Dataset related to the article "Complete phenotype rescue through the restoration of full-length dystrophin using CRISPR/Cas9 genome editing in Duchenne muscular dystrophy patient-derived iPSCs carrying the deletion of two exons."

This record contains raw data related to the article Dataset related to the article “Complete phenotype rescue through the restoration of full-length dystrophin using CRISPR/Cas9 genome editing in Duchenne muscular dystrophy patient-derived iPSCs carrying the deletion of two exons".

Here we describe for the first time the restoration of the full-length dystrophin protein, by CRISPR/Cas9, in an iPSC derived from a Duchenne patient carrying the deletion of two exons, allowing the recovery of the cardiac pathological phenotypes and mechanisms, as assessed from the transcriptional, structural, and functional point of view.