Gene Therapy Article for MD

[Guest guest]

I know we have CMT, not muscular dystrophy, I posted for gene therapy

technique

Take care

New step reported toward muscular dystrophy treatment

RANDOLPH E. SCHMID

Mon Aug 15, 5:57 PM ET

WASHINGTON (AP) - Scientists have taken a new step in the quest for

gene therapy for muscular dystrophy.

Three years after researchers experimenting with mice discovered that

gene therapy might one day work, another team has found a way to

deliver such therapy throughout the body. University of Pittsburgh

researchers report they were able to deliver a miniature gene, similar

to the defective gene involved in muscular dystrophy, throughout the

bodies of mice, showing that systemic therapy is possible.

Their findings are reported in the online edition of Proceedings of

the National Academy of Science.

In 2002, researchers at the University of Washington reported

successful treatment of muscular dystrophy by a virus containing a

gene that produces a protein lacking in people with the disease.

However, they found that virus had to be injected directly into each

muscle.

While the new study by the Pittsburgh team shows system-wide gene

treatment can be done, the scientists weren't able simply to replace

the defective gene with a good version because the gene itself is so

large it couldn't fit inside the disabled virus used to deliver it.

Instead, the researchers used a miniature gene, similar in function to

the defective one, and showed it could be delivered to muscles

throughout the mouse, and the animals exhibited some improvement.

" While we have much farther to go until we can say gene therapy will

work in children, we have shown here a glimmer of hope by presenting

the first evidence of a successful gene therapy approach that improved

both the general health and longevity in mice with congenital muscular

dystrophy, " said Dr. Xiao Xiao, associate professor of molecular

genetics and biochemistry.

Co-author Dr. Chungping Qiao said in a statement that it's probably

unrealistic to expect complete success using the mini-gene even though

it is similar to the defective one.

Future research will seek a way to deliver correct versions of the

defective gene throughout the body.

The National Institutes of Health describes muscular dystrophy as a

group of genetic diseases characterized by progressive weakness and

degeneration of muscles that control movement.

The Mayo Clinic reports that the most common muscular dystrophies

appear to be due to a genetic deficiency of the muscle protein

dystrophin. The various types of the disease affect more than 50,000

Americans. There is no cure, but medication and therapy can slow the

disease.

Copyright © 2005 Canadian Press

Copyright © 2005 All rights reserved