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
3:00 pm - 4:00 pm
The GRC Power Hourâ„¢
The GRC Power Hourâ„¢ is designed to address challenges women face in science and issues of diversity and inclusion. The program supports the professional growth of all members of our communities by providing an open forum for discussion and mentoring.
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