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

Program

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”

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”

“Practical Strategies”: Clinical Trials for Pompe Disease

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

Jacuzzi Family Vineyards

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