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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Autores principales:	Laura Siles, Sheila Ruiz-Nogales, Arnau Navinés-Ferrer, Pilar Méndez-Vendrell, Esther Pomares
Formato:	Artigo
Lenguaje:	Inglês
Publicado:	Elsevier 2023-06-01
Colección:	Molecular Therapy: Nucleic Acids
Materias:	MT: RNA/DNA Editing CRISPR-Cas9 gene editing human induced pluripotent stem cells Stargardt disease inherited retinal dystrophies
Acceso en línea:	http://www.sciencedirect.com/science/article/pii/S2162253123000525
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