13th Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases
Focus Topic: Genomic Editing
November 19 - 21, 2014 - Sonoma, California

Program

Wednesday, November 19, 2014 - Welcoming Reception

Thursday, November 20, 2014

Keynote Speaker

• Matthew H. Porteus, M.D., Ph.D. (Stanford University)
  “Genome Editing: The Time is Ripe for Translation”

Session I 

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

• Aravind Asokan, Ph.D. (University of North Carolina at Chapel Hill)
  “New AAV Tools for the Gene Editing Toolbox”

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

Session II   

• Charles A. Gersbach, Ph.D. (Duke University)
  “Custom Redesign of the Human Genome and Epigenome for Gene Therapy and Regenerative Medicine”
  

• Danwei Huangfu, Ph.D. (Sloan-Kettering Institute)
  “hESC-based Functional Human Genetics”

• David J. Segal, Ph.D. (UC Davis)
  “Genome Engineering at the Dawn of the Golden Age”

• Michele P. Calos, Ph.D. (Stanford University)
  “Genome Engineering in a Stem Cell Therapy for Muscular Dystrophy”

“Practical Strategies”: Targeted Editing Techiniques

• J. Keith Joung, M.D., Ph.D. (Harvard Medical School)
  “Targeted Genome and Epigenome Editing Using Engineered CRISPR-Cas and TALEN Technologies”

Jacuzzi Family Vineyards

Mini-Workshop: “Cell Engineering and Immunotherapy”

• Andrew M. Scharenberg, M.D. (University of Washington)

Session III

• Paula M. Cannon, Ph.D. (University of Southern California)
  “Targeted Gene Editing for HIV/AIDS”

• Leslie S. Kean, M.D., Ph.D. (Seattle Children's Research Institute)
  “Dissecting the Mechanisms of the Allo-Effect: During Transplantation, Graft-versus-host Disease and HIV Eradication”
  
  
• Donald B. Kohn, M.D. (UCLA)
  “Gene Therapy Using Hematopoietic Stem Cells”

Student/Fellow Presentations and Poster Session - Link to Information