Table 4.

Table for the gene therapy.

Method	Medicinal product	Targeted	Results	Research status
Genome editing	CRISPR/Cas9	mutated gene	repaired by homology-directed repair with a repair template	genetic correction in HCM hiPSC1-3 (55–57), and correct HCM caused by a GAGT-deletion in exon 16 of the MYBPC3 gene (58).
Exonskipping	antisense oligonucleotide	exonic splicing enhancer sequences of an inframe mutated exon	preventing binding of proteins involved in the splicing process	in newborn mice abolished cardiac dysfunction and prevented the development of leftventricularhypertrophy (59).
	CRISPR/Cas9	mutated DNA sequence	Permanently cut in-frame the mutated exon.
Allele-specific silencing	specific RNA interferene molecule	mutant mRNA	knocked-down mutant mRNA	eliminate the mutant allele and delay the progression of cardiomyopathy in Myh6-targeted knock-in mice (63).
RNA trans-splicing	specific RNA interferene molecule	pre-mRNA	competes with cis-splicing	successful 5′trans-splicing in the context of HCM in cardiomyocytes and in vivo in Mybpc3-targeted knock-in mice and hiPSC (61, 62, 64).
Gene replacement	full-length cDNA	mutated DNA	functional full-length protein	in Mybpc3-targeted knock-in mice/hiPSCs, which were retrieved from an HCM patient carrying a truncating MYBPC3 muta -tion (49, 65, 66).