Centro Cardiologico Monzino IRCCS
Published September 18, 2024 | Version v1
Dataset Restricted

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."

Description

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.

Files

Restricted

The record is publicly accessible, but files are restricted. Log in to check if you have access.

Additional details

Funding

European Commission
ERA-NET on Cardiovascular Diseases (JTC 2018) “Transnational Cardiovascular Research Projects driven by Early Career Scientists" JTC2018-046
Telethon Foundation
Clinical Projects 2019 GUP19012
Ministero della Salute
Ricerca Finalizzata GR-2021-12375403
Ministero della Salute
Ricerca Corrente 2764167