SATURDAY |
1:30 pm - 4:30 pm | Arrival and Check-in |
3:00 pm - 3:15 pm | Introductory Comments by GRC Site Staff / Welcome by the GRS Conference Chair |
3:15 pm - 4:00 pm | Disease Models |
| Discussion Leader: Christopher Wassif (NIH/NICHD) |
3:15 pm - 3:30 pm | Leigh Paton (University of Oxford)
"Novel ENU mouse model of Mucolipidosis II"
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3:30 pm - 3:38 pm | Discussion |
3:38 pm - 3:53 pm | Abdulfatah Alayoubi (University of Toronto)
"Gene Therapy of Farber Disease with Mechanistic Implications"
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3:53 pm - 4:00 pm | Discussion |
4:00 pm - 5:30 pm | Poster Session I |
5:30 pm - 7:30 pm | Lysosomal Disease Pathogenesis and Therapies |
| Discussion Leader: Anatalia Labilloy (University of Pittsburgh) |
5:30 pm - 5:50 pm | Allison Bradbury (Auburn University)
"Dramatic Therapeutic Response of Intracranial AAV Gene Therapy in Feline GM2 Gangliosidosis"
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5:50 pm - 6:00 pm | Discussion |
6:00 pm - 6:20 pm | Cristin Davidson (Albert Einstein College of Medicine)
"Efficacy of different cyclodextrins in the treatment of a murine model of Niemann-Pick type C disease"
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6:20 pm - 6:30 pm | Discussion |
6:30 pm - 6:50 pm | Tamar Farfel-Becker (Weizmann Institute of Science)
"The pathogenesis of neuronopathic Gaucher disease: glucosylceramide accumulation, neuroinflammation and neurodegeneration"
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6:50 pm - 7:00 pm | Discussion |
7:00 pm - 7:20 pm | Oscar Chen (University of Oxford)
"Defective transition metal ion homeostasis and hematological abnormalities in lysosomal storage disease animals and their clinical implications"
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7:20 pm - 7:30 pm | Discussion |
8:00 pm | Dinner |
SUNDAY |
7:30 am - 8:30 am | Breakfast |
9:00 am - 11:00 am | Lysosomes in Health and Disease |
| Discussion Leader: Heather Gray-Edwards (Auburn University) |
9:00 am - 9:20 am | Martina Malnar (Rudjer Boskovic Institute)
"Cholesterol levels modulate trafficking of APP and BACE1 in Niemann-Pick type C cells"
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9:20 am - 9:30 am | Discussion |
9:30 am - 9:50 am | Katrijn Coen (Catholic University of Leuven)
"Lysosomal calcium homeostasis defects, not proton pump defects, cause endo-lysosomal dysfunction in PSEN-deficient cells"
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9:50 am - 10:00 am | Discussion |
10:00 am - 10:20 am | Luis Tecedor (University of Iowa)
"Membrane microdomain properties, caveolin-1 transport and caveolae-related functions are impaired in brain endothelial lacking the Batten disease protein, CLN3"
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10:20 am - 10:30 am | Discussion |
10:30 am - 10:50 am | Ruth Milkereit (University of Toronto)
"Investigating the trafficking of LAPTM4 proteins"
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10:50 am - 11:00 am | Discussion |
11:00 am - 12:30 pm | Poster Session II |
| Coffee will be served in the poster area from 11:00 am - 11:30 am |
12:30 pm | Lunch |
1:30 pm - 2:30 pm | Career Progression Workshop |
| Discussion Leader: Andres Klein (Weizmann Institute of Science) |
| Panelists: |
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- Frances Platt (University of Oxford)
- Steven U. Walkley (Albert Einstein College of Medicine)
- Tony Futerman (Weizmann Institute of Science)
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2:30 pm - 3:00 pm | Evaluation Period |
| Fill out GRS Evaluation Forms |
3:00 pm | Gordon Research Seminar ends. For those attending the associated Gordon Research Conference, please check in at the GRC Office beginning at 4:00 pm. |