At any time, most of our DNA is "silenced" or switched off by a chemical modification, except at a few specific sites, called CG islands, which serve as "on" switches for our genes. Chedin's lab has been studying how these CG islands are protected from silencing; he thinks that too little silencing might play a role in the development of systemic lupus.
He will test his hypothesis by using as a model system the severe but rare autoimmune disorder called Aicardi-Goutires syndrome, which mimics early onset lupus. The syndrome affects infants, and in most cases leads to death by age 10 to 15. Understanding the biological mechanisms that underlie Aicardi-Goutires will generate critical insights into how the innate immune system can be triggered. If Chedin is successful, his efforts will lead to the development of new diagnostic tests and therapies for children affected with early onset lupus and related autoimmune diseases.
Satake is working to develop a new method to treat childhood leukemia, called molecular targeting. The approach is different from conventional chemotherapy drugs, which kill healthy blood cells and leukemic cells equally. Satake proposes to develop a unique method to deliver a type of molecule that interferes with gene expression, called siRNA. She seeks to deliver it specifically to the leukemia cells, but not to normal healthy cells. If she is successful, her narrow-target approach could prove a transformative pediatric cancer treatment.
Satake explained that while considerable gains have been made in curing pediatric cancers, there are still lethal forms of the disease that resist treatment.
"Even when we cure the cancer, many of those patients are known to have significant long-term side effects or late e
|Contact: Andy Fell|
University of California - Davis