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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...
Autores principales: | , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2021
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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 |