Table 2.

Examples of splicing-based therapeutic approaches (see text for details).

Disease	Therapeutic Approach	Target Gene	Regulated Exon
DMD	ASO	DMD	51
SMA	ASO	SMN2	7
Dystrophic epidermolysis bullosa (DEB)	ASO	COL7A1	70
FTDP-17	ASO	MAPT	10
	SMaRT	MAPT	1
Atherosclerosis	ASO	APOB	27
CF	ASO	CFTR	16
	SMaRT	CFTR	10
Ullrich congenital muscular dystrophy (UCMD)	siRNA	COL6A3	16
Growth hormone deficiency (GHD) type II	siRNA	GH1	3