


Science 1 English

Disease State


Science 2 English

Gene Therapy


Science 3 English

RNA Technologies

Genetic diseases can be caused by a mutation in the gene. Most commonly, one or more exons (parts of the gene) are missing, causing errors in the instructions for making a protein, which results in the body not being able to produce enough—or any—of the protein. The goal of exon skipping, is to act on the RNA to allow the body to make a version of the missing protein to bypass the mutation.

Science 4 English

Gene Editing

Sarepta is investigating ways to restore protein expression by removing—or excising—exons that contain a genetic mutation using gene editing technology such as CRISPR/Cas9.