Life Sciences

New data from P.H.J. Vanbilsen et al illuminate research in life sciences

Published in Gene Therapy Weekly, October 16th, 2008

According to recent research published in the journal Human Gene Therapy, "Huntington's disease (HD) is a dominantly inherited neurodegenerative disorder caused by the expression of mutant huntingtin protein (Htt). Suppression of Htt expression, using RNA interference, might be an effective therapy."

"However, if reduction of wild-type protein is not well tolerated in the brain, it may be necessary to suppress just the product of the mutant allele. We present a small interfering RNA (siRNA) that selectively reduces the endogenous mRNA for a heterozygous HD donor's pathogenic allele by approximately 80% by specifically targeting a single-nucleotide polymorphism...

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Source: Gene Therapy Weekly (2008-10-16)

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