CRISPR/Cas9-mediated genome editing via postnatal administration of AAV vector cures haemophilia B mice
Abstract Haemophilia B, a congenital haemorrhagic disease caused by mutations in coagulation factor IX gene (F9), is considered an appropriate target for genome editing technology. Here, we describe treatment strategies for haemophilia B mice using the clustered regularly interspaced short palindrom...
Saved in:
| Main Authors: | , , , , , , , , , , |
|---|---|
| Format: | article |
| Language: | EN |
| Published: |
Nature Portfolio
2017
|
| Subjects: | |
| Online Access: | https://doaj.org/article/b79acbb0c9004973b1526e004ab7e566 |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
Be the first to leave a comment!