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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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Autori principali: Laura Siles, Sheila Ruiz-Nogales, Arnau Navinés-Ferrer, Pilar Méndez-Vendrell, Esther Pomares
Natura: Artigo
Lingua:Inglês
Pubblicazione: Elsevier 2023-06-01
Serie:Molecular Therapy: Nucleic Acids
Soggetti:
MT: RNA/DNA Editing
CRISPR-Cas9
gene editing
human induced pluripotent stem cells
Stargardt disease
inherited retinal dystrophies
Accesso online:http://www.sciencedirect.com/science/article/pii/S2162253123000525
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