Tag: microRNA
-

Treating Huntington’s Disease and Beyond: Three Applications of miRNAs in Gene Therapy
A microRNA (miRNA) is on the news since Wednesday; many just haven’t been told. I am referring to AMT-130 by UniQure, the gene therapy slowing the progression of Huntington’s disease [1]. In a nutshell, the miRNA in AMT-130 reduces the level of the protein huntingtin in neurons, which a mutation has turned toxic in Huntington…
