Weill Cornell Medicine
Genetic Medicine
Weill Cornell Medicine
Research 1

Research

The Department of Genetic Medicine is a leader in gene therapy research, being the first to use a recombinant virus as a vehicle for in vivo gene therapy. Our faculty collaborates across various departments at Weill Cornell Medical College and Cornell University to refine, control, and target gene expression, supporting the translation of our research into practical treatments. Our research aims to develop treatments that tackle diseases at their genetic roots and can be divided into three main areas:

  1. Discovery Research: We test new theories about diseases and explore innovative ways to control and deliver genes to cells and tissues. Our current focus is on using viral vectors to deliver redesigned therapeutic proteins with the potential to be more effective than the native proteins they replace.
  2. Development Research: We enhance existing research by incorporating new technologies for better gene control and delivery. This includes using RNA interference to target gene expression to specific organs and optimizing viral vector targeting for disease treatment.
  3. Candidate Therapies: We develop and translate research into gene therapies and vaccines. Our strategies include using DNA-based viral vectors to treat central nervous system disorders, including Alzheimer's and vision loss. Additional research is aimed at enhancing orthopedic repair, protecting against allergies, treating metabolic disorders, and preventing damage due to oxidative stress.

The overall goal of our translational research program is to utilize our expertise in the fields of genetic therapies and personalized medicine to produce clinical solutions that address the molecular causes of human disease. 

Gene Therapy and Molecular Genetics
Personalized Medicine

Recent Publications

2024

Nakamura K, Henry T, Traverse J, Latter D, Mokadam N, Answini G, Williams A, Sun B, Burke C, Bakaeen F, DiCarli M, Chaitman B, Peterson M, Byrnes D, Ohman E, Pepine C, Crystal R, Rosengart T, Kowalewski E, Koch G, Dittrich H, Povsic T . 2024. Angiogenic Gene Therapy for Refractory Angina: Results of the EXACT Phase 2 Trial. Circ Cardiovasc Interv
Rojsajjakul T, Selvan N, De B, Rosenberg J, Kaminsky S, Sondhi D, Janki P, Crystal R, Mesaros C, Khanna R, Blair I . 2024. Expression and processing of mature human frataxin after gene therapy in mice. Sci Rep

2023

Zaidman C, Goedeker N, Aqul A, Butterfield R, Connolly A, Crystal R, Godwin K, Hor K, Mathews K, Proud C, Kula Smyth E, Veerapandiyan A, Watkins P, Mendell J . 2024. Management of Select Adverse Events Following Delandistrogene Moxeparvovec Gene Therapy for Patients With Duchenne Muscular Dystrophy. J Neuromuscul Dis
Rosenberg J, Fung E, Dyke J, De B, Lou H, Kelly J, Reejhsinghani L, Ricart Arbona R, Sondhi D, Kaminsky S, Cartier N, Hinderer C, Hordeaux J, Wilson J, Ballon D, Crystal R . 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. Hum Gene Ther
Rostami M, Leopold P, Vasquez J, de Mulder Rougvie M, Al Shakaki A, Hssain A, Robay A, Hackett N, Mezey J, Crystal R . 2023. Predicted deleterious variants in the human genome relevant to gene therapy with adeno-associated virus vectors. Mol Ther Methods Clin Dev
Pagovich O, Crystal R . 2023. Gene Therapy for Immunoglobulin E, Complement-Mediated, and Eosinophilic Disorders. Hum Gene Ther
Strulovici-Barel Y, Rostami M, Kaner R, Mezey J, Crystal R . 2023. Serial Sampling of the Small Airway Epithelium to Identify Persistent Smoking-dysregulated Genes. Am J Respir Crit Care Med
De B, Rosenberg J, Selvan N, Wilson I, Yusufzai N, Greco A, Kaminsky S, Heier L, Ricart Arbona R, Miranda I, Monette S, Nair A, Khanna R, Crystal R, Sondhi D . 2023. Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease. Hum Gene Ther
De B, Cram S, Lee H, Rosenberg J, Sondhi D, Crystal R, Kaminsky S . 2023. Assessment of Residual Full-Length SV40 Large T Antigen in Clinical-Grade Adeno-Associated Virus Vectors Produced in 293T Cells. Hum Gene Ther
Povsic T, Henry T, Traverse J, Anderson R, Answini G, Sun B, Arnaoutakis G, Boudoulas K, Williams A, Dittrich H, Tarka E, Latter D, Ohman E, Peterson M, Byrnes D, Pepine C, DiCarli M, Crystal R, Rosengart T, Mokadam N . 2023. EXACT Trial: Results of the Phase 1 Dose-Escalation Study. Circ Cardiovasc Interv
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