Genetic Medicine

About Us
About Us
The Department of Genetic Medicine at Weill Cornell leads a dynamic and innovative translational research program, advancing diverse fields such as Genetic Therapy and Personalized Medicine.
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Genetic Medicine Community
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Research
About Our Research
Our translational research program aims to leverage our expertise in genetic therapies and personalized medicine to develop clinical solutions that target the molecular causes of human diseases.
Gene Therapy and Molecular Genetics
Personalized Medicine
Clinical Trials
About Clinical Trials
The Department of Genetic Medicine advances treatments and diagnostics through diverse clinical trials, including drug testing and research to better understand diseases.
Active Trials & Recruitment
Completed Trials & Publications
Faculty & Staff
Belfer Gene Therapy Core Facility
About Our Facility
The Belfer Gene Therapy Core Facility (BGTCF) is a cutting-edge genetic medicine research facility.
BGTCF Faculty & Staff
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Crystal Clinic

Genetic Medicine

Completed Trials & Publications

Gene Therapy for APOE4 Homozygote of Alzheimer's Disease

Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis

AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis

Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)

Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis

Genotype - Phenotype Correlations of LINCL

Doxycycline for COPD in HIV-Infected Patients

Safety and Efficacy of a Lysophosphatidic Acid Receptor Antagonist in Idiopathic Pulmonary Fibrosis

Study to Assess the Efficacy and Safety of Simtuzumab (GS-6624) in Adults With Idiopathic Pulmonary Fibrosis (IPF) (RAINIER)

A Study of Lebrikizumab in Participants With Idiopathic Pulmonary Fibrosis (IPF)

Phase 3 Study With Ciprofloxacin Dispersion for Inhalation in Non-CF Bronchiectasis (ORBIT-4)

Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA) (AAVance)