Human DNA has roughly 20 000 genes. Each gene contains the code needed to make one or more proteins. In some cases, when a gene contains a mutation, the body makes too much or too little of the corresponding protein, or makes defective versions of the protein. Many diseases, including Huntington’s disease, cystic fibrosis and cancer, are caused by DNA mutations.
It is now possible to edit genes to remove mutations. One of the most popular gene editing tools, CRISPR, uses small strands of RNAs, called guide RNAs, that bind to the DNA surrounding a mutation and tell enzymes to cut and replace the mutated DNA with a healthy sequence. This treats the genetic cause of disease.
It is difficult to safely deliver gene-editing tools into cells because RNA and enzymes are rapidly degraded in the gut and in the blood stream. Arcturus’ proprietary unlocked nucleomonomer agent (UNA) Oligomer™ technology can be used to make guide RNAs that are protected from degradation and our patented LUNAR™ delivery system ensures gene-editing tools are delivered directly into target cells.