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

2025

Sondhi D, Kaminsky S, Rosenberg J, Rostami M, Hackett N, Crystal R . 2025. Twenty-Year Survival Analysis of Adeno-Associated Virus Vector Serotype 2-Mediated Gene Therapy to the Central Nervous System for CLN2 Disease. Hum Gene Ther

2024

Gioulvanidou M, Sarklioglu S, Chen X, Lebedeva I, Inalman Y, Pohl M, Bourne L, Andrew D, Lorenz I, Stiles K, Pagovich O, Hackett N, Kaminsky S, de Mulder Rougvie M, Crystal R . 2024. Vectorized Human Antibody-Mediated Anti-Eosinophil Gene Therapy. Hum Gene Ther
Günaydin C, Sondhi D, Kaminsky S, Lephart H, Leopold P, Hackett N, Khanna R, Crystal R . 2024. AAVrh.10 delivery of novel APOE2-Christchurch variant suppresses amyloid and Tau pathology in Alzheimer's disease mice. Mol Ther
Karan K, Andrzejewski S, Stiles K, Hackett N, Crystal R . 2024. Suppression of CNS APOE4 Expression by miRNAs Delivered by the S2 AAVrh.10 Capsid-Modified AAV Vector. Hum Gene Ther
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
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