Modeling PRPF31 retinitis pigmentosa using retinal pigment epithelium and organoids combined with gene augmentation rescue

Mutations in the ubiquitously expressed pre-mRNA processing factor (PRPF) 31 gene, one of the most common causes of dominant form of Retinitis Pigmentosa (RP), lead to a retina-specific phenotype. It is uncertain which retinal cell types are affected and animal models do not clearly present the RP p...

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Detalles Bibliográficos
Autores principales: Rodrigues, Amélie, Slembrouck-Brec, Amélie, Nanteau, Céline, Terray, Angélique, Tymoshenko, Yelyzaveta, Zagar, Yvrick, Reichman, Sacha, Xi, Zhouhuan, Sahel, José-Alain, Fouquet, Stéphane, Orieux, Gael, Nandrot, Emeline F., Byrne, Leah C., Audo, Isabelle, Roger, Jérôme E., Goureau, Olivier
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9381579/
https://www.ncbi.nlm.nih.gov/pubmed/35974011
http://dx.doi.org/10.1038/s41536-022-00235-6

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9381579/
https://www.ncbi.nlm.nih.gov/pubmed/35974011
http://dx.doi.org/10.1038/s41536-022-00235-6

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