Harbingers of Things to Come in Gene Therapy
Harbingers of Things to Come in Gene Therapy A relatively new gene therapy approach involves the correction or destruction of disease-causing RNA. This basic approach is being refined by Locanabio, which uses adeno-associated vectors to deliver RNA-binding proteins. In an evaluation of Locanabio’s delivery technology in a wild-type mouse, the company’s Claire Geddes, PhD, obtained the image shown here. It shows mouse cerebellum two weeks post deep cerebellar nuclei delivery with AAVrh10-GFP (green: cells expressing green fluorescent protein; red: Purkinje cells labeled with anti-Calbindi...