Cas9/gRNA targeted excision of cystic fibrosis-causing deep-intronic splicing mutations restores normal splicing of CFTR mRNA.
Cystic Fibrosis is an autosomal recessive disorder caused by mutations in the CFTR gene. CRISPR mediated, template-dependent homology-directed gene editing has been used to correct the most common mutation, c.1521_1523delCTT / p.Phe508del (F508del) which affects ~70% of individuals, but the efficien...
Na minha lista:
| Principais autores: | , , , |
|---|---|
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Public Library of Science (PLoS)
2017-01-01
|
| coleção: | PLoS ONE |
| Acesso em linha: | https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0184009&type=printable |
| Tags: |
Sem tags, seja o primeiro a adicionar uma tag!
|