| SUNDAY |
| 4:00 pm - 9:00 pm | Arrival and Check-in |
| 6:00 pm | Dinner |
| 7:30 pm - 7:40 pm | Welcome / Introductory Comments by GRC Site Staff |
| 7:40 pm - 9:10 pm | Immune Response to Viral Vectors |
| Discussion Leader: Paul Robbins (University of Pittsburgh) |
| 7:50 pm - 8:50 pm | Katherine High (The Children’s Hospital of Philadelphia) "Host Responses to Viral Vectors: The Final Frontier?" |
| 8:50 pm - 9:10 pm | Discussion |
| MONDAY |
| 7:30 am - 8:30 am | Breakfast |
| 9:00 am - 12:30 pm | Biodistribution and Targeting |
| Discussion Leader: John Engelhardt (The University of Iowa) |
| 9:00 am - 9:25 am | R. Jude Samulski (The University of North Carolina) "Clinical Update from Gene Therapy Trial for DMD" |
| 9:25 am - 9:35 am | Discussion |
| 9:35 am - 10:05 am | Renata Pasqualini & Wadih Arap (The University of Texas) "Ligand-Directed Vascular Targeting and Molecular Imaging" |
| 10:05 am - 10:15 am | Discussion |
| 10:15 am | Group Photo |
| 10:45 am | Coffee Break |
| 11:15 am - 11:40 am | Richard Vile (The Mayo Clinic) "Immune Cells as Carriers of Oncolytic Viruses to Treat Metastatic Tumours" |
| 11:40 am - 11:50 am | Discussion |
| 11:50 am - 12:15 pm | David Schaffer (University of California at Berkeley) "Molecular Evolution of Novel Viral Gene Delivery Vehicles" |
| 12:15 pm - 12:25 pm | Discussion |
| 12:30 pm | Lunch |
| 1:30 pm - 4:00 pm | Free Time |
| 4:00 pm - 6:00 pm | Poster Session I |
| 6:00 pm | Dinner |
| 7:30 pm - 9:30 pm | Viral Entry and Nuclear Translocation |
| Discussion Leader: R. Jude Samulski (The University of North Carolina) |
| 7:30 pm - 7:50 pm | John Engelhardt (The University of Iowa) "Endosomal Processing of rAAV2" |
| 7:50 pm - 8:00 pm | Discussion |
| 8:00 pm - 8:20 pm | Arun Srivastava (The University of Florida) "The Next Generation of Recombinant AAV Vectors" |
| 8:20 pm - 8:30 pm | Discussion |
| 8:30 pm - 8:50 pm | Mark A. Kay (Stanford University School of Medicine) "Combining Gene Therapy and RNAi for treating human disease" |
| 8:50 pm - 9:00 pm | Discussion |
| 9:00 pm - 9:20 pm | Anthony Welch (National Cancer Institute) "Developing Viral Vectors for Phase I Cancer Trials: Manufacturing and Toxicology Challenges" |
| 9:20 pm - 9:30 pm | Discussion |
| TUESDAY |
| 7:30 am - 8:30 am | Breakfast |
| 9:00 am - 12:30 pm | Host Cell Response to Viral Infection |
| Discussion Leader: Joseph Glorioso (University of Pittsburgh) |
| 9:00 am - 9:25 am | Karen Mossman (McMaster University) "The Innate Immune Response to Enveloped Virus Vectors" |
| 9:25 am - 9:35 am | Discussion |
| 9:35 am - 10:00 am | Erik Falck-Pedersen (Weill Cornell Medical College) "Antigen Presenting Cell Activation by Adenovirus Vectors" |
| 10:00 am - 10:10 am | Discussion |
| 10:10 am - 10:35 am | Paola Grandi (University of Pittsburgh) "Innate Immune Responses Limit HSV Oncolysis" |
| 10:35 am - 10:45 am | Discussion |
| 10:45 am | Coffee Break |
| 11:10 am - 11:35 am | Jay Nelson (Oregon Health & Sciences University) "CMV encoded miRNAs" |
| 11:35 am - 11:45 am | Discussion |
| 11:45 am - 11:55 am | Jarrod Johnson (University of North Carolina) "Subcellular processing of AAV2 generates a population of proteolytically modified virions that accumulate in the nucleolus and retain infectivity" |
| 11:55 am - 12:05 pm | Discussion |
| 12:05 pm - 12:20 pm | Michael Holmes (Sangamo) "Controlling HIV in vivo - Establishment of HIV Resistant CD4 T Cells
by Engineered Zinc Finger Protein Nucleases" |
| 12:20 pm - 12:30 pm | Discussion |
| 12:30 pm | Lunch |
| 1:30 pm - 4:00 pm | Free Time |
| 4:00 pm - 6:00 pm | Poster Session II |
| 6:00 pm | Dinner |
| 7:30 pm - 9:30 pm | Innate Immune Response to Viral Infection |
| Discussion Leader: James Wilson (The University of Pennsylvania) |
|
7:30 pm - 7:50 pm | Daniel Muruve (University of Calgary) "The Innate Immune Response to DNA" |
| 7:50 pm - 8:00 pm | Discussion |
| 8:00 pm - 8:20 pm | Glen R. Nemerow (The Scripps Research Institute) "Neutralization of Adenovirus by Naturally Occurring Antimicrobial Peptides" |
| 8:20 pm - 8:30 pm | Discussion |
| 8:30 pm - 8:50 pm | Yiping Yang (Duke University Medical Center) "Innate Immune Response to Viral Vectors" |
| 8:50 pm - 9:00 pm | Discussion |
| 9:00 pm - 9:20 pm | Sander van Deventer (Amsterdam Molecular Therapuetics) "Immunogenicity of AAV gene therapy vectors" |
| 9:20 pm - 9:30 pm | Discussion |
| WEDNESDAY |
| 7:30 am - 8:30 am | Breakfast |
| 9:00 am - 12:30 pm | Adaptive Immune Response to Viral Infection |
| Discussion Leader: Luigi Naldini (Fondazione San Raffaele del Monte Tabor) |
| 9:00 am - 9:25 am | Andrea Amalfitano (Michigan State University) "Adenovirus Interactions with the Innate and Adaptive Arms of the Immune System" |
| 9:25 am - 9:35 am | Discussion |
| 9:35 am - 10:00 am | James Wilson (The University of Pennsylvania) "The Impact of T Cell Responses on the Safety and Efficacy of DNA Viral Vectors" |
| 10:00 am - 10:10 am | Discussion |
| 10:10 am - 10:35 am | Luca Guidotti (The Scripps Research Institute) "Platelets Mediate Adaptive Immune Responses to Viral Vectors" |
| 10:35 am - 10:45 am | Discussion |
| 10:45 am | Coffee Break |
| 11:10 am - 11:35 am | Pedro Lowenstein (Cedars-Sinai Medical Center) "The molecular and cell biology of T-cell mediated elimination of adenovirus transduced brain cells: implications for clinical gene therapy" |
| 11:35 am - 11:45 am | Discussion |
| 11:45 am - 12:05 pm | Abraham Scaria (Genzyme Corporation) "Adaptive immune responses" |
| 12:05 pm - 12:15 pm | Discussion |
| 12:15 pm - 12:25 pm | Ann Galy (Genethon) "Micro RNA 142.3p-regulated vectors enable long-term AAV1-mediated gene transfer in skeletal muscle through the active suppression of transgene-specific CD4 and CD8 T cell immune responses" |
| 12:25 pm - 12:30 pm | Discussion |
| 12:30 pm | Lunch |
| 1:30 pm - 6:00 pm | Free Time |
| 4:00 pm - 6:00 pm | Poster Session (if needed) |
| 6:00 pm | Dinner |
| 7:00 pm - 7:30 pm | Business Meeting |
| (Nominations for the next Vice Chair; Fill out Conference Evaluation Forms; Discuss future Site & Scheduling preferences; Election of the next Vice Chair) |
| 7:30 pm - 9:30 pm | Approaches to Blocking the Immune Response to Viral Infection |
| Discussion Leader: Malcolm Brenner (Texas Children’s Hospital) |
| 7:30 pm - 7:50pm | Luigi Naldini (Fondazione San Raffaele del Monte Tabor) "Targeting Gene Transfer to Enhance the Efficacy and Safety of Gene Therapy for Inherited Diseases and Cancer" |
| 7:50 pm - 8:00 pm | Discussion |
| 8:00 pm - 8:20 pm | Roland Herzog (The University of Florida) "Immune Tolerance Induction to Therapeutic Proteins by Heptatic Gene Transfer" |
| 8:20 pm - 8:30 pm | Discussion |
| 8:30 pm - 8:50 pm | Maria Grazia Roncarolo (San Raffaele Telethon Institute for Gene Therapy) "Role of tolerogenic cells to modulate immune response to transgenes" |
| 8:50 pm - 9:00 pm | Discussion |
| 9:00 pm - 9:20 pm | John Iacomini (Harvard Medical School) "Developing gene therapy approaches to induce immunological tolerance" |
| 9:20 pm - 9:30 pm | Discussion |
| THURSDAY |
| 7:30 am - 8:30 am | Breakfast |
| 9:00 am - 12:30 pm | Suppression of the Immune Response by Gene Transfer |
| Discussion Leader: Maria Grazia Roncarolo (Fondazione San Raffaele) |
| 9:00 am - 9:25 am | Paul Robbins (University of Pittsburgh) "Treatment of autoimmune diseases using immunosuppressive DC-Derived Exosomes" |
| 9:25 am - 9:35 am | Discussion |
| 9:35 am - 10:00 am | Thierry VandenDriessche (University of Leuven) "Viral transduction of antigen-presenting cells: a double-edged sword?" |
| 10:00 am - 10:10 am | Discussion |
| 10:10 am - 10:35 am | David Scott (The University of Maryland) "Gene Therapy for Autoimmunity and Hemophilia: Role of B Cells as
Tolerogenic Antigen Presenting Cells" |
| 10:35 am - 10:45 am | Discussion |
| 10:45 am | Coffee Break |
| 11:10 am - 11:35 am | Garrison Fathman (Stanford University Medical Center) "DC’s Expressing IL-4 to Prevent and Possibly Treat T1D in NOD Mice" |
| 11:35 am - 11:45 am | Discussion |
| 11:45 am - 12:10 pm | Barrie J. Carter, Ph.D (Targeted Genetics Corporation) "Intra-articular delivery of an AAV vector for inflammatory joint disease: Comparison of preclinical and clinical studies" |
| 12:10 pm - 12:20 pm | Discussion |
| 12:30 pm | Lunch |
| 1:30 pm - 6:00 pm | Free Time |
| 6:00 pm | Dinner |
| 7:30 pm - 9:30 pm | Stimulation of the Immune Response by Gene Transfer |
| Discussion Leader: Garrison Fathman (Stanford University Medical Center) |
| 7:30 pm - 7:50 pm | Carl June (The University of Pennsylvania) "Lentiviral Vectors with Antisense HIV Payloads: Hitting the Target?" |
| 7:50 pm - 8:00 pm | Discussion |
|
8:00 pm - 8:20 pm | Malcolm K. Brenner (Texas Children’s Hospital) "Gene Transfer to Harness the Immune Response Against Cancer" |
| 8:20 pm - 8:30 pm | Discussion |
| 8:30 pm - 8:50 pm | Glenn Dranoff (Dana-Farber Cancer Institute) "Balancing Tumor Immunity and Inflammatory Pathology" |
| 8:50 pm - 9:00 pm | Discussion |
| 9:00 pm - 9:20 pm | Marjorie Robert-Guroff (National Cancer Institute, NCI/NIH) "Replication-Competent Adenovirus Vectors as Vehicles for HIV Vaccine Delivery" |
| 9:20 pm - 9:30 pm | Discussion |
| FRIDAY |
| 7:30 am - 8:30 am | Breakfast |
| 9:00 am | Departure |