Targeted corrective gene conversion (TCGC): Molecular systems for improved correction of gene mutations in muscle

Targeted corrective gene conversion (TCGC) holds much promise as a future therapy for many hereditary diseases in humans, but there still remain significant impediments to effective mutation correction and it is clear that significant work remains to improve TCGC to levels where it can be considered for translation to the clinical setting. Nevertheless, mutation correction frequencies varying between 0.0001% and 40% have been reported using chimeraplasty, oligoplasty, triplex-forming oligonucleotides, and small corrective PCR amplicons. W