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Allele-specific editing ameliorates dominant retinitis pigmentosa in a transgenic mouse model

Retinitis pigmentosa (RP) is a group of progressive retinal degenerations of mostly monogenic inheritance, which cause blindness in about 1:3,500 individuals worldwide. Heterozygous variants in the rhodopsin (RHO) gene are the most common cause of autosomal dominant RP (adRP). Among these, missense...

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Detalles Bibliográficos
Autores principales: Patrizi, Clarissa, Llado, Manel, Benati, Daniela, Iodice, Carolina, Marrocco, Elena, Guarascio, Rosellina, Surace, Enrico M., Cheetham, Michael E., Auricchio, Alberto, Recchia, Alessandra
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7896132/
https://www.ncbi.nlm.nih.gov/pubmed/33508235
http://dx.doi.org/10.1016/j.ajhg.2021.01.006