Chair
Barrie J. Carter, PhD

Speaker
Hal E. Broxmeyer, PhD
Understanding and Enhancing Stem/Progenitor Cell Function and Engraftment

Chair
R. Jude Samulski, PhD

Speakers

Steven A. Rosenberg, MD, PhD
Cancer Immunotherapy Using Genetically Modified T Cells

Ian MacLachlan, PhD
A Phase I Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Lipid Nanoparticle ApoB siRNA (ApoB-SNALP) in Hypercholesterolemic Subjects

Bill Hauswirth, PhD
Leber Congenital Amaurosis (LCA) Gene Therapy Clinical Trial

R. Jude Samulski, PhD
Designing AAV Vectors for Translational Studies: Update on DMD Phase 1 Trial

Chair
Carolyn Lutzko, PhD

Speakers
Pamela Itkin-Ansari, PhD
Development of Cell Based Therapies for Diabetes

Markus Grompe, MD
Hepatobiliary Stem/Progenitor Cells

Carolyn Lutzko, PhD
Developing Stem Cell Based Therapies for Lung Disease: Bringing Genes to Lung Stem Cells and Stem Cells to the Lung

Mervin C. Yoder, MD
Are Endothelial Progenitor Cells Derived from the Endothelial Lining of Blood Vessels?

Chair
Charles P. Venditti, MD, PhD

Speakers
Nathalie Cartier-Lacave, MD
Gene Therapy for Adrenoleukodystrophy

Alessandra Biffi, MD
HSC Gene Therapy for Metachromatic and Globoid Leukodystrophies: The Next Trials for Inherited Neurological Disorders

Ronald D.G. McKay, PhD

Robert D. Steiner, MD
CNS Transplantation of Neural Stem Cells in Neuronal Ceroid Lipofuscinoses: Phase I Trial Results

Chair
Roberto Cattaneo, PhD

Speakers
Lynn W. Enquist, PhD
Response of PNS Neurons to Herpesvirus Infection

Jeffrey Bergelson, MD
Cell Biology of Virus Entry

Bernard Moss, MD, PhD
Poxvirus Replication and Pathogenesis: Implications for Vector and Vaccine Development

Roberto Cattaneo, PhD
The Force Behind Targeting: How the Receptor-Triggered Membrane Fusion Mechanism of Measles Virus Works

Co-Chairs
Paula Clemens, MD
Alan J. Nixon, DVM, MS

Speakers
Edward M. Schwarz, PhD
Revitalizing Structural Allografts

Steven C. Ghivizzani, PhD
Gene Therapy for Achondroplasia

Joel R. Chamberlain, PhD
Systemic Delivery of RNAi Therapeutics for Treatment of Myotonic Dystrophy

Carmen Bertoni, PhD
Enhanced Cell-Mediated Regenerative Approaches to DMD Using RNA Interference

Chair
Bruce Bunnell, PhD

Speakers
Daniel Weiss, MD, PhD
Recent Advances in Gene and Cell Therapies for Lung Diseases

Paul B. McCray, Jr., MD
Lentiviral Vector Development for Prevention and Treatment of Cystic Fibrosis Lung Disease

Blair Roszell, MS
Two Step Differentiation of Mouse Embryonic Stem Cells to Definitive Endoderm and Lung Epithelial Cells

Peter I. Lelkes, PhD
Biomedical Engineering in the Lung

John F. Engelhardt, PhD
New Developments in Animal Models of Cystic Fibrosis

Speakers
Cheryl L. McDonald, MD
Introductory Remarks

Daniel Rosenblum, MD
The National Gene Vector Biorepository and Coordinating Center (NGVBCC) and
How the Clinical and Translational Science Awards (CTSA) Program can Support Cell and Gene Therapy Investigators

Catherine McKeon, PhD
The NIDDK Molecular Therapy Centers and Cell Therapy Program

David G. Badman, PhD
The NIH Rapid Access to Interventional Development Program (NIH-RAID)

Roy S. Wu, PhD
The NCI Experimental Therapeutics Program (NExT) and the Chemical Biology Consortium (CBC)

Danilo A. Tagle, PhD
The NINDS Translational Research Program

Lisbeth Welniak, PhD
Production Assistance for Cellular Therapies (PACT)

Sonia Skarlatos, PhD
The Center for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases
The NHLBI Gene Therapy Resource Program (GTRP)
The Science Moving Towards Research Translation and Therapy (SMARTT) Program

Ying Huang, PhD
Communication with FDA/CBER/OCTGT (Office of Cellular, Tissue and Gene Therapies)

Open Microphone Question and Answer Period

Speakers
Jeffrey Chamberlain, PhD
Paula Clemens, MD

Speakers
Timothy P. Cripe, MD, PhD
Samuel D. Rabkin, PhD

Chair
Christian L. Lorson, PhD

Speakers
Christian L. Lorson, PhD
Introduction: Perspectives on SMA Therapeutics

C. Frank Bennett, PhD
RNA Targeting Therapeutics for the Treatment of Motor Neuron Diseases

Marco Passini, PhD
Gene and Antisense Therapies in SMA Mice

Brian K. Kaspar, PhD
Rescue of a Spinal Muscular Atrophy Mouse Model by Early Postnatal Delivery of SMN with scAAV9 and Clinical Development Plans

Petra Kaufman, MD
SMA Clinical Trials -  NINDS Perspective