Single AAV-mediated mutation replacement genome editing in limited number of photoreceptors restores vision in mice
Replacing mutant genes with wildtype copies using adeno-associated virus (AAV) has been explored for the treatment of inherited retinopathies, but the low cargo limit restricts its use. Here the authors describe a single AAV platform that allows local replacement of a mutated sequence with its wildt...
Saved in:
| Main Authors: | , , , , |
|---|---|
| Format: | article |
| Language: | EN |
| Published: |
Nature Portfolio
2020
|
| Subjects: | |
| Online Access: | https://doaj.org/article/cf43721ae08c4135a4828716cd4ce36c |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|