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High-Efficiency CRISPR/Cas9-Mediated Correction of a Homozygous Mutation in Achromatopsia-Patient-Derived iPSCs

Achromatopsia is an autosomal recessive disorder, in which cone photoreceptors undergo progressive degeneration, causing color blindness and poor visual acuity, among other significant eye affectations. It belongs to a group of inherited retinal dystrophies that currently have no treatment. Although...

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Detalles Bibliográficos
Autores principales: Siles, Laura, Gaudó, Paula, Pomares, Esther
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9964936/
https://www.ncbi.nlm.nih.gov/pubmed/36835061
http://dx.doi.org/10.3390/ijms24043655

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