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From Genes to Therapy

A critical challenge for the coming years is to identify targets and design effective therapeutic strategies for inflammatory diseases. The revolution in human genetics has revealed a large number of genes and pathways associated with these diseases, and emerging methodologies are starting to systematically analyze the cellular and molecular circuitry underlying disease. At the Broad Institute, we are inventing approaches to identify the best targets and develop new therapies.

Inflammatory bowel disease

Inflammatory bowel diseases (IBD), which include Crohn’s disease and ulcerative colitis, affect more than a million people in the United States and have no known cause. The current model of IBD is based on a combination of increased genetic risk and an immune system that overreacts to environmental and microbial stresses. Here at the Broad we combine human genetics, genetic and chemical screens, and population-based cohort studies to uncover the basic mechanisms of IBD, which may one day be used to develop effective treatments.

Team members: Xavier

Lupus nephritis

Lupus nephritis is a devastating disease and remains an unsolved medical problem. Current therapies are not effective enough, lead to numerous adverse effects, and allow too many patients to progress to end stage renal disease. We are interested in using novel molecular and cellular methods to study the pathogenic mechanisms of lupus nephritis. Using genetic and epigenetic analyses coupled with powerful perturbation technologies to test gene functions in human cells and mouse models, we hope to identify the critical drivers of this disease and the basis for therapeutic responses. Our goal is to propose and develop new therapeutic strategies, including personalized approaches to each individual’s disease.

Team members: Hacohen

Multiple Sclerosis

Multiple sclerosis affects more than 200,000 individuals in the United States each year. No single genetic variant, environmental factor, or malfunction in the immune cells can be held responsible for the disease. Instead, a combination of multiple mutations, environmental factors, and cellular malfunctions conspire together to increase one’s risk of the disease. As such, we take a multi-pronged approach to studying MS, which includes mapping the genetic architecture of the disease, investigating the functional consequences of genetic loci associated with MS susceptibility, and clinical research.