Annual Gene Therapy Symposium for
Heart, Lung, and Blood Diseases
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UC Davis


14th Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases
Focus Topic: Genetic Disease Applications
November 18 - 20, 2015 - Sonoma, California



Donald B. Kohn, M.D.Thursday, November 19, 2015

Keynote Speaker

  • Donald B. Kohn, M.D. (UCLA)
    “Gene Therapy for Blood Cell Diseases with Autologous Hematopoietic Stem Cells”

Session I 

  • John C. Burnett, Ph.D. (City of Hope)
    “RNA-Based Approach to Eliminate Persistent HIV-1 Infection and Latency”
  • Paloma H. Giangrande, Ph.D. (University of Iowa)
    “Cell-Targeted RNA Therapies for Cardiovascular Disease”
  • David V. Schaffer, Ph.D. (UC Berkeley)
    “Directed Evolution of New Viruses for Therapeutic Gene Delivery”
Student/Fellow Presentations and Poster Session - Link to Information

Session II   

  • Manuela Corti, P.T., Ph.D. (University of Florida)
    “Gene Therapy for Friedreich's Ataxia: Pathway to the Clinic”
  • Steven J. Gray, Ph.D. (University of North Carolina at Chapel Hill)
    “Development of a First-in-Human Intrathecal scAAV9 Gene Therapy for Giant Axonal Neuropathy”
  • James M. Wilson, M.D., Ph.D. (University of Pennsylvania)
    “CNS Gene Therapy for Neuropathic Lysosomal Storage Diseases”

Barry J. Byrne, M.D., Ph.D.“Practical Strategies”: Clinical Trials for Pompe Disease

  • Barry J. Byrne, M.D., Ph.D. (University of Florida)


Jacuzzi Family Vineyards

Reception  Reception  Reception 

Friday, November 21, 2014

Hans-Peter Kiem

Mini-Workshop: “Cell and Gene-Based Therapy”

  • Hans-Peter Kiem, M.D. (University of Washington)
    “Engineering Hematopoietic Stem Cells for the Treatment of HIV and Cancer”

Session III

  • Anna La Torre, Ph.D. (UC Davis)
    “Tackling Retinal Ganglion Cell Degenerations: Stem Cell Approach to Ocular Disease Therapies”

  • Mervin C. Yoder, M.D. (Indiana University)
    “Generating Endothelial Colony Forming Cells from Induced Pluripotent Stem Cells”

  • Bernard Thébaud, M.D., Ph.D. (Ottawa Hospital Research Institute)
    “Cell Therapies for Monogenetic Neonatal Lung Diseases”

  • David Warburton, D.Sc., M.D., F.R.C.P., F.R.C.S. (Children's Hospital Los Angeles)
    “LungMAP: A Useful Public Resource to Understand Normal Human and Mouse Alveolarization, Integrated at Multiple Scales and Multiple Levels of Analysis”

Student/Fellow Presentations and Poster Session - Link to Information


Supported by the National Heart, Lung, and Blood Institute, National Institutes of Health. Additional support provided by the National Institute of Allergy and Infectious Diseases; California Institute for Regenerative Medicine (CIRM); UC Davis Office of Research; California National Primate Research Center, Stem Cell Program, and the UC Davis Clinical and Translational Science Center.