Understanding the genes associated with human diseases is a critical first step toward the development of more effective therapies. The identities of these genes, and the culprit changes within them, have been largely impossible to uncover for most common diseases. A revolution in human genetics is now underway that makes these discoveries possible for a range of metabolic, immune-related and psychiatric diseases. Mark Daly will discuss this scientific revolution, what the new genetic discoveries can and cannot tell us, and how this knowledge can help seed ideas for novel treatments.
The human body is made up of at least 100 distinct types of cells -- brain, liver, bone, and skin to name a few -- but all of them share the exact same genetic blueprint or "genome." You can imagine the human genome as a piece of origami paper that can be folded in different ways to give rise to these different cell types. John Rinn will discuss this DNA folding process, the surprising roles RNA plays in this process, and how this relates to human biology and disease.
There are hundreds of thousands of proteins in our bodies, many of them with links to human disease. But only a tiny fraction of these proteins is considered "druggable" -- that is, able to be targeted by drugs. Thanks to a new generation of drug discovery technologies, that tiny fraction is now on the rise. Angela Koehler will describe one of these new technologies and how it can be used to identify potential drugs that do the impossible: bind to and disrupt the functions of "undruggable" proteins.
Few diseases are as old, or have had as great an impact on history, as Tuberculosis (TB). More than a century after the discovery of the microbe that causes TB, and 50 years after the first antibiotic treatments, TB remains a worldwide killer. New technologies are enabling novel strategies for studying, diagnosing and treating this disease. James Galagan will discuss the history and biology of TB, and how genomics is providing new insight into topics ranging from the progression of the disease and the genetics of drug resistance to the evolution of the TB microbe.