Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing
Cystic fibrosis is caused by mutations in the CFTR chloride channel. Here, the authors develop a gene therapy approach using the programmable nuclease AsCas12a to correct a splicing mutation in CFTR, and show efficient repair of the mutation and recovery of CFTR function in patient-derived organoids...
Saved in:
| Main Authors: | , , , , , , , , |
|---|---|
| Format: | article |
| Language: | EN |
| Published: |
Nature Portfolio
2019
|
| Subjects: | |
| Online Access: | https://doaj.org/article/d7c2cba4da6b42e3a01044d6646c73ee |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|