. 2022 Aug 5;13:947749. doi: 10.3389/fphys.2022.947749

TABLE 2.

Efficacy comparison of major DMD therapies.

Therapy	Object	Method	Result	References
Dystrophin gene therapy	DMD boy	The AAV2.5 vector was injected into one arm’s biceps brachii muscle	All patients had recombinant AAV genomes but no cellular immune response. AAV2. 5, the carrier is non-hazardous and well tolerated	Bowles et al. (2012)
micro-dystrophin gene therapy	GRMD dog	The rAAV2/8-micro-dystrophin expression vector was injected intravenously	The symptoms of muscular dystrophy were relieved, and there was no toxicity or adverse immune effects from rAAV administration	Le Guiner et al. (2017)
micro-dystrophin	mdx/mTR (G2) mouse	Systemic injection of rAAV6-micro- dystrophin	Mice’s muscle force was increased	Ho et al. (2018)
gene therapy	mdx/mTR (G2) mouse	Systemic injection of rAAV6-micro- dystrophin	Mice’s muscle force was increased	Ho et al. (2018)
Utrophin gene therapy	mdx	AAV-Utro intravenous injection	Histological and physiological lesions of muscular dystrophy could be avoided in mouse and large dog models	Song et al. (2019)
	mouse
	GRMD dog
Autologous cell therapy	mdx/SCID mouse	Dystrophin was transfected with a monoclonal antibody and implanted into a mouse’s muscle or artery	The elastic properties of 80% muscle fibers were restored to wild-type performance, which was more fatigue resistant	Iyer et al. (2018)
Exosome mediated cell therapy	mdx	Cardiac injection of cardiac progenitor cells (CDCs)	CDCs and their exosomes temporarily restored the expression of partial full-length dystrophin in mdx mice	Aminzadeh et al. (2018)
Exosome mediated cell therapy	mouse	Cardiac injection of cardiac progenitor cells (CDCs)		Aminzadeh et al. (2018)
Prednisone	DMD boy	Prednisone 0.75 mg/kg per day was administered on a 10 days on/10 days off basis	When compared to historical controls, prednisone 10 on/10 off has fewer side effects and extends the ambulant phase by 1 year	Straathof et al. (2009)
Pharmaco therapy	DMD boy			Straathof et al. (2009)
Prednisone Pharmaco therapy	DMD boy	two prednisone schedules (daily 0.75 mg/kg/day and weekend 10 mg/kg/wk), 12 months	Over a 12-month period, weekend prednisone dosing was as safe and effective as daily prednisone in preserving muscle strength and preventing body mass index increases in boys with DMD.	Escolar et al. (2011)
Idebenone	mdx	4 weeks to 10 months old, 200 mg/kg body weight	It improved voluntary running ability, prevented heart failure, reduced cardiac inflammation and fibrosis, and improved cardiac diastolic dysfunction	Buyse et al. (2009)
Pharmaco therapy	mouse	4 weeks to 10 months old, 200 mg/kg body weight		Buyse et al. (2009)
Laminin-111pharmaco therapy	GRMD dog	The protein Mslam-111 was injected into the craniotibial muscle	It increased muscle fiber regeneration and repair, enhanced muscle strength and reduced muscle fibrosis	Barraza-Flores et al. (2019)
sActRIIB-Fcpharmaco therapy	mdx	Soluble activin receptor sactriib FC was injected to block myostatin	Muscle mass increased	Hulmi et al. (2013)
sActRIIB-Fcpharmaco therapy	mouse		Muscle mass increased	Hulmi et al. (2013)
Resveratrol traditional Chinese medicine therapy	mdx	4 weeks of age,5 mg/kg bw/day and 15 weeks of treatment	The expression of immune cell markers CD86 and CD163 decreased, and muscle necrosis was alleviated.	Woodman et al. (2021)
Resveratrol traditional Chinese medicine therapy	mouse	4 weeks of age,5 mg/kg bw/day and 15 weeks of treatment		Woodman et al. (2021)
Voluntary wheel running	mdx	8 weeks of voluntary wheel running (3–4 days a week)	Prolonged skeletal muscle suspension time and increased dystrophin expression had a positive effect on skeletal muscle and myocardial function	Kogelman et al. (2018)
Voluntary wheel running	mouse	8 weeks of voluntary wheel running (3–4 days a week)		Kogelman et al. (2018)
Voluntary wheel running	mdx	12 weeks of voluntary low resistance wheels	Central nucleus muscle fiber decreased, skeletal muscle contraction function improved and fatigue decreased, and there was no sign that exercise was harmful	Baltgalvis et al. (2012)
Voluntary wheel running	mouse	12 weeks of voluntary low resistance wheels		Baltgalvis et al. (2012)
Voluntary wheel running	mdx	7 weeks of voluntary wheel running	The oxidative capacity and autophagy markers of skeletal muscle was increased, and were even higher than in healthy mice	Hulmi et al. (2013)
Voluntary wheel running	mouse	7 weeks of voluntary wheel running		Hulmi et al. (2013)
Treadmill training	mdx	Running at a speed of 12 m/min for 30 min, twice a week, with a 48-h or 72-h interval (12 times in total)	Impaired muscle regeneration and the failure of endogenous anti-inflammatory signals exacerbate chronic inflammation and result in more severe muscle phenotypes	Camerino et al. (2014)
Treadmill training	mouse			Camerino et al. (2014)
Treadmill training	mdx	For 6 weeks, 5 days per week, use the treadmill to gradually increase the load	There was no significant improvement in diaphragm	Morici et al. (2017)
Treadmill training	mouse		There was no significant improvement in diaphragm	Morici et al. (2017)
Treadmill training	mdx	4 m/min or 8 m/min, three times a week, 30 min each time, for a total of 6 months	Improved muscle rigidity and strength, respiratory capacity and cardiac function; decreased the cross-sectional area of adipocytes	Zelikovich et al. (2019)
Treadmill training	mouse			Zelikovich et al. (2019)
Progressive swimming	Progressive swimming	Four times a week for a total of 4 weeks, 15 min per day in the first week, 20 min per day in the second week, and 30 min per day in the third and fourth weeks	The levels of lipid peroxidation in gastrocnemius, diaphragm, hippocampus and striatum were significantly decreased	Nocetti et al. (2021)
micro-dystrophin	mdx	rAAV6:µDysH3 and rAAV6:µDys5	Both rAAV have the same dose response to prevent muscle damage caused by repeated high-intensity exercise	Rodgers et al. (2020)
exercise+	mouse	Gene therapy + repeated high-intensity exercise
gene therapy	mouse	Gene therapy + repeated high-intensity exercise
A pair of AAVs exercise+ gene therapy	mdx	A pair of AAV vectors were injected into the caudal vein to express nNOS bound dystrophin gene	It improved histopathology, increased muscle strength and prevented eccentric contraction injury and muscle strength decline caused by chronic exercise	Zhang et al. (2013)
A pair of AAVs exercise+ gene therapy	mouse
	mdx4cv
	mouse
Exercise +	mdx mouse	Running at a speed of 12 m/min for 30 min, twice a week for 4–8 weeks. Pentoxifylline (50 mg/kg/day) was injected during exercise	The Ca²⁺channel activity in mdx mice treated with exercise + pentoxifylline was similar to that in wild-type mice	Rolland et al. (2006)
pentoxifylline	mdx mouse			Rolland et al. (2006)