Molecular and Experimental Medicine

Introduction

Living organisms have developed a number of mechanisms to defend the body against injury. Among these are the ability to kill microorganisms that invade tissues, staunch the flow of blood from a wound, and eliminate most cells that have become abnormal and could become cancerous. Paradoxically, the body's defense mechanisms can also be the cause of a number of debilitating and life-threatening diseases.

The majority of studies in the Molecular and Experimental Medicine Department seek to understand the life processes that, when disturbed, lead to disease.

Autoimmune disease, such as scleroderma, lupus erythematosus, and Sjogren's syndrome, represent a failure of the body's defense system that regulates recognition of self and non-self. Understanding the molecular targets of the immune attack is expected to lead to a better understanding of the immune system, the nature of these disorders, and how to prevent or treat them.

One way that the immune response can cause illness is in the attack of cells that harbor viral proteins. For example, the factors that regulate the attack on liver cells that are producing different components of hepatitis virus are being investigated in mice. These studies could well lead to ways that could prevent the destruction of the liver in patients with chronic hepatitis. The role of genetic variability is also being investigated in a broad range of life processes, including inflammatory mediators and blood clotting proteins.

Thus, research in this Department is wide-ranging and involves several related fields of study, including: blood coagulation, the biology of platelets, hemodynamics and bleeding disorders, and the role of thrombosis in cardiovascular and cerebrovascular diseases. The Department also includes a clinical research component in the areas of the biochemistry of anticancer drugs, inflammation, and cancer-causing genes.

TSRI can point to a number of important achievements in the clinical arena. Among them:

• Development of 2-chlorodeoxyadenosine for the treatment of hairy cell leukemia, other lymphoid neoplasms, and autoimmune disease.
• Purification of the antihemophilic Factor VIII, a coagulation protein lacking in people with hemophilia A. Monoclate, the purified concentration of Factor VIII, enables hemophiliacs to receive donated blood plasma that is free of viral contamination.
• Development of improved methods of diagnosing and treating patients with Gaucher's disease, a rare chronic congenital disorder of lipid metabolism.