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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...

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Detalles Bibliográficos
Autores principales: Major, Lauren, McClements, Michelle E., MacLaren, Robert E.
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