Research Frontiers

Dr. Ron Kopito and Dr. Brigit Riley

The “top-down” approach

On a basic level, it is known that HD is caused by a mutation in huntingtin that causes it to gain a new toxic function and harm the cell. But, as Dr. Kopito explains, there is not a single, direct route from the genetic mutation to the disease. Rather, the mutated huntingtin protein has different effects all over the cell. Dr. Kopito feels that when studying HD, it is hard to start by isolating each of the possible small problems that may be caused by the mutated huntingtin protein. Starting from the endpoint of the disease cascade will not provide much insight into understanding the disease. Rather, you have to study the disease as a whole to gain insight into each of the small steps, and how they interact with one another. This approach would be considered a top-down approach. So, Dr. Kopito likes to use animal models like mice to study the disease, rather than looking at HD only in tissue culture or in vitro. That way, he can look at the whole disease in the context of an animal, rather than just a fraction of it in the context of a test tube or Petri dish.

Last Modified: 09/11/2007

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