BioCentury
ARTICLE | Tools & Techniques

RNA is for activation

February 6, 2014 8:00 AM UTC

Although siRNA-based therapeutics have recently been clinically validated as a strategy to knock down gene expression, a lesser-known function for short double-stranded RNAs is their ability to turn on the expression of target genes. Now, an academic team sponsored by MiNA Therapeutics Ltd. has found a way to attack liver cancer in rats by using a short activating RNA to upregulate expression of a tumor suppressor,1 and the company plans to advance the program to the clinic this year.

siRNAs target mRNA coding regions and are used to knock down gene expression via targeted cleavage by an RNA-induced silencing complex (RISC).The clinical development of this strategy was pioneered by Alnylam Pharmaceuticals Inc., which last month entered into a major collaboration to develop products with the Genzyme Corp. unit of Sanofi.2...