Rare Genetic Diseases

Many diseases do not have cures, yet from deleterious mutations in the genome. Many of these are rare disorders that do not benefit from extensive research or drug development. Current therapies mask symptoms more than solve the basis of the disorder. Dietrich Stephan of NewBase describes a novel custom drug design platform that shows amazing potential in animal models. A small oligonucelotide with a specific backbone can bind to its complementary sequence in DNA or RNA, derailing RNA polymerase, or dissociating other regulatory proteins.  The design is highly specific and shows great promise against a myriad of disorders, including Myotonic Dystrophy, Huntington's Disease, and various cancers. This episode features a deep dive into molecular biology, so please do not hesitate to ask questions, it is interesting technology.