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July 02, 2004
RNAi As A Huntington's Treatment
I've got wonderful readers on this website! Thanks to the reader who kindly posted the link to the article mentioned yesterday on RNAi.
In a nutshell, this article talks about research using RNAi as the way to 'cure' Huntington's Disease. This lab is in the early stages of Huntington's/RNAi research. They are working on proving the concept that their RNAi method will work on the huntingtin protein. Then they will start testing various methods of delivering their treatment - one of which includes surgery.
They figure they've got at least three years of animal testing so this particular lab is still many years away from providing a cure. This is all very exciting stuff!
These disorders, of which Huntington’s disease is one, are caused by the expansion of triplet repeat DNA sequences, Tiscornia said. Specifically, Huntington’s disease is caused by the expansion of a CAG repeat in exon 1 of the gene huntingtin.
“The traditional approach in gene therapy is to find a disease caused by a protein being mutated, and then providing that protein through a lentiviral vector — finding something that is not there and then giving it back,” Tiscornia explained. “In these diseases, the problem is the opposite … so RNAi was, on paper, perfectly suited for this.”
Tiscornia’s project, the first of its kind in the Verma lab, is looking to establish a proof of concept that siRNAs targeting huntingtin and delivered using lentiviral vectors can slow or halt the progression of Huntington’s disease in a transgenic mouse model of the disease.
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“We’re testing the limits of the technology,” he added. “Once we know what those limits are, then we can think about what to do later.”
Posted by Dave at July 2, 2004 11:16 PM
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