Correction of exon 2, exon 2–9 and exons 8–9 duplications in DMD patient myogenic cells by a single CRISPR/Cas9 system
Author:
Funder
AFM-Téléthon
Publisher
Springer Science and Business Media LLC
Link
https://www.nature.com/articles/s41598-024-70075-5.pdf
Reference49 articles.
1. Hoffman, E. P., Brown, R. H. Jr. & Kunkel, L. M. Dystrophin: The protein product of the Duchenne muscular dystrophy locus. Cell 51, 919–928. https://doi.org/10.1016/0092-8674(87)90579-4 (1987).
2. Mercuri, E., Bonnemann, C. G. & Muntoni, F. Muscular dystrophies. Lancet 394, 2025–2038. https://doi.org/10.1016/S0140-6736(19)32910-1 (2019).
3. Monaco, A. P. et al. Isolation of candidate cDNAs for portions of the Duchenne muscular dystrophy gene. Nature 323, 646–650. https://doi.org/10.1038/323646a0 (1986).
4. Beggs, A. H., Koenig, M., Boyce, F. M. & Kunkel, L. M. Detection of 98% of DMD/BMD gene deletions by polymerase chain reaction. Hum. Genet. 86, 45–48. https://doi.org/10.1007/BF00205170 (1990).
5. Babbs, A. et al. From diagnosis to therapy in Duchenne muscular dystrophy. Biochem. Soc. Trans. 48, 813–821. https://doi.org/10.1042/BST20190282 (2020).
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