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- W3089653236 abstract "Gene editing in cells has revolutionized the paradigm of modern cell biology. The ease of use of the CRISPR/Cas9 system for gene manipulation has led to rapid and wide adoption across many fields, including biological research, biotechnology and medicine. This technology allows gene modification by producing a specific double stranded cut in the DNA and the repair by homology-independent targeted insertion (HITI) of DNA. We used CRISPR/CAS9 to modify specific mutations in-vitro in a muscular disorder to show its potential use as a therapeutic tool. We used cells from patients diagnosed with Nemaline myopathy with homozygous loss of exon 55 in the NEB gene due to a founder mutation. In addition, we corrected the exon 55 deletion in myotube cells from an animal model of Nemaline Myopathy (NEB-55). Taking advantage of DNA repair mechanism, we use the HITI to reintroduce exon 55 and re-establish its expression at the RNA and protein level. This study provides another piece of evidence that CRISPR/CAS9 based genome editing is a viable therapeutic avenue that can be pursued for treatment of neuromuscular disorders." @default.
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- W3089653236 date "2020-10-01" @default.
- W3089653236 modified "2023-09-25" @default.
- W3089653236 title "HIGHLIGHTS ACROSS MYOLOGY" @default.
- W3089653236 doi "https://doi.org/10.1016/j.nmd.2020.08.351" @default.
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