2008 LYSOSOMAL DISEASES AND THE BRAIN CONFERENCE
a conference hosted by The Children's Gaucher Research Fund

2006 Conference Agenda
All Times are Pacific Standard Time

Friday, June 23, 2006
2:00 p.m. -
6:00 p.m. 		Conference Registration - Front Lobby of Hotel
6:00 p.m. -
9:00 p.m.		 Cocktail Reception and Dinner with Presentation: "A Tribute to William Krivit"
Saturday, June 24, 2006
7:00 a.m. -
8:15 a.m.		 Conference Registration/Full Breakfast
8:15 a.m. Welcome, Greg Macres (CGRF)
Session 1. Basic science - pathological mechanisms.
Chairs: Tony Futerman Ph.D. and Col. Kondi Wong M.D.
8:30 a.m. "Overview of pathological mechanisms in sphingolipid storage disease of the brain"
Tony Futerman Ph.D. (Weizmann Institute of Science)
8:50 a.m. "GM1-ganglioside as mediator of ER-stress-and mitochondria-elicited neuronal apoptosis"
Sandro D'Azzo Ph.D. (St. Jude Children's Research Hospital )
9:35 a.m. "TRP-ML channels and the function of intracellular organelles" Shmuel Muallem Ph.D. (Univ. of Texas Southwestern Medical Ctr )
10:20 a.m. Coffee Break
10:50 a.m. "A conditional knockout mouse model for Gaucher disease"
Francis Choy Ph.D. (University of Victoria)
Lorne Clarke M.D. (Univ. of British Columbia)
11:20 a.m. "Molecular findings in the brain of Gaucher disease mouse model based on expression analysis"
Sung-Chul Jung M.D. Ph.D. (Ewha Womans University)
11:50 a.m. "Novel mouse models for type 1 and type 2 Gaucher disease for use in the development of new therapies"
Stefan Karlsson M.D. Ph.D. (Lund University Hospital )
12:20 p.m. Lunch
1:05 p.m. "Pathogenic mechanisms that underlie Juvenile Batten Disease"
David A. Pearce Ph.D. (Univ. of Rochester School of Medicine)
1:50 p.m. "Impact of systemic inflammation on chronic neurodegenerative disease"
Hugh Perry MA DPhil FMedSci (Univ of Southampton)
2:35 p.m. Concluding remarks. "Developments since the 2004 conference?"
Greg Grabowski M.D. (Children's Hos. Research Foundation)
3:00 p.m. -
5:00 p.m.		 Poster Session
6:00 p.m. -
9:30 p.m.		 Cocktail Reception/Dinner
Presentation: "Gaucher Disease & Parkinsonism - Is There a Connection"
Ellen Sidransky M.D. (National Institutes of Health, NHGRI)
Matt Farrer Ph.D. (Mayo Clinic, Jacksonville Florida)
Panel Discussion: "Is There a Connection"
Sunday, June 25, 2006
7:00 a.m. -
8:15 a.m.		 Full Breakfast
8:15 a.m. Session 2. LSD therapy.
Chairs Roscoe Brady M.D. / Raphael Schiffmann M.D.
8:15 a.m. "Overview of therapeutic options"
Roscoe Brady M.D.
8:30 a.m. "Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors"
Beverly L. Davidson Ph.D. (Roy J. Carver Professor - University of Iowa)
9:15 a.m. "Intracranial delivery of CLN2 in LINCL"
Ronald G. Crystal M.D. (Weill Med. College of Cornell University)
10:00 a.m. Coffee Break
10:30 a.m. "Therapy and pathogenesis of metachromatic leukodystrophy: Studies in a mouse model"
Volkmar Gieselman M.D. (University of Bonn)
11:15 a.m. "Combined therapy for the treatment of globoid cell leukodystrophy"
David Wenger Ph.D. (Jefferson Medical College)
12:00 p.m Lunch
12:30 p.m. "Understanding and ameliorating misfolding diseases"
Jeffrey W. Kelley Ph.D. (The Scripps Research Institute, La Jolla, California)
1:15 p.m. "Cell- Fusion as a Tool for Generating a Neurological Disease Model 8"
Ehud Goldin Ph.D. (National Inst of Health - NHGRI )
2:00 p.m. "Stem cell research and the brain - relevance for LSDs"
Evan Snyder M.D. Ph.D. (Burnham Institute for Med. Research, La Jolla, Ca)
2:45 p.m. Concluding remarks. "Therapy - what's on the horizon?"
Raphael Schiffmann M.D.

Contact Us: Tel: 916.797.3700 • Fax: 916.797.3707 • e-mail: research@childrensgaucher.org
P.O. Box 2123 • Granite Bay, California 95746-2123