(MENAFN - ProactiveInvestors - Australia) Benitec Biopharma (ASX:BLT) research on muscular dystrophy shows encouraging in-vitro results via gene silencing.
The research targets the silencing of a gene (PABPN1), the mutant form of which causes the disease.
Data collected supports the development of a vector for combined silencing of the mutant gene by ddRNAi and replacement with a normal gene.
The data confirms that the delivery of a unique multi-shRNA cassette, Pabparna, to cells expressing the target gene is highly effective at silencing the target gene in those cells, reducing the expression levels to around 10%.
If achieved in vivo, this level of silencing by the clinical candidate is likely to result in significant improvement of the muscle disease, currently untreatable.
The researchers expect the optimal therapeutic to be based on both suppression of the target mutant gene and the replacement with a healthy gene.
Successful results at the stage of in vivo testing will be crucial to committing to advance the program into the clinic.
The program is being collaborated with Professor George Dickson from the Royal Holloway, University of London, and Dr Capucine Trollet from the Institut de Myologie.