Drug candidate for treating spinal muscular atrophy identified
5 Nov 2009, 1358 Hrs

Add comment          Mail          Print          Write to Editor

Washington, Nov 5 Scientists have identified a chemical cousin of the common antibiotic tetracycline that might be useful in treating spinal muscular atrophy (SMA), a currently incurable disease that is the leading genetic cause of death in infants.

The finding is based on a study conducted by Adrian Krainer, Ph.D., of Cold Spring Harbor Laboratory (CSHL) and scientists from Paratek Pharmaceuticals and Rosalind Franklin University of Medicine and Science.

SMA is caused by mutations in a gene called Survival of Motor Neuron 1 (SMN1), resulting in a decrease in the levels of SMN protein in the motor neurons of the spinal cord - the cells that control muscle activity.

Without the protein, these neurons degenerate, and infants born with the mutations progressively lose the ability to move, swallow, and breathe.

There are no approved therapies for the treatment of SMA.

The new molecule boosts the levels of SMN protein in cells by fixing a mistake in a cellular processing mechanism called RNA splicing.

In the study, the scientists report this fix in both mouse models of SMA, as well as in cells isolated from SMA patients.

Unlike previously identified molecules that stimulate SMN production, the tetracycline-like compound is a unique therapeutic candidate in that it is a small molecule that specifically alters RNA splicing by directly targeting the splicing reaction.

The research appears in the journal Science Translational Medicine. (ANI)

Related Latest

New pathways to metabolise sexual assault drug found Erectile dysfunction drug 'improves heart function in kids' MJ was in denial about drug addiction, says sis Janet Designer drug billed as " cross between cocaine and ecstasy" hit online Two Afghan drug peddlers caught with heroin in Amritsar Gene therapy shows promise in muscular dystrophy battle Independent candidate accuses PPP of massive fraud in Gilgit-Baltistan polls Drug 'can shrink lung cancer tumours' Nano bubblegum to enhance drug delivery in the gut Agassi should not have hidden or lied about drug abuse: Navratilova

Related Latest

Green Park stadium's scoreboard, the only manually operated scoreboard in India Dalai Lama hopes PM will discuss Tibet issue with the U.S. leadership 3 injured in fire at Jodhpur's handloom showroom White House prepares for first state visit by Manmohan Singh Bamboo fencing on Nainital's hillside roads to prevent mishaps Indian Navy vessel on solo circumnavigation of world reaches New Zealand Fort Hood gunman couldn't wait to join Qaeda imam in paradise in the afterlife AirAsia set to become most connected non-local airline in India Clarke's ultimate dream is Australian captaincy Winning more important to Murray than money

• Green Park stadium's scoreboard, the only manually operated scoreboard in India
• Dalai Lama hopes PM will discuss Tibet issue with the U.S. leadership
• White House prepares for first state visit by Manmohan Singh
• 3 injured in fire at Jodhpur's handloom showroom
• Bamboo fencing on Nainital's hillside roads to prevent mishaps

• भारतीय क्रिकेट की मजबूत दीवार
• कंगारूओं के आगे ढेर, चीतों से कैसे मुकाबला करेंगें शेर
• Reducing greenhouse gases may not be enough to slow global warming
• बीकानेर महापौर चुनावः एक विश्लेषण
• Deepawali: The Festival Of Lights