Itoero Posted December 21, 2016 Share Posted December 21, 2016 (edited) There is currently a clinical trial taking place in Europe and Canada in which they test the ASO strategy in patients with Huntingtons disease. When the genetic sequence of a particular gene is known to be causative of a particular disease, it is possible to synthesize a strand of nucleic acid (DNA, RNA or a chemical analogue) that will bind to the messenger RNA (mRNA) produced by that gene and inactivate it, effectively turning that gene "off". (gene silencing) This synthesized nucleic acid is termed an "anti-sense" oligonucleotide.(ASO) Antisense oligonucleotides have been researched as potential drugs for diseases such as cancers, diabetes, Amyotrophic lateral sclerosis (ALS), Duchene muscular dystrophy and diseases such as asthma, arthritis and pouchitis with an inflammatory component. https://en.wikipedia.org/wiki/Antisense_therapy The outcome of the trial in 1 - 2 years could affect the development of other therapies. It's very hopeful Edited December 21, 2016 by Itoero Link to comment Share on other sites More sharing options...
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