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Benitec Announces Positive In Vitro Data from Muscular Dystrophy Program

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Benitec Biopharma this week announced that it has obtained positive in vitro data from its early-stage program in oculopharyngeal muscular dystrophy.

As a result, Benitec said it and academic collaborators will begin developing a vector that will use expressed RNAi to silence the mutant form of the PABPN1 gene, which causes the condition, and deliver a normal version of the gene.

Data from the cell-culture studies indicate that Benitec's multi-shRNA cassette can silence target gene expression to around 10 percent. The company is preparing to replicate this level of inhibition in animal models this year.

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