(9), 597-598. Nature Publishing Group
- Subject
- Hepatology
Genome editing
business.industry
Intervention (counseling)
Genetic enhancement
Gastroenterology
MEDLINE
Medicine
Adult liver
Safe delivery
business
Bioinformatics
- Language
- ISSN
- 1759-5053
1759-5045
Gene editing to correct inherited liver disorders has promise for future therapeutic intervention, but lack of effective and safe delivery of the gene-editing machinery to hepatocytes complicates its clinical application. Two studies now report efficient delivery to the liver of non-human primates, providing proof of concept for novel treatment of inherited hypercholesterolaemia.