Allele-specific CRISPR-Cas9 editing inactivates a single-nucleotide variant associated with collagen VI muscular dystrophy.

Mol Ther Nucleic Acids

Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry (NCNP), Tokyo, Japan.

Published: December 2024

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Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC11460449PMC
http://dx.doi.org/10.1016/j.omtn.2024.102330DOI Listing

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