Navigation Links
Potential Lifesaver Drug Announced

Can what is done be undone? In a part answer to that adage researchers have announced the developing //of a miracle drug to treat genetic disease- those which one is born with and which usually is not correctable.

Obviously the scientific world is too thrilled for silence. The drug named as PTC124 and been worked upon by researchers from PTC Therapeutics in New Jersey and those from University of Pennsylvania and Massachusetts , is slated to be licensed for public use by 2009.

The drug which trains the crosshairs on diseases caused by faulty genes, has been so far successful on Duchenne muscular dystrophy (DMD) and cystic fibrosis. The drug is being tested on humans now after promising results on mice and researchers hope it could eventually be used to treat sufferers of around 2,000 other types of hereditary disease.

DMD is known to afflict one in 3,500 boys and is marked by weakness and muscle wasting. Children born with cystic fibrosis suffer digestion and respiratory problems and tend to die early. Both these conditions are caused by so-called "nonsense mutations" in the child's DNA meaning that the sufferer lacks a vital protein.

Accordingly, in most genetic conditions, between 5-15 per cent of cases are caused by a defect called a “nonsense mutation”.

Genes are instruction manuals for cells to make proteins, but nonsense mutations in effect introduce a command halfway through that stops production. The kind of protein disrupted determines the nature of the disease.

In Duchenne muscular dystrophy, for example, the protein necessary for normal muscle development is not made, and the fatal wasting disease is the result. In hemophilia, it is the gene for the clotting agents factor VIII or factor IX that is disrupted.

Till now no known cure for either CF or DMD and sufferers rarely live beyond their 30s. Yet now the researchers have announced that mice suffering from a form of DMD began 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.



'"/>




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
Post Your Comments:
*Name:
*Comment:
*Email:


(Date:10/13/2017)... LUIS OBISPO, Calif. (PRWEB) , ... October 13, 2017 , ... ... Alzheimer’s or dementia. However, many long-term care insurance companies have a waiver for care ... is the 90-day elimination period, when the family pays for care, is often waived, ...
(Date:10/13/2017)... ... 13, 2017 , ... Yisrayl Hawkins, Pastor and Overseer at The House of ... popular and least understood books in the Holy Scriptures, Revelation. The Book of Revelation ... scholars for centuries. Many have tossed it off as mere rubbish, but Yisrayl Hawkins ...
(Date:10/13/2017)... ... ... On The Brink”: the Christian history of the United States and the loss of ... William Nowers. Captain Nowers and his wife, Millie, have six children, ten grandchildren, ... Navy. Following his career as a naval aviator and carrier pilot, he spent ...
(Date:10/12/2017)... ... October 12, 2017 , ... The company has developed ... consumer and regulatory authorities worldwide. From Children’s to Adults 50+, every formula has ... highest standard. , These products are also: Gluten Free, Non-GMO, Vegan, Soy ...
(Date:10/12/2017)... ... October 12, 2017 , ... Information about the technology: , ... to enable prevention of a major side effect of chemotherapy in children. Cisplatin ... patients. For cisplatin, hearing loss is FDA listed on-label as a dose limiting ...
Breaking Medicine News(10 mins):
(Date:10/12/2017)... 2017   Divoti USA will engrave and ... standard of the latest FDA requirements, which stipulates new criteria regarding ... in need of Medical ID jewelry such as Medical ID Bracelets, ... engraved in terms of the new FDA requirements . ... Divoti offers this dark mark fiber ...
(Date:10/11/2017)... , Oct. 11, 2017  Caris Life Sciences ... on fulfilling the promise of precision medicine, today announced ... joined Caris, Precision Oncology Alliance™ (POA) as its 17 ... centers, the St. Jude Crosson Cancer Institute will help ... the use of tumor profiling, making cancer treatment more ...
(Date:10/10/2017)... Oct. 10, 2017  NDS received FDA 510(k) clearance in May ... battery-powered display stand specifically designed for endoscopy environments. An innovative secondary ... a clinical solution to support the improvement of patient outcomes, procedural ... ... ...
Breaking Medicine Technology: