Plenary Session I       
8:30 a.m. – 9:30 a.m.

Symposium 1: Genetic Diseases I: Cardiovascular Disorders
Chair: Art Beaudet
Speakers:

Ronald Crystal – Enhancing tissue vascularity with localized expression of genes coding for angiogenic mediators
Ken Chien – Reversal of heart failure via AAV mediated cardiotropic delivery of a phospholamban inhibitory peptide
Victor Dzau – Title to be announced
Robert Rosenberg – New advances in heparan sulfates and their implication for gene therapy

Symposium 2: Cancer Gene Therapy I: Gene Based Strategies
Chair: Malcolm Brenner
Speakers:

Pervin Anklesaria - E1A Gene therapy for treatment of cancer
Evan Hersh – Non-viral vectors for gene delivery of immunotherapy, pre-clinical and clinical studies
Jack Roth – Strategies for tumor suppressor gene replacement in cancer
Savio Woo – Immuno-modulatory gene therapy for metastatic disease

Symposium 3: Musculoskeletal Disorders
Chair: Paul Robbins
Speakers:

Thomas Rando – Gene modification for point mutations and frame-shift deletions using RNA/DNA chimeric oligonucleotides
Jay Lieberman – Title to be announced
Chris Evans – Title to be announced
Jon Wolff – The intravascular delivery of naked DNA

Symposium 4: Hematopoiesis
Chair: Richard Mulligan
Speakers:

John Dick – Characterization of human stem cells
R. Keith Humphries – Genetic enhancement of hematopoietic stem cells for transplantation and gene therapy
Ihor Lemischka – Stem cells to genes and back
Catherine Verfaillie – Bone marrow multipotent stem cells

Workshop 1: AAV Vectors
Moderators: Robert Kotin, R. Jude Samulski
Speakers:

William Hauswirth – Gene therapy of retinal disease
Arun Srivastava – Role of cellular proteins in AAV-mediated transduction
Matthew Weitzman – The role of viral and cellular proteins in AAV replication
Mark Kay – Mechanisms of AAV transduction in vivo
Robert Kotin– Title to be announced
Jude Samulski – Parrovirus vectors pros and cons
Saswati Chatterjee – Analysis of rAAV transduced cells in an in vivo model

Workshop 2: Retroviral Vectors
Moderators: Ted Friedmann, Dusty Miller
Speakers:

David Emery – Use of chromatin insulators in oncoretrovirus vectors
Ralph Dornburg – Cell-type-specific gene delivery into neurons using SNV-derived retroviral vectors
Christopher Baum – Retroviral vector design for controlled selection of gene-modified hematopoietic cells in vivo
Dusty Miller – Jaagsiekte sheep retrovirus (JSRV) vectors for airway gene delivery
W. French Anderson – Matrix-targeted retroviral vectors for cancer
Theodore Friedmann – Biodistribution and attachment mechanisms of retrovirus and lentivirus vectors

Workshop 3: Non-Viral Vectors I: Naked DNA and Physical Methods
Moderators: Phil Felgner, Leaf Huang
Speakers:

Dexi Liu – Mechanism of hydrodynamics-based transfection
Ken Dev – In vivo gene therapy using pulsed electric fields
Robert Malone – Direct gene delivery: from serendipity to pulsed electrical fields
Louis Smith – Enhancement of plasmid delivery to skeletal muscle
Phil Felgner – Transcriptionally active PCR fragments for gene therapy, genomics and DNA vaccines
Leaf Huang – Gene transfer by naked DNA

Workshop 4: Cystic Fibrosis
Moderator: Michael Welsh
Speakers:

Terence Flotte – Long-term safety considerations with regard to the use of Recombinant Adeno-Associated Virus Vectors
Pam Zeitlin – AAV gene therapy for Cystic Fibrosis lung disease
Thomas Scanlin – Trafficking and nuclear localization of lactosylated polylysine/cDNA complexes in cystic fibrosis airway epitholial cells
Robert Walters - Binding of AAV5 to apical membrane sialic acid facilitates gene transfer to differentiated human airway epithelia

Workshop 5: Vascular Diseases
Moderators: Jeffrey Isner, H. Kirk Hammond
Speakers:

Shahin Rafii – Mobilization, expansion and recovery of transplantable endothelial and hematopoietic stem cells by adenoviral delivery of angiogenic factors
Roger Hajjar – Targeting signaling pathways in heart failure
Walter Koch – Targeting G protein signaling in pathological vascular proliferation
H. Kirk Hammond – Gene transfer and the treatment of n cardiovascular disease
Jeffrey Isner – Novel applications of VEGF gene transfer

Cell Genesys – Gene Therapy and the Tumor Vasculature
James G. McArthur PhD, Session Chair
Room 611-614

The growth of de novo blood vessels, or angiogenesis, is contributing factor in a multitude of diseases ranging from cancer, to ocular degeneration to rheumatoid arthritus. The identification of novel anti-angiogenic proteins has created several opportunities for creating gene therapeutics for these diseases. This session will explore the similarities and differences between diseased and normal vasculature and how these differences might be exploited in developing anti-angiogenic gene therapies. The speakers will discuss the interactions of the cells of the vasculature and tumors and how this understanding is leading to improvements in anti-angiogenic therapies. This will be a thought-provoking session on this exciting area of gene therapy.

Valentis – Gene Medicines for the 21st Century
Alain Rolland, PharMD, PhD, Session Chair
Room 606-609

Gene medicines (plasmid-based expression systems containing a therapeutic gene formulated with synthetic gene delivery systems) represent an attractive new class of biopharmaceutical products that use the human body to produce therapeutic proteins in a natural and controllable manner, thus potentially enabling an improved quality of medical treatment. This Symposium will bring together eminent scientists from academia and industry to present their latest findings on clinical development and potential future medical applications of gene medicines for the treatment of several serious diseases.

Program schedule for Friday >