Cargando…
Allele-specific gene editing to rescue dominant CRX-associated LCA7 phenotypes in a retinal organoid model
Cases of Leber congenital amaurosis caused by mutations in CRX (LCA7) exhibit an early form of the disease and show signs of significant photoreceptor dysfunction and eventual loss. To establish a translational in vitro model system to study gene-editing-based therapies, we generated LCA7 retinal or...
| Autores principales: | , , , , |
|---|---|
| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Elsevier
2021
|
| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8580887/ https://www.ncbi.nlm.nih.gov/pubmed/34653402 http://dx.doi.org/10.1016/j.stemcr.2021.09.007 |