Exon Skipping Through Chimeric Antisense U1 snRNAs to Correct Retinitis Pigmentosa GTPase-Regulator (RPGR) Splice Defect

Inherited retinal dystrophies are caused by mutations in more than 250 genes, each of them carrying several types of mutations that can lead to different clinical phenotypes. Mutations in Retinitis Pigmentosa GTPase-Regulator (RPGR) cause X-linked Retinitis pigmentosa (RP). A nucleotide substitution...

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Detalles Bibliográficos
Autores principales: Covello, Giuseppina, Ibrahim, Gehan H., Bacchi, Niccolò, Casarosa, Simona, Denti, Michela Alessandra
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc., publishers 2022
Materias:
Original Papers
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9416563/
https://www.ncbi.nlm.nih.gov/pubmed/35166581
http://dx.doi.org/10.1089/nat.2021.0053

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9416563/
https://www.ncbi.nlm.nih.gov/pubmed/35166581
http://dx.doi.org/10.1089/nat.2021.0053

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