Research Area
Diabetes

Grant Type
Network

Year
2001

Abstract

The general goal of my laboratory is to understand how signals from outside the cell lead to determination of the cell’s choice of growth, development, or differentiation. We are studying signal transduction, with particular reference to regulation of NF-kB involved in innate and adaptive immunity. We are also studying the molecular and biochemical function of BRCA1/BRCA2 involved in hereditary breast cancer.

Our other major focus is in gene therapy, where we have developed novel vectors based on lentiviruses, which have been shown to infect a wide variety of tissues in vivo. Specifically, we have shown that infection of liver, lung, brain, and hematopoietic stem cells, which upon transduction show long-term production of foreign protein. We are using these vectors to deliver the insulin gene to hematopoietic stem cells with a view toward providing basal levels of insulin. We are also generating vectors where expression of the insulin gene is regulated. We believe that these studies will help us understand the complex biological functions leading to cell growth, and development of methods to cure genetic or acquired diseases.

The specific research initiatives include: 1) the isolation of pancreatic stem cells, and evaluation of a variety of agents that may play important roles in determining the development of insulin-producing cells, 2) assessing the role of a category of cell adhesion molecules important in growth and differentiation in both pancreatic and human embryonic stem cells, and 3) elucidating the role of cell surface receptors that provide “cues” from outside the cell that determine whether a cell proliferates, differentiates, or dies. The success of these studies would be an important step toward creating beta cells that could be transplanted into patients with Type 1 diabetes.