Table 1. Muscular dystrophies and myostatin inhibition.

Disease	Mode of inheritance	Gene locus	Gene products	Myostatin blockage	Ref [Method of myostatin inhibition]
Duchenne	XR	Xp21	Dystrophin	Effective in mdx mouse	Bogdanovich et al., (8) [1] Wagner et al., (21) [2] Bogdanovich et al., (9) [3] Nakatani et al., (17) [4]
LGMD1C (CAV3)	AD	3p25	Caveolin-3	Effective in model mouse	Ohsawa et al., (10) [5]
LGMD2A (CAPN3)	AR	15q15	Calpain-3	Gene therapy is effective	Bartoli et al., (11) [6]
LGMD2D (SGCA)	AR	17q12-21	α-sarcoglycan	Gene therapy is not effective	Bartoli et al., (11) [6]
LGMD2F (SGCD)	AR	5q33-34	δ-sarcoglycan	Early therapy is effectiveTreat early	Parsons et al., (22) [1, 2]
MDC1A(LAMA2)	AR	6q22	Lamininα-2	Not effective in dy mouse Severe fat loss	Li et al., (12) [2]

The effects of myostatin blockade on various types of muscular dystrophy are summarized. Myostatin inhibition is applicable as a therapy for multiple types of muscular dystrophy. Transgenic approaches, systemic injection and gene therapy have been tried. Myostatin blockade by myostatin antibodies, modified myostatin propeptide or follistatin-derived peptides is effective for ameliorating the pathophysiology in mdx mice. Myostatin inhibition is also effective for ameliorating several types of limb-girdle-type muscular dystrophy caused by mutations of caveolin-3 or calpain-3. Effective therapy would be possible by early treatment. It is noteworthy that elimination of myostatin does not improve the phenotypes of laminin-α2-deficient model mice. Method of myostatin inhibition is shown as brackets.

^[1]myostatin antibody treatment;

^[2]crossing with myostatin K/O mice;

^[3]myostatin propeptide treatment;

^[4]crossing with mutated follistatin Tg mice;

^[5]crossing with myostatin propeptide Tg mice;

^[6]AAV-mediated mutated myostatin propeptide expression. References are shown with parentheses.