Gene Therapy

Study results from University of Massachusetts in the area of gene therapy published

Published in Gene Therapy Weekly, July 30th, 2009

"RNA interference (RNAi) mediates sequence-specific gene silencing, which can be harnessed to silencing disease-causing genes for therapy. Particularly suitable diseases are those caused by dominant, gain-of-function type of gene mutations," researchers in the United States report.

"In these diseases, the mutant gene generates a mutant protein or RNA product, which possesses toxic properties that harm cells. By silencing the mutant gene, the toxicity can be lessened because the amount of the toxic product is lowered in cells. In this report, we tested RNAi therapy in a mouse model for amyotrophic lateral sclerosis (ALS), which causes motor neuron degeneration,...

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Source: Gene Therapy Weekly (2009-07-30)

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