( growing new muscles - after being treat...)Potential Lifesaver Drug Announced,Health

growing new muscles - after being treated with a the drug.

Says lead researcher Lee Sweeney, who is leading the research: " There are literally thousands of genetic diseases that could benefit from this approach. “What’s unique about this drug is it doesn’t just target one mutation that causes disease, but a whole class of mutations.”

So how does the drug work? PTC124 works by binding to a part of the cell called the ribosome, which translates genetic code into protein, and allowing it to ignore nonsense mutations. The gene can be read straight through and a normal protein is produced. According to the report published in the journal Nature, the drug is already being tried out on 100 humans.

At the same time PTC124 is just one of a new generation of treatments which are bringing hope to sufferers of the genetic disease cystic fibrosis, such as gene therapy. Professor David Porteous at the Edinburgh University is pioneering this. Porteous hailed the announcement of PTC124 as "very exciting". "It will be very important to see if this exciting new drug can be used safely and effectively in the clinic," he was quoted. "But PTC124 or similar products could only ever be a small part of the solution as the vast majority of CF patients have a different type of mutation from the rare nonsense mutation which is specifically targeted by this class of compounds. Gene therapy is the obvious best hope."

Gene therapy involves manufacturing a fully working version of the cystic fibrosis gene and introducing it into the cells lining the lung where it is needed.



'"/>




Page: 1 2
Related medicine news :

1. Potential New Cancer Gene Identified
2. Lack of Smell And Its Potential Risks
3. New Blood Thinner Pill - First Potential Alternative to Warfarin in 50 years
4. The Potential Of Cadaver Transplantation Programme In India
5. Another Potential Of Stem Cells Discovered: Used For Growing Cartilage
6. Yoga Guru Ramdev Invited to Tap Ayurveda Potential by Jharkhand Govt
7. A Protein In Yoghurt Has The Potential To Fight E. coli
8. Gold Therapy- A Potential Treatment For Autoimmune Diseases
9. Whirlpool Bath Tubs Contain Potentially Lethal Bacterial Strains
10. Potential Benefits Of New Nanotechnology
11. Hormone Can Foretell Presence Of Potentially Fatal Pulmonary Hypertension