Mirtron-mediated RNA knockdown/replacement therapy for the treatment of dominant retinitis pigmentosa

Rhodopsin (RHO) gene mutations are a common cause of autosomal dominant retinitis pigmentosa (ADRP). The need to suppress toxic protein expression together with mutational heterogeneity pose challenges for treatment development. Mirtrons are atypical RNA interference effectors that are spliced from...

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Detalles Bibliográficos
Autores principales: Orlans, Harry O., McClements, Michelle E., Barnard, Alun R., Martinez-Fernandez de la Camara, Cristina, MacLaren, Robert E.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8368061/
https://www.ncbi.nlm.nih.gov/pubmed/34400638
http://dx.doi.org/10.1038/s41467-021-25204-3

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8368061/
https://www.ncbi.nlm.nih.gov/pubmed/34400638
http://dx.doi.org/10.1038/s41467-021-25204-3

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