SCIENTIFIC PROGRAM
Last update, May 12, 2009
PDF-Version of this programm.
| Wednesday, June 3 | |
|---|---|
| 17:00-19:00 | Registration (open throughout the meeting) |
| 19:00 | Opening remarks and welcome (A. Kohlschütter) |
| Public Evening Lecture | |
| Thomas Jentsch (Berlin): | |
| Chloride transport in endosomes and lysosomes : role in lysosomal storage and neurodegeneration | |
| 20:00-22:00 | Get-together |
| Thursday, June 4 | |
Sessions I and II | |
| Soluble NCL proteins (CLN1, CLN2, CLN5, CLN10/Cath D) Chair: Jonathan D. Cooper, David A. Pearce | |
| 09:00-09:20 | Alfried Kohlschütter (Hamburg): |
| Clinical overview | |
| 09:20-09:45 | Sandra L. Hofmann (Dallas): |
| Metabolic profiling of infantile neuronal ceroid lipofuscinosis | |
| 09:45-10:10 | Robert Steinfeld (Göttingen) |
| Structural features of tripeptidylpeptidase 1 | |
| Speakers selected from abstracts | |
| 10:10-10:25 | Shannon L. Macauley (Saint Louis): |
| The role of astrocyte activation in infantile neuronal ceroid lipofuscinosis | |
| 10:25-10:50 | Karen D. Parfitt (Claremont): |
| Synaptic transmission is altered in palmitoyl-protein thioesterase 1 (PPT-1) Drosophila mutants | |
| 10:45-11:15 | Break |
| Soluble NCL proteins (continued) | |
| 11:15-11:40 | Aija Kyttälä (Helsinki): |
| Molecular characterization of CLN5 interactions | |
| 11:40-12:05 | Jaana Tyynelä (Helsinki) |
| Cathepsin D and alpha-synuclein processing, aggregation, and toxicity in vivo | |
| Speakers selected from abstracts | |
| 12:05-12:20 | Mia-Lisa Schmiedt (Helsinki): |
| The neuronal ceroid lipofuscinosis protein CLN5: New insights into cellular processing and transport | |
| 12:20-12:35 | Matthew C. Micsenyi (Bronx): |
| Pathological p62/sequestosome 1 in the CNS of classical late-infantile neuronal ceroid lipofuscinosis | |
| 12:30-14:15 | Lunch |
| Poster viewing | |
Sessions III and IV | |
| Membrane proteins, channels, transporters (CLN3, CLN6, CLN7, CLN8, ClCs) Chair: Sara Mole, Thomas Braulke | |
| 14.15-14:35 | Jonathan W. Mink (Rochester): |
| Clinical overview | |
| 14:35-15:00 | David A. Pearce (Rochester) |
| Pathological consequences of mutations in CLN3 | |
| 15:00-15:25 | Sara E. Mole (London): |
| Using fission yeast S. pombe to understand CLN3 function | |
| Speakers selected from abstracts | |
| 15:25-15:40 | Rachel Kama (Rehovot): |
| Yeast Btn1 is a Golgi protein involved in late endosome-Golgi transport | |
| 15:40-15:55 | Richard Tuxworth (London): |
| Synapse retraction and footprinting in a drosophila system for studying CLN3 function | |
| 16 :00 | Break |
| Membrane proteins/channels, transporters (continued) | |
| 16:30-16:55 | Paul Saftig (Kiel): |
| Old and new lysosomal membrane proteins: Insight into their function | |
| Speakers selected from abstracts | |
| 16:55-17:10 | Bernd Schröder (Kiel): |
| Novel lysosomal membrane proteins: molecular characterisation of “transmembrane protein 192” (TMEM192) and “Disrupted in renal carcinoma 2” (DIRC2) | |
| 17:10-17:25 | Bruno Gasnier (Paris): |
| Intracellular localization and biochemical characterization of CLN7, a putative lysosomal transporter associated with late-infantile ceroid lipofuscinosis | |
Session V | |
| Comparative studies in different NCL diseases Chair: Jaana Tyynelä, Anu Jalanko | |
| 17:25-17:50 | Jonathan D. Cooper (London): |
| The same, but different: Comparative morphological studies in the NCLs | |
| 17:50-18:15 | Sunita Biswas (Boston): |
| A genetics directed approach to NCL therapeutics | |
| Speakers selected from abstracts: | |
| 18:15-18:30 | Claire Russell (London): |
| A variety of zebrafish NCL models display similar NCL-like phenotypes | |
| 19:30 | Dinner: Institute of Zoology, Martin-Luther-King-Platz 3 |
| Friday, June 5 | |
Session VI | |
| Retinal degeneration Chair: Hans H. Goebel, Klaus Rüther | |
| 09:00-09:25 | Klaus Rüther (Berlin): |
| Retinal function in NCL patients and mouse models | |
| 09:25-09:50 | Robin R. Ali (London): |
| Gene therapy for inherited retinal degeneration: from mouse to man | |
| Speakers selected from abstracts: | |
| 09:50-10:05 | Sarah N.R. Pressey (London): |
| Evaluating proposed NCL candidate genes: evidence for distinct neuropathological phenotypes of Clcn6-/- and Clcn7-/- mice | |
| 10:05-10:20 | Colleen S. Stein (Iowa City): |
| CLN3 modulates the function of an ion channel | |
| 10:20-10:35 | Luis Tecedor (Iowa City): |
| A study of CLN3 function in mouse brain endothelial cells | |
| 10:45 | Break |
Session VII | |
| Autophagy and other pathomechanismsChair: Paul Saftig, Stephan Storch | |
| 11:00-11:25 | Yasuo Uchiyama (Tokyo): |
| Involvement of autophagy in pathogenesis of neuronal ceroid lipofuscinosis | |
| Speakers selected from abstracts: | |
| 11:25-11:40 | Alessandro Simonati (Verona): |
| Features of autophagy in CLN3 and CLN6 fibroblasts both in vivo and in vitro | |
| 11:40-11:55 | Giovanna Galliciotti (Hamburg): |
| Mechanisms of neurodegeneration in late infantile neuronal ceroid lipofuscinosis CLN6 | |
| 11:55-12:10 | Jill M. Weimer (Rochester): |
| Function role of CLN6-CRMP2 protein interaction in nclf model of vliNCL | |
| 12:10-12:25 | Mervi Kuronen (Helsinki): |
| Galactolipid deficiency is a hallmark of early disease pathogenesis in the neuronal ceroid lipofuscinosis 8 mouse, Cln8[mnd] | |
| 13:00-14:30 | Lunch |
| Poster viewing | |
| Social afternoon | |
| 15:00 | Boat trip to Alster lake, city, harbor Sightseeings |
| 19:00 | Banquet: Restaurant River-Kasematten, St. Pauli Fischmarkt 28-32 |
| Saturday, June 6 | |
Session VIII - With participation of NCL families | |
| Experimental NCL therapies Chair: Rose-Mary Boustany, Robert Steinfeld | |
| 09:00-09:20 | Michael Chang (Iowa City): |
| Novel therapies for the NCLs | |
| 09:20-09:40 | Stephen Huhn (Palo Alto): |
| CNS transplantation of purified human neural stem cells in infantile and late-infantile neuronal ceroid lipofuscinoses: Results of the phase 1 trial | |
| 09:40-10:05 | Ronald G. Crystal (New York): |
| Adenoassociated virus-mediated gene therapy for the CNS manifestations of lysosomal storage disorders | |
| 10:05-10:25 | Glyn Dawson (Chicago): |
| Neuron-specific chemical chaperone delivery in INCL | |
| Speakers selected from abstracts: | |
| 10:25-10:40 | Michael Chang (Iowa City): |
| CNS-directed enzyme therapy | |
| 10:40-10:55 | Angela Schulz (Hamburg): |
| The German NCL registry – developing a tool for the evaluation of experimental therapies | |
| 11:00 | Break |
Session IX - With participation of NCL families | |
| Current medical management Chair: Ruth Williams, Alfried Kohlschütter | |
| 11:30-11:50 | Andreas Richterich (Hamburg): |
| Psychiatric symptoms and palliative psychopharmacology in children and adolescents with NCL | |
| Speakers selected from abstracts: | |
| 11:50-12:05 | Heather R. Adams (Rochester): |
| Further validation of a clinical rating scale for Batten disease | |
| 12:00-12:30 | Summaries of Sessions I-VII |
| 12:30 | Late breaking newsTwo presentations |
| 13:15 | Poster awards, best scientific presentation award, young investigator award |
| 13:30 | Lunch |
| Farewell | |
| Workshops and discussion groups | |
| 14:30-16:00 | Workshop I – Rare NCL Gene Identification Consortium |
| Sara Mole, London | |
| 16:00-17:30 | Workshop II – International NCL Patient Registry |
| Angela Schulz, Hamburg | |
| 14:30-17:30 | NCL family representatives: Discussion group |