Figure 2.

Potential therapeutic approaches to correcting or ameliorating the effects of MYBPC3 haploinsufficiency in HCM at different mechanistic stages. Such approaches could include: correction of the mutant allele through gene editing technology, restoring full expression of wild-type MYBPC3; stop codon readthrough for truncating mutations, resulting in expression of a mutant but functional full-length protein; increasing expression of the wild-type protein by modulating its turnover; and targeting of downstream maladaptive mechanisms without affecting MYBPC3 protein expression.