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?
Recent News
- Weill Cornell Department of Genetic Medicine initiates NIH-funded gene therapy clinical trial aimed at preserving heart health in individuals with Friedreich’s ataxia (February 2022)
- DGM awarded NIH National Institute of Biomedical Imaging and Bioengineering R01 grant to study non-invasive methods to image AAV gene vectors in vivo
Recent Publications
- De, B. P., Rosenberg, J. B., Selvan, N., Wilson, I., Yusufzai, N., Greco, A., Kaminsky, S. M., Heier, L. A., Ricart Arbona, R. J., Miranda, I. C., Monette, S., Nair, A., Khanna, R., Crystal, R. G., & Sondhi, D. (2023). Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease. Human Gene Therapy, 34(17-18), 905-916. doi:10.1089/hum.2023.067
- Rosenberg, J. B., Fung, E. K., Dyke, J. P., De, B. P., Lou, H., Kelly, J. M., Reejhsinghani, L., Ricart Arbona, R. J., Sondhi, D., Kaminsky, S. M., Cartier, N., Hinderer, C., Hordeaux, J., Wilson, J. M., Ballon, D. J., & Crystal, R. G. (2023). Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid. Human Gene Therapy, Article P977. doi:10.1089/hum.2023.060
- Munoz-Zuluaga, C., Gertz, M., Yost-Bido, M., Greco, A., Gorman, N., Chen, A., Kooner, V., Rosenberg, J. B., De, B. P., Kaminsky, S. M., Borczuk, A., Ricart Arbona, R. J., Martin, H. R., Monette, S., Khanna, R., Barth, J. A., Crystal, R. G., & Sondhi, D. (2023). Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia. Human Gene Therapy, 34(13-14), 605-615. doi:10.1089/hum.2023.020
- Rosenberg, J. B., De, B. P., Greco, A., Gorman, N., Kooner, V., Chen, A., Yost-Bido, M., Munoz-Zuluaga, C., Kaminsky, S. M., Rostami, M., Monette, S., Crystal, R. G., & Sondhi, D. (2023). Safety of Intravenous Administration of an AAV8 Vector Coding for an Oxidation-Resistant Human α1-Antitrypsin for the Treatment of α1-Antitrypsin Deficiency. Human Gene Therapy, 34(3-4), 139-149. doi:10.1089/hum.2022.192
- Havlicek, D. F., Rosenberg, J. B., De, B. P., Hicks, M. J., Sondhi, D., Kaminsky, S. M., & Crystal, R. G. (2020). Cocaine vaccine dAd5GNE protects against moderate daily and high-dose "binge" cocaine use. PloS One, 15(11), e0239780. doi:10.1371/journal.pone.0239780