Table 1.

Current therapy strategies.

	Type	Administration	Expectation	Stage	Comment	Ref.
Drug Therapy
Prednisone	Glucocorticoid steroid	Taking orally	Reduce inflammatory response	Phase I/II study	Undesirable situations may occur due to suppressing the immune system.	[20]
MYO-029	Antibody	Injected intravenously	Neutralize myostatin protein	Phase I/II study	Myostatin inhibition resulted in a minor improvement in muscle.	[26]
Anti-myostatin antibody	Antibody	Injected intraperitoneally	Inhibition of follistatin, which is an endogenous inhibitor of myostatin	Experimental study on a murine model	Increase in muscle mass but not in functional muscle.	[28]
AMBMP	Small molecule	Injected intraperitoneally	As a Wnt agonist activates CaMKII	Experimental study on a murine model	Induction of slow oxidative genes.	[43]
Gene Therapy
pAAV-CMV-mSeAPpropmyoD76A vector	Plasmid DNA	Injected intramuscularly	Inhibition of myostatin	Experimental study on a murine model	Increase in muscle mass and absolute power	[27]
CAPN3 gene transfer via AAV vector,	Plasmid DNA	Systemic injection	Replacement of functional CAPN3 gene	Experimental study on a murine model	CAPN3 overexpression caused cardiac toxicity.	[31]
CAPN3 gene, and cardiac-specific microRNA-208a transfer via AAV	Plasmid DNA	Systemic injection	Replacement of functional CAPN3 gene and overcoming cardiac toxicity	Experimental study on a murine model	CAPN3 expression and no cardiac toxicity were achieved.	[31]
AAVrh74.tMCK.hCAPN3 vector	Plasmid DNA	Injected intravenously	Replacement of functional CAPN3 gene, overcoming off-target and toxic effects	Experimental study on a primate model	CAPN3 expression, no toxicity, and skeletal-muscle-specific vector were achieved.	[37]
rAAV-C3+miRT and rAAV-C3	Plasmid DNA	Injected intravascularly and intramuscularly	Replacement of functional CAPN3 gene and overcoming cardiac toxicity	Experimental study on a primate model	In murine models, overexpression of CAPN3 is more prone to cardiac toxicity than in primates, due to physiological differences. CAPN3 expression increased in both applications and no cardiac toxicity was observed.	[34]
Combined Therapy (Cell- and Gene-Based)
IPSCs	CRISPR-Cas9 and stem cell	Injected intramuscularly	Replacement of functional CAPN3 in myogenic progenitor and mature muscle cells expressing CAPN3	Experimental study on a murine model	CAPN3 mRNA levels were increased.	[44]