DRNA - Dicerna announces positive effect of RNAi technology in pre-clinical studies

Announcing new preclinical data, Dicerna Pharmaceuticals ([[DRNA]] +3.0%) said that its GalXC-Plus™ RNAi technology had the potential to deliver a ‘deep and sustained’ messenger RNA (mRNA) knockdown against specific gene targets in the Central Nervous System.Rather than targeting the proteins synthesized by genes, Ribonucleic acid interference, or RNAi silences the disease-causing genes themselves by the targeted destruction of the messenger RNA formed from the genes.Data from a preclinical mouse study have indicated that a single dose of an unconjugated GalXC-Plus molecule targeting the ALDH2 gene led to a dose-dependent reduction of up to 92% knockdown in target mRNA across the CNS.The effect was sustained through the trial conclusion at 28 days, the company said, adding that a 90% target mRNA silencing occurred after 28 days in non-human primate (“NHP”) studies following a single dose.There were no adverse observations for any of the cohorts in the trials. ALDH2 gene is a

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Dicerna announces positive effect of RNAi technology in pre-clinical studies