Author

Eleonora Roschi

Wellcome Sanger Institute

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Genomics· 1

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Approach:experimental assay· 1 Organism:human· 1

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Arturo Flores-Tufiño· 1 shared Bernd WissingeriD· 1 shared Katarína ŠtingliD· 1 shared Laura KuehleweiniD· 1 shared Pietro De AngeliiD· 1 shared Salome Spaag· 1 shared Stefanida ShliagaiD· 1 shared Susanne KohliD· 1 shared

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1 shared paperGenomics

Arturo Flores-Tufiño

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1 paper

Recent·Most cited

GenomicsbioRxivPreprintMar 27, 2024

EDSpliCE, a CRISPR-Cas9 gene editing platform to rescue splicing, effectively corrects inherited retinal dystrophy-associated splicing defects

Pietro De Angeli, Stefanida Shliaga, Arturo Flores-Tufiño…·Susanne Kohl

EDSpliCE corrects splicing defects in inherited retinal dystrophy using CRISPR-Cas9 with a single gRNA and exonuclease, showing effective splicing rescue in minigene assays and patient-derived fibroblasts.

DOI 10.1101/2024.03.27.587013

humanexperimental assay

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