Plenary
Session II
8:00 a.m. 9:00 a.m.
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Keynote
Speaker:
Don C. Wiley, PhD |
Break
9:00 a.m. 9:30 a.m. |
Presidential
Symposium
9:30 a.m. 12:00 p.m. |
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Awards
Outstanding Contribution: Alain Fischer
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Presidential
lecture: |
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John Gurdon
Stem Cells
Mark
Groudine Transcription
Jeffrey
Bluestone Immunity
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Meet
the Professor Lunches (ticketed
session)
12:15 p.m. 1:15 p.m. |
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W. French
Anderson, MD In Utero Gene Therapy
Helen
Blau, PhD Stem Cells in Gene Therapy
Malcolm Brenner, MD Clinical Trials/Regulatory Affairs
Beverly
L. Davidson, PhD Developing Vectors Gene for Delivery to the Brain
Fred
Gage, PhD Neurobiology
Joseph Glorioso, PhD Herpes virus vectors
Katherine High, MD Gene Transfer for Hemophilia
Dusty Miller, PhD Should there be Limits to the Use of Components
From Pathogenic Viruses in Gene therapy vectors?
Richard Mulligan, PhD New Strategies and Technologies for Gene
Therapy
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Simultaneous
Oral Abstract Sessions
1:30 p.m. 4:00 p.m. |
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Workshops
1:30 p.m. 4:00 p.m. |
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Workshop
6: Lentiviral Vectors
Moderators:
Luigi Naldini, Susan Kingsman
Speakers:
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Garry
Nolan Lentiviruses: Delivery and delivered
Jakob
Reiser HIV 1-based lentiviral vectors: Tools for gene delivery and
gene discovery
Didier
Trono Lentivirus genevasion: from mechanistic exploration to
finalistic exploitation
Tal Kafri
Safe production of lentivirus vectors
Susan
Kingsman Lentivectors for the analysis and treatment of
neurodegenerative diseases
Luigi
Naldini Lentiviral vectors: Performance in experimental gene transfer
and potential for gene therapy
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Workshop
7: Adenoviral Vectors
Moderators:
Stefan
Kochanak, Jeffrey Chamberlain
Speakers:
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Art
Beaudet Progress with helper-dependent adenoviral vectors
Wei-Wei
Zhang Pre-clinical efficacy and safety studies of MiniAdFVIII for
treatment of hemophilia A
Tom
Wickham Progress towards genetically retargeting adenoviral vectors
Stefan
Kochanak "Gutless" adenoviral vectors: vector development,
production and applications
Jeffrey
Chamberlain Efficacy of gutted adenoviral vectors in normal and
diseased muscle
Dinko
Valerio Improved adenoviral vectors for gene therapy and vaccines
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Workshop
8: Novel Technologies
Moderator:
David
Russell
Speakers:
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John
Rossi Novel intracellular localization strategies for anti-HIV RNA
therapeutic agents
Perry
Hackett The "Sleeping Beauty" transposon system for gene
delivery and expression into mammalian chromosomes
David
Russell Homologous gene targeting by AAV vectors
Michele Calos Site-specific genomic integration
Chris
Contag Developing in vivo measurements of gene transfer
Yen Choo
Designer zinc finger transcription factors for specific gene
regulation
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Workshop
9: Non-Viral Vectors II
Moderator:
Jean-Paul
Behr
Speakers:
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Cyrus
Safinya DNA-lipid complexes and interactions with cells:
supramolecular assembly and gene delivery
Sun Won
Kim Tailor-made polymeric carriers for delivery of therapeutic
plasmid DNA
Alexander
Kabanov Polyplex-based gene delivery systems: current state and major
impediments
Yasufumi
Kaneda The device for the enhancement of transgene expression and its
use for cancer gene therapy
Jean-Paul
Behr Dimerizable cationic detergents
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Workshop
10: Fanconi Anemia
Moderators:
Marcus Grompe, Odille Cohen-Haguenauer
Speakers:
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Alan DAndrea
Interaction of the Fanconi Anemia proteins in a common pathway
Grover
Bagby Molecular pathogenesis of bone marrow failure in Fanconi Anemia
Francesco
Galimi Gene therapy of Fanconi Anemia by lentiviral vectors
Christopher
Walsh Fanconi Anemia: a model disorder for stem cell gene transfer
Odille
Cohen-Haguenauer Selective growth advantage and functional correction
of FA-A patients primary BM: comparing retrovirus SV40 and lentiviral
vectors
Marcus
Grompe Murine models of Fanconi anemia: pathophysiology and treatment
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Workshop
11: DNA Vaccines
Moderator:
Margaret
Liu
Speakers:
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Harriet
Robinson DNA priming and recombinant pox virus boosters, real
progress towards an HIV vaccine
Jeffrey
Ulmer DNA delivery technologies
Larry
Steinman Suppressive DNA vaccination for tolerance to autoimmune
disease
John
Gutheil Safety profile of plasmid DNA based therapy of cancer
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Exhibits
Open
4:00 p.m. 7:00 p.m. |
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Business
Meeting
4:15 p.m. 5:00 p.m. |
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Poster
Sessions
4:30 p.m. 7:00 p.m. |
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Corporate
Symposia
7:00 p.m. 10:00 p.m. |
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Targeted
Genetics Biology of Gene Delivery
Barrie J.
Carter, PhD, Session Chair
Room 606-609
Understanding
the biology of gene delivery systems is crucial to developing the most
efficient and specific systems suited for individual disease applications.
Increased insights into the entry and trafficking of gene delivery systems
and the fate of the delivered genome in the target cell nucleus enables two
approaches to developing appropriate delivery systems for individual
applications. First, understanding more clearly the biology of currently
available systems permits a more judicious choice of applications. Second,
this also forms the basis for development of advanced delivery systems with
increased efficiency, stability and targeting specificity. General concepts
of understanding trafficking pathways of gene delivery systems, how to
modify or enhance these and how to obtain targeting via direct delivery or
by modifying structure of gene delivery systems will be discussed by several
speakers using a viral system, AAV, and synthetic delivery system as
examples.
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Avigen
AAV Vectors in Clinical and Preclinical Research
Alan
McClelland, PhD, Session Chair
Room 611-614
Vectors
derived from AAV show promise in a number of animal model systems where
efficient gene transfer, long term gene expression and a lack of toxicity
have been observed. As a result there is considerable interest in the
clinical evaluation of AAV vectors, although at present only a small number
of clinical trials are underway. This symposium will highlight recent
developments in the clinical investigation of AAV vectors, and preclinical
studies with AAV vectors which may provide a basis for clinical trials in
the near future.
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Program
schedule for Saturday >
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