12th Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases
Focus Topic: Tolerance and Immune Modulation
November 20-22, 2013 - Sonoma, California

Program

Wednesday, November 20, 2013 - Welcoming Reception

Thursday, November 21, 2013

Keynote Speaker

• Katherine A. High, M.D. (The Children's Hospital of Philadelphia)
  
  “Immune Responses to AAV Vectors: Overcoming Barriers to Successful Gene Therapy”

Session I: Vectors  

• R. Jude Samulski, Ph.D. (University of North Carolina)
  “Designing AAV Vectors for Clinical Studies”

• Michael C. Holmes, Ph.D. (Sangamo BioSciences, Inc.)
  “In Vivo Genome Editing with Zinc Finger Nucleases”

• Paloma H. Giangrande, Ph.D. (University of Iowa)
  “Cell-Targeted RNA Therapies”

Session II   

• Joshua M. Hare, M.D. (University of Miami)
  “Cardiac Regeneration: From Concept to Clinic”
  

• H. Kirk Hammond, M.D. (University of California, San Diego)
  “Brief Update of AC6 Gene Transfer for Clinical CHF and Gene Transfer of Peptides with Paracrine Actions that Benefit the Heart”

• Robert T. Tranquillo, Ph.D. (University of Minnesota)
  “Implantation of Completely Biological Engineered Arteries into the Sheep Femoral Artery”

“Practical Strategies”: Immune Modulation

• Roland W. Herzog, Ph.D. (University of Florida)
  

Jacuzzi Family Vineyards

Mini-Workshop: “Stem/Progenitor Cells and the Immune System”

• Katarina Le Blanc, M.D., Ph.D. (Karolinska Institute)
  “Bringing Mesenchymal Stem Cells into the Clinic”

• Claudia dos Santos, M.D. (University of Toronto)
  “Mechanisms of Protection and Informed Target Discovery for Gene and Stem Cell Therapy in Sepsis”

Session III

• Tippi MacKenzie, M.D. (University of California, San Francisco)
  “Mechanisms of Tolerance Induction After In Utero Hematopoietic Cell Transplantation”

• Eirini Papapetrou, M.D., Ph.D. (University of Washington)
  “Modelling Myelodysplasia with Human Pluripotent Stem Cells"”
  
  
• R. Scott McIvor, Ph.D. (University of Minnesota)
  "Effect of Immune Response on Transgene Expression: Sleeping Beauty Medicated Gene Transfer to the Liver and AAV Mediated Gene Transfer to the Brain"

Student/Fellow Presentations and Poster Session - Link to Information

Session IV

• Brigitte Gomperts, M.D. (University of California, Los Angeles)
  “Using Induced Pluripotent Stem Cells to Advance Our Understanding of Lung Disease”

• Barry R. Stripp, Ph.D. (Cedars-Sinai Medical Center)
  “Microenvironmental Factors Regulating Progenitor Cell Fate in Airways”
  

• Paul B. McCray, M.D. (University of Iowa)
  “Gene Transfer to Airway Epithelia: Pre-clinical Studies in the Cystic Fibrosis Pig Model”