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Efficient correction of ABCA4 variants by CRISPR-Cas9 in hiPSCs derived from Stargardt disease patients

Inherited retinal dystrophies comprise a broad group of genetic eye diseases without effective treatment. Among them, Stargardt disease is the second most prevalent pathology. This pathology triggers progressive retinal degeneration and vision loss in children and adults. In recent years, the evolut...

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Detalles Bibliográficos
Autores principales:	Siles, Laura, Ruiz-Nogales, Sheila, Navinés-Ferrer, Arnau, Méndez-Vendrell, Pilar, Pomares, Esther
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2023
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10034418/ https://www.ncbi.nlm.nih.gov/pubmed/36969552 http://dx.doi.org/10.1016/j.omtn.2023.02.032

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