October 17, 2007
UMass Medical uses RNAi treatment for Huntington's disease
By Cory Hopkins
Researchers from UMass Medical School in Worcester, Massachusetts General Hospital in Boston and Alnylam Pharmaceuticals Inc. of Cambridge have discovered an RNAi-based treatment that has proven effective at treating Huntington's disease in animals.
The results of a recent pre-clinical study on RNAi therapy for Huntington's disease will be published today in the journal Proceedings of the National Academy of Sciences.
Researchers discovered that a single injection of small interfering RNAs, molecules that mediate traditional RNAi, targeting the huntingtin gene responsible for causing Huntington's disease resulted in improved symptoms of the disease in animals, including markedly better motor performance, according to Alnylam. The therapy was found to be effective up to one week on a single injection, with few side effects.
RNAi, discovered at UMass Medical in the late 1990s by Craig Mello, who was awarded a Nobel Prize in medicine for the discovery in 2006, works by bonding to specific "target" genes in a cell, in turn switching those genes on or off and preventing them from producing proteins that cause disease or other ailments.
The results of a recent pre-clinical study on RNAi therapy for Huntington's disease will be published today in the journal Proceedings of the National Academy of Sciences.
Researchers discovered that a single injection of small interfering RNAs, molecules that mediate traditional RNAi, targeting the huntingtin gene responsible for causing Huntington's disease resulted in improved symptoms of the disease in animals, including markedly better motor performance, according to Alnylam. The therapy was found to be effective up to one week on a single injection, with few side effects.
RNAi, discovered at UMass Medical in the late 1990s by Craig Mello, who was awarded a Nobel Prize in medicine for the discovery in 2006, works by bonding to specific "target" genes in a cell, in turn switching those genes on or off and preventing them from producing proteins that cause disease or other ailments.
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