StemCell Therapy

Our translational research program includes many projects in the fields of genetic therapies and personalized medicine. The field of genetic therapies comprises gene and stem cell therapies and our laboratory has extensive expertise in both areas. Our group was the first to use a recombinant virus as a vehicle for in vivo gene therapy and we have carried out human trials of gene therapy for cystic fibrosis, cardiac ischemia, cancer and central nervous system disorders. Among the current projects are gene transfer strategies for cancer, inherited CNS disorders, 1-antitrypsin deficiency, anti-bioterrorism applications and development of vaccines. We also operate the clinical vector production laboratory of the Belfer Gene Therapy Core Facility, which has produced adenovirus and adeno-associated virus vectors that have been used in numerous human studies. Current projects in the field of stem cell therapy include characterization of the roles of cancer stem cells in lung cancer and the role of airway epithelium stem cells in chronic obstructive pulmonary disease.

Personalized medicine is the use of information and data from an individual's genotype, or level of gene expression to stratify complex diseases, select a medication or dose of a medication, provide a therapy, or initiate a preventative measure that is specifically suited to that patient. In addition to genetic information, other factors, including imaging, laboratory, and clinical information about the disease process or the patient are integrated into the process of developing personalized medicine. Our group utilizes microarray technologies for genome-wide characterization of gene expression, single nucleotide polymorphism and copy number variation profiles on clinical samples as the basis for projects aimed at indentifying candidate genes associated with complex disease such as chronic obstructive pulmonary disease.

The overall research program of the group includes close collaborations with other laboratories at Weill Cornell and elsewhere, including Malcolm Moore's group at Memorial Sloan Kettering Cancer Center for stem cell projects. Of particular note are our collaborations on personalized medicine projects with colleagues at Weill Cornell Medical College-Qatar and Hamad Medical Corporation in Doha, Qatar and collaborations on Bioinformatics and Biostatistical Genetics with several laboratories at Cornell-Ithaca, including Andy Clark and Jason Mezey.

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Department of Genetic Medicine - Weill Cornell Medical College

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