Every year, nearly 8 million children are born with genetic diseases caused by mutations in DNA. There are 6,000-7,000 known genetic disorders – examples include ALS, cystic fibrosis, muscular dystrophy, or Fuchs’ dystrophy. For over 95% of these diseases, no FDA-approved treatments or cures exist.
CRISPR is a revolutionary gene-editing tool with the ability to edit nearly any DNA sequence within the human genome, and the potential to correct a multitude of disease-causing mutations. While traditional medical research has focused on developing pharmaceuticals to treat the symptoms of genetic disorders, correcting DNA mutations directly could provide permanent, one-time cures.
However, the clinical promise of CRISPR is limited by the absence of a safe and effective method to deliver the genome-editing technology into cells within the human body (in vivo). The Hunterian technology uses a novel genetic element to enable in vivo delivery via a single adeno-associated virus (AAV).