Doctor claims DNA treatment
for Duchenne muscular dystrophy
Yomiuri Shimbun
A professor of Kobe University's School of Medicine on Wednesday said that he
had successfully treated Duchenne muscular dystrophy (DMD) through the use
of DNA, which encourages production of a protein necessary to move
muscle. After the new treatment was given to a 10-year-old boy suffering
from DMD, the professor said he found messenger ribonucleic acid (mRNA), which
is required to produce the protein dystrophin in the boy's
body. Matsuo added that it was certain that dystrophin would also be
produced in his body. The new therapy has the potential to delay the
progression of the disease, Matsuo said. DMD is a genetic disease that
renders victims incapable of moving their muscles. It is found only among
boys, mostly between the ages of 4 and 5 years old. The disease occurs in one
in 3,500 boys. To produce dystrophin, mRNA is required. But because of a
lack of part of mRNA's base sequences, DMD patients cannot produce the
protein. Matsuo drip-fed synthetic DNA that adjusts the deficient
mRNA's base sequences, once a week, four times between Oct. 29 and Nov.
19. The doctor discovered complete mRNA on Nov. 5. He said he examined whether
dystrophyn was produced in the boy's muscle on Wednesday last week and the
result of the test would be available within this month. But according to
Matsuo, the therapy was only to convert DMD into milderBecker's muscular
dystrophy and is not considered as a breakthrough treatment so far.