CRISPR mediated exon-skipping is a method of genome editing that can be used to develop life-saving therapeutics for people with rare and ultra-rare genetic disorders. CRD is explaining the science behind these technologies as well as how they are the key to treating these diseases.

​​We are proud to announce the creation of our Scientific Advisory Board (SAB)! Joining the SAB are leading experts in gene therapy and neuromuscular disorders who will help advance our preclinical and clinical development of gene therapy and antisense oligonucleotide programs.

We previously lived in Central California and raised organically fed pork on pasture. When Kal was diagnosed with Duchenne...