Horae Gene Therapy Center Faculty
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Sylvain Cecchini, PhD
Academic Role: Associate Professor, Director of Large AAV Core Service Center
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Eleonora D'Ambrosio, MD
Academic Role: Physician and Assistant Professor, Department of Neurology
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Terence Flotte, MD
Academic Role: Dean T.H. Chan School of Medicine, Provost and Executive Deputy Chancellor, Professor, Pediatrics
Focus: Recombinant adeno-associated virus (AAV) gene therapy vectors for, cystic fibrosis (CF), alpha-1-antitrypsin deficiency (AAT) and fatty oxidation disorders (FAO).
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Guangping Gao, PhD
Academic Role: Professor and Founding Chair of the Department of Genetic and Cellular Medicine, Director Li Weibo Institute for Rare Diseases Research, Director of the Horae Gene Therapy Center & Viral Vector Core
Focus: Adeno-associated virus vector mediated gene transfer for gene therapy & gene therapy of inherited neurodegenerative Canavan Disease.
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Dominic Gessler, MD, PhD
Academic Role: Assistant Professor, Department of Neurosurgery
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Heather Gray-Edwards, DVM, PhD
Academic Role: Associate Professor, Department of Radiology
Focus: Development of MRI-based methods to examine disease progression of neurodegenerative disorders. Evaluating effectiveness of adeno-associated virus (AAV)-mediated gene therapy.
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Alisha Gruntman, DVM, PhD, DACVIM
Academic Role: Assistant Professor, Department of Pediatrics
Focus: Gene therapy and neuromodulation for respiratory insufficiency in genetic diseases. Respiratory physiology and neurophysiology core.
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Allison Keeler, PhD
Academic Role: Assistant Professor, Department of Pediatrics
Focus: Gene therapy and cell engineering for single gene disorders and novel immunotherapeutics
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Qinglan Ling, PhD
Academic Role: Assistant Professor, Department of Microbiology
My lab uses genetic approaches to understand and develop treatments for mitochondrial diseases. We develop the treatments in a translatable manner to directly benefit the patient population. The diseases that we are currently working on involve genes that are encoded in both the nuclear DNA and mitochondrial DNA, with neurological manifestations.
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Miguel Sena-Esteves, PhD
Academic Role: Associate Professor, Department of Neurology
Focus: Gene therapy approaches for the treatment of neurodegenerative lysosomal storage diseases such as GM1-gangliosidosis and GM2-gangliosidoses (Tay-Sachs and Sandhoff diseases).
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Jae-Hyuck Shim, PhD
Academic Role: Professor, Department of Medicine - Division of Rheumatology/Department of Immunology and Microbiology
Focus: Using genetic medicine treat to and/or cure skeletal rare diseases with monogenic mutations, such as fibrodysplasia ossificans progressiva or osteogenesis imperfecta.
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Phil Tai, PhD
Academic Role: Assistant Professor, Department of Microbiology and Physiological Systems
Focus: Development of next-gen sequencing methodologies to profile AAV vector genome quality, and discovery and characterization of natural AAV capsid isolates for vectorization and gene therapy.
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Dan Wang, PhD
Academic Role: Assistant Professor, RNA Therapeutics Institute
Focus: AAV-mediated RNA therapeutics and gene editing therapeutics; AAV biology; Gene therapy for rare diseases; Animal modeling.
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Jun Xie, PhD
Academic Role: Associate Professor, Department of Microbiology and Physiological Systems
Focus: AAV vector technology and AAV-mediated miRNA therapeutics.
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Guocai Zhong, PhD
Academic Role: Assistant Professor, RNA Therapeutics Institute
Focus: RNA switch engineering and regulatable gene therapy development for fatal genetic disease, metabolic disorders, and infectious diseases.
