NeuroscienceMolecular Therapy — Nucleic AcidsPeer-reviewed
Functional benefit of CRISPR/Cas9-induced allele deletion for RYR1 dominant mutation
Mathilde Beaufils, Margaux Melka, Julie Brocard…·Isabelle Marty
This study demonstrates the functional benefit of deleting a dominant RYR1 mutation using CRISPR/Cas9 in patient-derived muscle cells.
6 citations