Sunday
4:00 pm - 8:00 pm
Arrival and Check-in
6:00 pm - 7:00 pm
Dinner
7:30 pm - 7:40 pm
Introductory Comments by GRC Site Staff / Welcome from the GRC Chair
7:40 pm - 9:30 pm
Keynote Session: Lysosome Systems Biology
7:40 pm - 8:15 pm
"Dynamics and Diversity in Autophagy"
8:15 pm - 8:25 pm
Discussion
8:25 pm - 9:00 pm
"Exploiting the Lysosome's Unlimited Capabilities"
9:00 pm - 9:10 pm
Discussion
9:10 pm - 9:25 pm
"Lysosome Metabolomics: A Novel Approach to Elucidate the Molecular Function of Batten Disease Genes"
9:25 pm - 9:30 pm
Discussion
Monday
7:30 am - 8:30 am
Breakfast
8:30 am - 9:00 am
Group Photo
9:00 am - 12:30 pm
Lysosomal Biology and Disease Insights
9:00 am - 9:20 am
"The Role of Lysosomes in T-Cell Homeostasis"
9:20 am - 9:30 am
Discussion
9:30 am - 9:50 am
"GBA and Autophagic Cell Death"
9:50 am - 10:00 am
Discussion
10:00 am - 10:30 am
Coffee Break
10:30 am - 10:45 am
"A Role for Lysosomal Damage in Niemann-Pick Disease Type C Pathogenesis"
10:45 am - 10:50 am
Discussion
10:50 am - 11:10 am
"Transfer of Biomolecules by Lysosomes Through Tunneling Nanotubes"
11:10 am - 11:20 am
Discussion
11:20 am - 11:40 am
"Lysosomal Cholesterol and the Niemann-Pick C1 Signaling Complex"
11:40 am - 11:50 am
Discussion
11:50 am - 12:10 pm
"Microbial Hijacking of Lysosomal Disease Proteins"
12:10 pm - 12:20 pm
Discussion
12:20 pm - 12:30 pm
General Discussion
12:30 pm - 1:30 pm
Lunch
1:30 pm - 4:00 pm
Free Time
4:00 pm - 6:00 pm
Poster Session
6:00 pm - 7:00 pm
Dinner
7:30 pm - 9:30 pm
Lysosome Biology as a Guide to Understanding LSD Pathogenesis
7:30 pm - 7:50 pm
"TFEB and Lysosome Positioning"
7:50 pm - 8:00 pm
Discussion
8:00 pm - 8:20 pm
"The Highway to Lysosomal Maturation"
8:20 pm - 8:30 pm
Discussion
8:30 pm - 8:50 pm
"Krabbe Disease: The Psychosine Hypothesis and Acid Ceramidase as a Therapeutic Target"
8:50 pm - 9:00 pm
Discussion
9:00 pm - 9:15 pm
"Roles and Regulation of Lysosome Biogenesis and Function During Development"
9:15 pm - 9:20 pm
Discussion
9:20 pm - 9:30 pm
General Discussion
Tuesday
7:30 am - 8:30 am
Breakfast
9:00 am - 12:30 pm
9:00 am - 9:20 am
"Cell-Autonomous Expression of Lyosomal Hydrolase GALC: A New Model of Krabbe Disease"
9:20 am - 9:30 am
Discussion
9:30 am - 9:50 am
"AAV Therapies in Large Animal Models of LSDs"
9:50 am - 10:00 am
Discussion
10:00 am - 10:15 am
"Next-Generation Antibody-Guided Enzyme Replacement Therapy in Pompe Disease Mice"
10:15 am - 10:20 am
Discussion
10:20 am - 10:30 am
General Discussion
10:30 am - 11:00 am
Coffee Break
11:00 am - 11:20 am
11:20 am - 11:30 am
Discussion
11:30 am - 11:50 am
"Modulating Glycosphingolipids for GBA-Related Parkinson's Disease"
11:50 am - 12:00 pm
Discussion
12:00 pm - 12:15 pm
"Chronic Gaucher Disease Mouse Model"
12:15 pm - 12:20 pm
Discussion
12:20 pm - 12:30 pm
General Discussion
12:30 pm - 1:30 pm
Lunch
1:30 pm - 4:00 pm
Free Time
4:00 pm - 6:00 pm
Poster Session
6:00 pm - 7:00 pm
Dinner
7:30 pm - 9:30 pm
Emerging Tools to Study Lysosomal Diseases
7:30 pm - 7:50 pm
"Patching the Endo-Lysosome"
7:50 pm - 8:00 pm
Discussion
8:00 pm - 8:20 pm
"Cell Permeable Probes for Visualizing Lysosome-Organelle Interactions"
8:20 pm - 8:30 pm
Discussion
8:30 pm - 8:50 pm
"Imaging the Dynamics of Endocytic Traffic"
8:50 pm - 9:00 pm
Discussion
9:00 pm - 9:15 pm
"A pH-Correctable, DNA-Based Fluorescent Reporter for Lysosomal Calcium"
9:15 pm - 9:20 pm
Discussion
9:20 pm - 9:30 pm
General Discussion
Wednesday
7:30 am - 8:30 am
Breakfast
9:00 am - 12:30 pm
Mechanisms of Pathogenesis
9:00 am - 9:20 am
"Cathepsins and NPC Disease"
9:20 am - 9:30 am
Discussion
9:30 am - 9:50 am
"Prosaposin and Lysosomal Trafficking in FTLD"
9:50 am - 10:00 am
Discussion
10:00 am - 10:15 am
"Lysosomal Storage, Neurodegeneration and Albinism Due to Effects of a De Novo CLCN7 Mutation on Lysosomal Acidification"
10:15 am - 10:20 am
Discussion
10:20 am - 10:30 am
General Discussion
10:30 am - 11:00 am
Coffee Break
11:00 am - 11:20 am
"Cystinosis Through the Lens of a Synthetic Lethal Interaction"
11:20 am - 11:30 am
Discussion
11:30 am - 11:50 am
"Acid Ceramidase Deficiency and Farber Disease Models"
11:50 am - 12:00 pm
Discussion
12:00 pm - 12:15 pm
"Galactosylceramidase Is Required for Brainstem Development that Is Critical in the Pathogenesis of Krabbe Leukodystrophy"
12:15 pm - 12:20 pm
Discussion
12:20 pm - 12:30 pm
General Discussion
12:30 pm - 1:30 pm
Lunch
1:30 pm - 4:00 pm
Free Time
4:00 pm - 6:00 pm
Poster Session
6:00 pm - 7:00 pm
Dinner
7:00 pm - 7:30 pm
Business Meeting
Nominations for the Next Vice Chair; Fill in Conference Evaluation Forms; Discuss Future Site and Scheduling Preferences; Election of the Next Vice Chair
7:30 pm - 9:30 pm
Big Data and the Lysosome
7:30 pm - 7:50 pm
"Using Big Data to Advance Therapeutics"
7:50 pm - 8:00 pm
Discussion
8:00 pm - 8:20 pm
"Functional Genomics of the Lysosome in Parkinson's Disease"
8:20 pm - 8:30 pm
Discussion
8:30 pm - 8:45 pm
"Elucidating Cellular Autonomy in Peripheral Globoid Cell Leukodystrophy"
8:45 pm - 8:50 pm
Discussion
8:50 pm - 9:05 pm
"CNS-Directed AAV9 Gene Therapy for the Treatment of Canine Globoid Cell Leukodystrophy (Krabbe Disease)"
9:05 pm - 9:10 pm
Discussion
9:10 pm - 9:30 pm
General Discussion
Thursday
7:30 am - 8:30 am
Breakfast
9:00 am - 12:30 pm
Advances in Clinical Approaches for the LSDs
9:00 am - 9:20 am
"Gene Editing for Lysosomal Storage Diseases"
9:20 am - 9:30 am
Discussion
9:30 am - 9:50 am
"Taking It to the Clinic for Rare Diseases"
9:50 am - 10:00 am
Discussion
10:00 am - 10:15 am
"Bicistronic AAV Gene Therapy for Tay-Sachs Disease"
10:15 am - 10:20 am
Discussion
10:20 am - 10:30 am
General Discussion
10:30 am - 11:00 am
Coffee Break
11:00 am - 11:20 am
"Clinical Advances in NPC Therapy"
11:20 am - 11:30 am
Discussion
11:30 am - 11:50 am
"A Stepwise Mechanism for ER-to-Golgi Transport of Lysosomal Enzymes: Implications for Therapy"
11:50 am - 12:00 pm
Discussion
12:00 pm - 12:15 pm
"Acid Sphingomyelinase Deficiency Causes Variable Cerebellar Pathology that Can Be Ameliorated by N-Butyl-Deoxynojirimycin in the Mouse Model"
12:15 pm - 12:20 pm
Discussion
12:20 pm - 12:30 pm
General Discussion
12:30 pm - 1:30 pm
Lunch
1:30 pm - 4:00 pm
Free Time
4:00 pm - 6:00 pm
Poster Session
6:00 pm - 7:00 pm
Dinner
7:30 pm - 9:30 pm
Channeling Lysosomal Diseases
7:30 pm - 7:50 pm
"TRPML1 and Severe Combined Deficiency Diseases"
7:50 pm - 8:00 pm
Discussion
8:00 pm - 8:20 pm
"TRPML1 Manages Lysosome Size"
8:20 pm - 8:30 pm
Discussion
8:30 pm - 8:50 pm
"Targeting Endo-Lysosomal Two-Pore Channels in Health and Disease"
8:50 pm - 9:00 pm
Discussion
9:00 pm - 9:15 pm
"TRPML1 Links Lysosomal Calcium to Autophagosome Biogenesis"
9:15 pm - 9:20 pm
Discussion
9:20 pm - 9:30 pm
Closing Remarks
Friday
7:30 am - 8:30 am
Breakfast
9:00 am
Departure