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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Main Authors: Laura Siles (Author), Sheila Ruiz-Nogales (Author), Arnau Navinés-Ferrer (Author), Pilar Méndez-Vendrell (Author), Esther Pomares (Author)
Format: Book
Published: Elsevier, 2023-06-01T00:00:00Z.
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3rd Floor Main Library

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Call Number: A1234.567
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