Terence Flotte, M.D.   Lab | Faculty Page

Dean School of Medicine, Provost and Executive Deputy Chancellor, Professor, Pediatrics

Reccombinant adeno-associated virus (AAV) gene therapy vectors for cystic fibrosis, alpha-1-antritrypsin deficiency (AAT) and fatty oxidation disorders (FAO).



Guangping Gao, Ph.D. Lab | Faculty Page

Director Gene Therapy Center & Vector Core, Scientific Director UMMS-China Program Office, Professor, Molecular Genetics & Microbiology 

Adeno-associated virus (AAV) vector mediated gene transfer for gene therapy, molecular mechanisms of AAV evolution and diversity, molecular & cellular mechanisms of AAV vector transduction, & gene therapy of inherited neurodegenerative Canavan Disease.


Miguel Sena-Esteves, Ph.D. Faculty Page

Associate Professor, Department of Neurology

Neurological diseases such as: Lysosomal storage, GM1-gangliosidois, Tay-Sachs and Sandhoff, Alzheimer’s, Ataxia Telangiectasia; along with brain tumors and brain tumor migration.


Christian Mueller, Ph.D.  | Faculty Page

Assistant Professor, Department of Pediatrics

Cystic fibrosis related inflammation and immunology, AAV mediated delivery of shRNA and miRNAs for RNAi therapy.


Hemant Khanna, Ph.D.  | Faculty Page

Assistant Professor, Department of Ophthalmology


Claudio Punzo, Ph.D. Lab|Faculty Page

Assistant Professor, Department of Ophthalmology