Overview

The Department of Gene and Cell Medicine is driven by basic research with the goal of understanding and overcoming the stumbling blocks preventing the development of successful gene and cell therapy treatments from bench to bedside. Thus, the Center is taking a multi-prong approach, investigating various fundamental areas of research that will help overcome this problem.

Initially, research focused on various aspects of gene therapy, such as understanding basic virology , efficient gene delivery into the nucleus of cells, and incorporation of these genes into the genome.

The Center has since broadened its scope to integrate gene therapy and stem cell biology. In addition to targeting diseased tissues directly, like the liver, the Center is also studying how to deliver genes to stem cells, specifically hematopoietic stem cells. Since hematopoietic stem cells develop into the various types of peripheral blood cells, learning how to insert genes into these cells could potentially lead to the treatment of various hematological diseases. Others are working on embryonic stem cells.

While cellular therapies hold the potential to treat many different diseases, they also carry the risk of stimulating the immune system in a way that reduces their effectiveness. Therefore, a fundamental understanding of immunology; both at the molecular and transplant levels will be key to the success of gene and cell therapies.

This combination of interdisciplinary research is already proving fruitful. Initial studies in laboratory animal models of cancer have been moved into human clinical trials, conducted in collaboration with faculty from Mount Sinai's clinical departments.

The Center's unique approach of looking at the whole scope of gene therapy and stem cell biology represents a powerful paradigm to exciting future therapeutics.