Horae Gene Therapy Center Faculty

  • Mai ElMallah

    Mai ElMallah, M.D.

    Academic Role: Assistant Professor, Department of Pediatrics, Division of Pulmonary Medicine

    Focus: Gene therapy and neuromodulation for respiratory insufficiency in genetic diseases.  Respiratory physiology and neurophysiology core.

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  • Terence  Flotte

    Terence Flotte, M.D.

    Academic Role: Dean 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

    Guangping Gao, Ph.D.

    Academic Role: Professor Department of Microbiology and Physiological Systems, Director Gene Therapy Center & Vector Core, Scientific Director UMMS-China Translational Research Initiatives

    Focus: Adeno-associated virus vector mediated gene transfer for gene therapy & gene therapy of inherited neurodegenerative Canavan Disease.

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  • Hemant Khanna

    Hemant Khanna, Ph.D.

    Academic Role: Assistant Professor, Department of Ophthalmology

    Focus: Molecular mechanisms and treatment strategies for inherited retinal and macular degenerative diseases.

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  • Chris Mueller

    Chris Mueller, Ph.D

    Academic Role: Assistant Professor, Department of Pediatrics

    Focus: Developemnt of gene therapeuitc approaches for genetic disorders of the lung, liver and central nervous system.

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  • Claudio Punzo

    Claudio Punzo, Ph.D

    Academic Role: Assistant Professor, Department of Ophthalmology

    Focus: Retinal degenerative diseases that affect photoreceptors such as Retinitis Pigmentosa and Age-Related Macular degeneration.

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  • Miguel Sena-Esteves

    Miguel Sena-Esteves, Ph.D.

    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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  • Qiushi Tang

    Qiushi Tang, M.S.

    Academic Role: Instructor, Department of Pediatrics

    Focus: Adeno-associated virus (AAV) gene therapy research for alpha-1-antitrypsin deficiency (AAT) disease.

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  • Jun Xie

    Jun Xie, Ph.D

    Academic Role: Instructor, Department of Microbiology and Physiological Systems

    Focus: AAV vector technology and AAV-mediated miRNA therapeutics.

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  • Li Zhong

    Li Zhong, M.D

    Academic Role: Research Associate Professor, Department of Pediatrics; Director: Vector development of Vector Core

    Focus: Molecular biology of human parvoviruses and their use as vectors in human gene therapy as well as gene therapy for hematopoietic diseases and neurodegenerative diseases.

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