New drug may cure genetic disorders

[Al-khiyal]

LONDON April 23 (UPI) -- A new pill that can correct faulty genes and cure some genetic illnesses, called PTC124, should be out in Britain within the next three years.

The new pill reportedly promises to be able to treat or help thousands of conditions, some of which are incurable, like muscular dystrophy and cystic fibrosis, The Times of London reported Monday.

The drug is in its final phase of clinical trials and could be licensed by 2009.

"There are literally thousands of genetic diseases that could benefit from this approach," said Lee Sweeney of the University of Pennsylvania, who is leading the research. "What's unique about this drug is it doesn't just target one mutation that causes disease, but a whole class of mutations."

PTC124 reportedly targets one particular type of mutation that causes many different symptoms depending on which gene is mutated. That makes it potentially useful against many inherited disorders.

New drug may cure genetic disorders

[amalgamate]

That sounds like fantacy. genetic mutations come from an abnormal change in DNA codon paterns, which in turn causes abnormal phisological effects to the chormosome which in turn produce abnormal characteristics in a person.

you're telling me (or this article states) that a pill can get down to the root cause of gene mutation and rearange the DNA condons that got dislocated and put them back in 'normal' sequence?


Tell me more, i'm all ears...

[amalgamate]

well, let's take a close look-see at this PTC 124:

PTC-124, a 1,2,4-oxadiazole compound, is in development by PTC Therapeutics Inc as an orally active small molecule that can override nonsense stop translation signals to produce full-length proteins. ...In vivo investigations revealed that PTC-124 was effective in restoring the production of full-length protein in animal models of cystic fibrosis (CF) and Duchenne muscular dystrophy (DMD).

Drug Evaluation


ah ha, i c said the blind man.

well as a matter of fact then, some mutation could arise from another level other than the genes- the protein formation level. Even when a gene is good, an error can occur when translating it into a protein (proteins come from gene translation). If the translation stops to early, the protein that is formed is actually incomplete, or in other words, mutated. mutated proteins are inactive. they cannot do the job they're supposed to do and are harmful to the body.

cystic fibrosis and muscular dystrophy i'm assuming are a cause of abnormal protein synthesis. so if i'm understanding this correctly, the PTC 124 can override this unnecessary stop and make the translation go to completion thereby producing an active, normal protein.




CooooooL