| | Gene Therapy Center : Our Faculty |
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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). |  |
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Guangping Gao, Ph.D. Lab | Faculty PageDirector 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. |  |
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Miguel Sena-Esteves, Ph.D. Faculty PageAssociate 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. |  |
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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. |  |
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| | Assistant Professor, Department of Ophthalmology |  |
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| | Assistant Professor, Department of Ophthalmology |  |
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