Cut and Paste: Spinal Muscular Atrophy with Nusinersen

In order to make protein, the gibberish portions of RNA are removed to create a coherent message. This is called RNA splicing.

RNA splicing of a specific gene is defective in Spinal Muscular Atrophy. A new drug called “nusinersen” is attempting to repair this splicing defect.

This video was produced in collaboration with Cold Spring Harbor Laboratory.