OSAKA -- A mouse with a severe type of muscular dystrophy showed muscle recovery and improvements in its symptoms after being implanted with skeletal muscle stem cells generated from human induced pluripotent stem (iPS) cells, a Japanese research group announced.
Hidetoshi Sakurai, associate professor of regenerative medicine at Kyoto University's Center for iPS Cell Research and Application, and other group members announced their finding in the electronic edition of the American science journal Stem Cell Reports on July 3, Japan time.
Duchenne muscular dystrophy (DMD) is a genetic abnormality of the X chromosome, which prevents the production of proteins called dystrophin, and leads to progressive deterioration of muscle fibers. According to the research group, DMD occurs exclusively in males -- one in 3,500 men -- and it is estimated that Japan has around 5,000 patients.
The group generated human iPS cells to differentiate into skeletal muscle stem cells with high regenerative capability, and found at 12 weeks of differentiation, or in the later stage, that a certain type of skeletal muscle stem cell adjusts well after transplant and induces the most muscle recovery. When this type of stem cell was transplanted to a mouse's right calf, it showed a slight recovery of the muscle.
Though the group's finding is one step closer to the realization of a transplant treatment for DMD patients, transplants can only be made in sections at this point, and in the meantime, it is expected that treatment can only be provided for body parts including fingers, arms and respiratory muscles.
Associate professor Sakurai commented, "We hope to make practical use (of our finding) by combining it with other treatment methods."
(Japanese original by Koki Matsumoto, Osaka Science & Environment News Department)