PITTSBURGH, Aug. 15 Researchers from the University of Pittsburgh report the first study to achieve success with gene therapy for the treatment of congenital muscular dystrophy (CMD) in mice, demonstrating that the formidable scientific challenges that have cast doubt on gene therapy ever being feasible for children with muscular dystrophy can be overcome. Moreover, their results, published in this week's online edition of the Proceedings of the National Academy of Sciences (PNAS), indicate that a single treatment can have expansive reach to muscles throughout the body and significantly increase survival.

CMD is a group of some 20 inherited muscular dystrophies characterized by progressive and severe muscle wasting and weakness first noticed soon after birth. No effective treatments exist and children usually die quite young.

Despite gene therapy being among the most vigorously studied approaches for muscular dystrophy, it has been beset with uniquely difficult hurdles. The genes to replace those that are defective in CMD are larger than most, so it has not been possible to apply the same methods successfully used for delivering other types of genes. And because CMD affects all muscles, an organ that accounts for 40 percent of body weight, gene therapy can only have real therapeutic benefit if it is able to reverse genetic defects in every cell of the body's 600 muscle groups.

By using a miniature gene, similar in function to the one defective in CMD, and applying a newly developed method for "systemic" gene delivery, the Pitt researchers have shown that gene therapy for muscular dystrophy is both feasible and effective in a mouse model of especially profound disease. Using this approach, the team, led by Xiao Xiao, Ph.D., associate professor of orthopaedic surgery and molecular genetics and biochemistry at the University of Pittsburgh School of Medicine, report that treated mice had physiological improvements in the muscles of the heart, di
'"/>


15-Aug-2005

Page: 1 2 3
Related biology news :

1. Is nutrient loading a smaller problem than we think?
2. Consumption of small amounts of dark chocolate associated with reduction in blood pressure
3. A small leak will sink a great ship
4. Fever after smallpox vaccination tied to individual genetic variations
5. University of Liverpool launches 10 million small animal teaching hospital
6. Study shows big power of small RNAs, not just proteins, in halting cancer
7. New algorithms classify outcomes of nonsmall cell lung cancer patients
8. Cisplatin is more effective than carboplatin for treating nonsmall cell lung cancer
9. Innovative smallpox vaccine research study to be conducted at Case Medical Center
10. Enzyme delivered in smaller package protects cells from radiation damage
11. Why small dogs are small