RNA Editing for Genetic Treatment

In recent years, extensive research presented gene therapy as a groundbreaking approach for curing genetic diseases. For the most part, genetic modulation is performed on the mutated gene at the DNA level. This results in a stable change, that provides a long-lasting refuge from the disease. Nevertheless, DNA editing tools still hold many dangers. For one, the editing enzyme might cut the DNA strand in an off-target site, causing permanent genetic abnormalities. To overcome this challenge, RNA editing methods have arisen to treat specific genetic diseases. In an article published in Molecular Therapy: Nucleic Acids, Prof. Shay Ben-Aroya, Prof. Dror Sharon and Prof. Erez Levanon describe a new development for treating genetic retinal diseases using the  ADAR (adenosine deaminase acting on RNA) enzyme. ADAR is naturally expressed in human cells, and is known to mediate only RNA editing without altering the DNA. This makes it a promising target for treating genetic diseases without risking farther damage.