SCIENTISTS say they are closer to helping people with hereditary diseases after restoring almost normal heart function to members of a family who suffer from a dangerous cardiac disease.
Medical researchers cured the family of dilated cardiomyopathy (DCM), a disease that causes heart rhythm disorders, using technology to identify mutated genes which they targeted with drugs.
The research, led by a team from Sydney's Victor Chang Cardiac Institute and St Vincent's Hospital, has been published in The Journal of the American College of Cardiology.
The researchers began by screening the 42 members of the family who had a history of DCM.
They were then given drugs which genetic sequencing led researchers to believe would target and "specifically reverse the consequences of the gene defect", Victor Chang Cardiac Institute Professor Jamie Vandenberg said.
Drugs can slow the progress of DCM, but when it inevitably reaches what researchers call "end stage" the only option for sufferers is a heart transplant, Prof Vandenberg said.
Thanks to new research and the decreasing costs and difficulties of gene sequencing, it is now possible to identify mutated genes and prescribe medication that targets the cause of hereditary diseases.
In the case of inherited DCM, it's as close to a cure as is yet available, Prof Vandenberg says.
"Some of these patients went from having a life expectancy of six to 12 months to now having almost normal heart function again," he told AAP.
"If you do understand what the underlying gene defect is, there's a possibility that you can identify a specific drug that will work and will work very effectively.
About 1 in 2000 Australians are affected by DCM, which accounts for about 10 per cent of heart failures.
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