15th Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases
Focus Topic: Precision Medicine
November 16 - 18, 2016 - Sonoma, California

Program

Thursday, November 17, 2016

Keynote Speaker

• Bruce A. Sullenger, Ph.D. (Duke University)
  “Precisely Controlling Blood Coagulation with Antithrombotic Aptamers and Antidotes”

Session I 

• Brian D. Brown, Ph.D. (Mount Sinai School of Medicine)
  “Investigating T Cell and Stem Cell Interactions”

• Mark A. Kay, M.D., Ph.D. (Stanford University)
  “Novel rAAVs for Classical Gene Therapy and Genome Editing”

• Theresa M. Reineke, Ph.D. (University of Minnesota)
  “Polymer Vehicles for Cellular and In vivo Delivery of Nucleic Acids”

Session II   

• Larry W. Kwak, M.D., Ph.D. (City of Hope National Medical Center)
  “A Comprehensive Cancer Center’s Strategy for Precision Medicine”
  

• Eirini Papapetrou, M.D., Ph.D. (Mount Sinai School of Medicine)
  “Dissecting the Contributions of Cooperating Cancer Gene Mutations to Phenotype and Drug Susceptibility with Patient-Derived iPSCs”

• Edgardo Rodriguez, Ph.D. (University of Florida)
  “AAV-based Precision Approaches to Neurodegenerative Disease”

“Practical Strategies”: Genome Editing and Precision Medicine

• Matthew H. Porteus, M.D., Ph.D. (Stanford University)
  

Jacuzzi Family Vineyards

Mini-Workshop: “Precision Imaging ”

• Benjamin L. Franc, M.D. (University of California, San Francisco)
  “Precision Imaging in Precision Medicine”

Session III

• Jennifer E. Adair, Ph.D. (Fred Hutchinson Cancer Center)
  “Expanding the Availability of Cell and Gene Therapy to Address Global Health Burdens”
  
  
• Tippi C. MacKenzie, M.D. (University of California, San Francisco)
  “Precision Medicine for Maternal-Fetal Health”
  
  
• Joseph G. Hacia, Ph.D. (University of Southern California)
  “Quantitative Cell Image-based High Content Screening Identifies Small Molecules that Rescue Peroxisome Assembly Defects in Cells from Patients with Zellweger Spectrum Disorder”
  
  

Student/Fellow Presentations and Poster Session - Link to Information

Session IV

• Brigitte Gomperts, M.D. (University of California, Los Angeles)
  “Three-Dimensional Bioengineered Lung Tissue for Personalized Disease Modeling”
  
  
• Paul B. McCray, M.D. (University of Iowa)
  “Precision Medicine and Cystic Fibrosis: CRISPR/Cas9 Editing of Two Different Classes of CFTR Mutations”