Cargando…
New CRISPR Tools to Correct Pathogenic Mutations in Usher Syndrome
Inherited retinal degenerations are a leading cause of blindness in the UK. Significant advances have been made to tackle this issue in recent years, with a pioneering FDA approved gene therapy treatment (Luxturna(®)), which targets a loss of function mutation in the RPE65 gene. However, there remai...
| Autores principales: | , , |
|---|---|
| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
MDPI
2022
|
| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9569511/ https://www.ncbi.nlm.nih.gov/pubmed/36232969 http://dx.doi.org/10.3390/ijms231911669 |