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...

Descripción completa

Detalles Bibliográficos
Autores principales: Chirco, Kathleen R., Chew, Shereen, Moore, Anthony T., Duncan, Jacque L., Lamba, Deepak A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2021
Materias:
Article
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

Internet

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

Ejemplares similares