Genetic Therapies - Using DNA as a drug



A large number of diseases are caused by DNA mutations in the genome (inherited diseases) or by acquiring DNA of pathogens (acquired diseases). We will discuss how viruses and other jumping DNA elements can be used to help alleviate or to cure disease states. These strategies, called gene therapy, have recently showed some stunning successes to cure immuno-deficiencies that previously confined patients to isolation (bubble boy), to cure bleeding disorders (hemophilia) and to activate the immune system to fight cancers. This approach will change how medicine is practiced for the treatment of disease using DNA as a drug.



Nikunj Somia, PhD, is an associate professor in the Department of Genetics, Cell Biology and Development. His work is focused on developing vectors for transferring DNA into cells using the human immunodefiency virus (HIV) as a trojan horse. Dr. Somia previously served on the Recombinant DNA Committee (RAC) that advised the NIH director on new technologies using recombinant DNA in humans.


R. Scott McIvor, PhD, is professor in the Department of Genetics, Cell Biology and Development and the Center for Genome Engineering. Dr. McIvor uses several different methods for introducing DNA into cells and tissues for correction of inherited enzyme deficiencies that cause storage disease or immunodeficiency and for treatment of cancer. He works with several biotech companies that are developing products for DNA therapy, including Immusoft Corp where he serves as chief development officer.


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