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

2006 CONFERENCE FEATURED SPEAKERS
Speakers are listed in alphabetical order, by last name.

Roscoe Brady, M.D.
Scientist Emeritus, Developmental and Metabolic Neurology Branch - National Institutes Of Health - Bethesda, Maryland
Email: robrady@codon.nih.gov
Talk: "Overview of therapeutic options"

Francis Y.M. Choy, Ph.D.
Professor, Centre for Biomedical Research, Department of
Biology, University of Victoria, Canada
Email: fchoy@uvic.ca
Talk: "A conditional knockout mouse model for Gaucher disease"

Lorne A. Clarke, M.D.
Associate Professor Medical Genetics, University of British
Columbia, Children's and Women's Health Center of BC, Canada
Email: lclarke@cw.bc.ca
Talk: "A conditional knock out of murine GBA"

Ronald G. Crystal, M.D.
Professor and Chairman, Department of Genetic Medicine, Weill Medical College of Cornell University, New York, NY
Email: lad2014@med.cornell.edu
Talk: "Intracranial delivery of CLN2 in LINCL"

Beverly L. Davidson, Ph.D.,
Roy J Carver
Professor Internal Medicine, Neurology, Physiology & Biophysics, Roy J. and Lucille J. Carver College of Medicine, University of Iowa
Email: beverly-davidson@uiowa.edu
Talk: "Functional correction of CNS phenotypes in a
lysosomal storage disease model using adeno-associated virus type 4 vectors"

Alessandra d'Azzo, Ph.D.
Member and Endowed Chair, Department of Genetics , St. Jude Children's Research Hospital, Memphis, Tennessee
Email: sandra.dazzo@STJUDE.ORG
Talk: "GM1-ganglioside as mediator of ER-stress-and
mitochondria-elicited neuronal apoptosis"

Matt Farrer PhD.,
Director, Molecular Genetics Laboratory and Core,
Morris K. Udall Parkinson's Disease Research Center of Excellence,
Birdsall Bldg.,
Mayo Clinic, Department of Neuroscience, Jacksonville, FL
Email: farrer.matthew@mayo.edu Talk: "Parkinson’s Disease"

Tony Futerman Ph.D.
Professor, Department of Biological Chemistry, Weizmann
Institute of Science, Israel
Email: tony.futerman@weizmann.ac.il
Talk: "Overview of pathological mechanisms in
sphingolipid storage disease of the brain"

Volkmar Gieselmann M.D.
Professor , Institute of Physiological Chemistry, University of Bonn, Germany
Email: gieselmann@institut.physiochem.uni-bonn.de
Talk: "Therapy and pathogenesis of metachromatic
leukodystrophy: Studies in a mouse model"

Ehud Goldin, Ph.D.
Scientist, National Institutes of Health, NHGRI, Bethesda,
Maryland
Email: eg16x@nih.gov
Talk: "Cell- Fusion as a Tool for Generating a Neurological Disease Model 8"

Gregory Grabowski M.D.
Director, Division and Program in Human Genetics, Children's Hospital Research Foundation of Cincinnati, University of
Cincinnati, Ohio
Email : greg.grabowski@cchmc.org
Talk: "What have we learned about pathological
mechanisms since the last LSD and the brain conference?"

Sung-Chul Jung,M.D.,Ph.D.
Associate Professor, Department of Biochemistry, College of Medicine, Ewha Womans UniversityS. Korea
Email: jungsc@ewha.ac.kr
Talk: "Molecular findings in the brain of Gaucher disease mouse model based on expression analysis"

Stefan Karlsson Ph.D., M.D.,
Professor of Molecular Medicine, Lund Strategic Center for Stem Cell Biology and Cell Therapy, Lund University Hospital, Sweden
E-mail: Stefan.Karlsson@molmed.lu.se
Talk: "Novel mouse models for type 1 and type 2 Gaucher disease for use in the development of new therapies"

Jeffrey W. Kelly Ph.D.
Lita Annenberg Hazen Professor of Chemistry, Dean of Graduate Studies; Vice President Academic Affairs, The Scripps Research
Institute, La Jolla, California
Email: wihae@scripps.edu
Talk: "Understanding and ameliorating misfolding diseases"

Shmuel Muallem, Ph.D.
Professor of Physiology, Department of Physiology, University of Texas Southwestern Medical Center -
Email: shmuel.muallem@utsouthwestern.edu
Talk: "TRP-ML channels and the function of intracellular
organelles"

David A. Pearce, Ph.D.
Associate Professor, Center for Aging and Developmental Biology, University of Rochester School of Medicine, New York -
Email: David_Pearce@urmc.rochester.edu
Talk: "Pathogenic mechanisms that underlie Juvenile Batten Disease"

V. Hugh Perry, MA DPhil, FMedSci
Director of Research, Professor of Experimental Neuropathology, CNS Inflammation Group, University of Southampton, UK -
Email: vhp@soton.ac.uk
Talk: "Impact of systemic inflammation on chronic neurodegenerative disease"

Raphael Schiffmann M.D.
Chief, Clinical Investigations, National Institutes of Health, DMNB, Bethesda, Maryland -
Email: schiffmr@ninds.nih.gov
Talk: "Therapy - what's on the horizon?"

Ellen Sidransky M.D.
Chief, Section on Molecular Neurogenetics, MGB, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland -
Email: sidranse@irp.nimh.nih.gov
Talk: "Gaucher Disease and Parkinsonism"

Evan Snyder M.D., PhD.
Professor, Director, Stem Cell Program, Burnham Institute for
Medical Research, La Jolla, California -
Email: shyde@burnham.org
Talk: "Stem cell research and the brain - relevance for LSDs"

David A. Wenger, Ph.D.
Jefferson Medical College, Department of Neurology, Philadelphia, Pennsylvania
Email: david.wenger@jefferson.edu
Talk: "Combined therapy for the treatment of globoid cell leukodystrophy"

Col. Kondi Wong M.D. USAF MC
Chief, Division of Neuropathology, Wilford Hall Medical Center,
San Antonio, Texas -
Email: kondi.wong@lackland.af.mil
Talk: "Overview of pathological mechanisms in sphingolipid storage disease of the brain"

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