Disease Targets
- Friedreich’s ataxia
- Alzheimer’s disease
- Cocaine addiction
- Alpha-1 antitrypsin deficiency
- Eosiniphilia/Eosinophilic esophagitis
- Angiogenesis for heart disease, fat transplant, and surgical wound healing
Gene Therapy and Molecular Genetics
Gene therapy research is a cutting-edge field of study that aims to treat or prevent diseases by modifying or correcting genetic material within a patient's cells. This innovative approach involves introducing genetic material into a person's cells to replace missing or defective genes, or to introduce new functions.
The Department of Genetic Medicine has an enduring history of accomplishment in the field of gene therapy. Our department chair, Ronald G Crystal, is the first physician to administer a genetically modified, replication-deficient virus to a human patient in an approved clinical trial. We continue to be pioneers in the field, combining state-of-the-art genetic and molecular techniques to identify new targets, develop new methods, and translate our research on complex diseases to the clinic.
Research Questions
- Can we enhance and monitor viral vector delivery to target organs?
- How can gene therapy be used to treat substance abuse disorders?
- Can we leverage epigenetic gene regulation to treat complex disorders?
News
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The investigational gene therapy involves a single, intravenous administration of a modified adeno-associated virus that delivers the normal frataxin (FXN) gene to the heart (and other organs). This is the first clinical gene therapy to treat Freidreich’s ataxia, and the goal of this initial clinical study is to assess safety and initial estimates of efficacy in treating heart disease associated with Friedreich’s ataxia. This clinical trial is funded by a grant with Co-PIs from the Department of Genetic Medicine (Dr. Ronald G. Crystal and Dr. Stephen Kaminsky) and the Department of Cardiology (Dr. Johnathan W. Weinsaft).