Gene Therapy

Biomedical research has reached a critical threshold with the many scientific triumphs of the last century, most recently the complete sequencing of the human genome. With such tools in hand, researchers are now moving at an accelerated pace to create new and innovative therapeutics for the 21st century. Two of the most exciting new approaches are gene therapy and stem cell biology, which promise to revolutionize medicine and health care in decades to come. These approaches have the potential to provide a cure for both genetic and acquired diseases, including hemophilia, cystic fibrosis, diabetes, cancer, heart disease, AIDS, Parkinson's, and Alzheimer's, just to name a few.

Despite dramatic progress providing proof of principle that inherited disorders in laboratory animals can be corrected permanently by gene therapy, the field is still in its infancy. Since the first gene therapy clinical trial began in 1990, none has developed beyond the human trial stage to become U.S. FDA-licensed products. Much fundamental work must be done before gene therapy can be widely used to treat human diseases.

Anticipating the new horizons of biomedical research, the Department of Gene and Cell Medicine was established in 1996 at Mount Sinai School of Medicine to promote and accelerate the science and technology on the development of gene and cellular therapeutics. The Department is driven by basic research with the goal of understanding and overcoming the obstacles that prevent the development of successful gene and cell therapy treatments from the bench to the bedside.

The backbone of the Department's research is its faculty, who are recruited to Mount Sinai not only for their outstanding scientific instincts and accomplishments but also for a rare combination of independent investigation and collaborative spirit. They represent a rich tapestry of disciplines, which complement each other, including molecular biology of gene expression, gene regulation and delivery, biology of stem cell development and differentiation into various tissues, virology, tumor immunology, as well as transplant immunology. "Harnessing interdisciplinary expertise is essential to efficient translation of research and development into clinically effective therapies and safe product development," explains Savio Woo, professor and founding chairman, Department of Gene and Cell Medicine.

The Department's scientists work within an infrastructure set up to advance research from basic science to animal models to federally supported clinical trials, all on site. This environment fosters creativity and collaboration not only among researchers within the Department, but also with the larger community of Mount Sinai. Since its conception few years ago, the Center has already moved promising gene therapy results in laboratory animal models of cancer to clinical translational studies, and now human clinical trials are conducted in collaboration with faculty of various Mount Sinai clinical departments.

"Our Department is unique because we look at the whole scope of gene and cellular therapies. Though our studies are strictly basic science initiated, diversity and the ability of faculty in different disciplines to work next door to each other is generating new knowledge that will be useful in the clinic," says Woo.