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 disease-causing mutations. Gene editing is one of the newest classes of medicines and holds the promise of removing the genetic causes of cancer, cardiovascular diseases, neurodegenerative diseases and rare diseases. Gene editing medicines may also be effective against infectious organisms.
Gene editing tools such as CRISPR and TALEN use a mixture of enzymes, RNA and DNA to cut out mutated DNA and replace it with a healthy sequence. This treats the genetic cause of disease.
RNA, DNA and enzymes are rapidly degraded in the gut and in the blood stream. Arcturus’ proprietary LUNAR technology can be used to deliver gene editing tools directly into target cells where they can edit out disease-causing mutations.
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