Abstract
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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