Clinical and Therapeutic Evaluation of the Ten Most Prevalent CRB1 Mutations

Mutations in the Crumbs homolog 1 (CRB1) gene lead to severe inherited retinal dystrophies (IRDs), accounting for nearly 80,000 cases worldwide. To date, there is no therapeutic option for patients suffering from CRB1-IRDs. Therefore, it is of great interest to evaluate gene editing strategies capab...

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Detalles Bibliográficos
Autores principales: Lopes da Costa, Bruna, Kolesnikova, Masha, Levi, Sarah R., Cabral, Thiago, Tsang, Stephen H., Maumenee, Irene H., Quinn, Peter M. J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9953187/
https://www.ncbi.nlm.nih.gov/pubmed/36830922
http://dx.doi.org/10.3390/biomedicines11020385

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9953187/
https://www.ncbi.nlm.nih.gov/pubmed/36830922
http://dx.doi.org/10.3390/biomedicines11020385

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