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
Discussion Leaders: Beverly Davidson (Perelman School of Medicine, University of Pennsylvania / Children's Hospital of Philadelphia, USA) and Timothy Cox (University of Cambridge, United Kingdom)
7:40 pm - 8:15 pm
Ana Maria Cuervo (Albert Einstein College of Medicine, USA)
"Dynamics and Diversity in Autophagy"
8:15 pm - 8:25 pm
Discussion
8:25 pm - 9:00 pm
Andrea Ballabio (Telethon Institute of Genetics and Medicine (TIGEM), Italy)
"Exploiting the Lysosome's Unlimited Capabilities"
9:00 pm - 9:10 pm
Discussion
9:10 pm - 9:25 pm
Monther Abu-Remaileh (Whitehead Institute for Biomedical Research, MIT, USA)
"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
Discussion Leaders: Paul Saftig (Christian Albrechts University of Kiel, Germany) and Carmine Settembre (University of Naples / Telethon Institute of Genetics and Medicine (TIGEM), Italy)
9:00 am - 9:20 am
Chia-Lin Hsu (National Yang-Ming University, Taiwan)
"The Role of Lysosomes in T-Cell Homeostasis"
9:20 am - 9:30 am
Discussion
9:30 am - 9:50 am
Tony Futerman (Weizmann Institute of Science, Israel)
"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
Elaine Liu (University of Michigan, USA)
"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
Chiara Zurzolo (Institut Pasteur, France)
"Transfer of Biomolecules by Lysosomes Through Tunneling Nanotubes"
11:10 am - 11:20 am
Discussion
11:20 am - 11:40 am
Roberto Zoncu (University of California, Berkeley, USA)
"Lysosomal Cholesterol and the Niemann-Pick C1 Signaling Complex"
11:40 am - 11:50 am
Discussion
11:50 am - 12:10 pm
Frances Platt (University of Oxford, United Kingdom)
"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.
Organizers: Frances Platt (University of Oxford, United Kingdom) and Beverly Davidson (Perelman School of Medicine, University of Pennsylvania / Children's Hospital of Philadelphia, USA)
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
Discussion Leaders: Steven Walkley (Albert Einstein College of Medicine, USA) and Andrea Ballabio (Telethon Institute of Genetics and Medicine (TIGEM), Italy)
7:30 pm - 7:50 pm
Rosa Puertollano-Moro (National Institutes of Health, USA)
"TFEB and Lysosome Positioning"
7:50 pm - 8:00 pm
Discussion
8:00 pm - 8:20 pm
Shawn Ferguson (Yale University School of Medicine, USA)
"The Highway to Lysosomal Maturation"
8:20 pm - 8:30 pm
Discussion
8:30 pm - 8:50 pm
Mark Sands (Washington University School of Medicine in St. Louis, USA)
"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
Carmine Settembre (University of Naples / Telethon Institute of Genetics and Medicine (TIGEM), Italy)
"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
Reversing Pathogenesis
Discussion Leaders: Jeffrey Medin (Medical College of Wisconsin, USA) and Mark Sands (Washington University School of Medicine in St. Louis, USA)
9:00 am - 9:20 am
Christina Mikulka (Washington University School of Medicine, USA)
"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
Charles Vite (University of Pennsylvania, USA)
"AAV Therapies in Large Animal Models of LSDs"
9:50 am - 10:00 am
Discussion
10:00 am - 10:15 am
Maria Praggastis (Regeneron, USA)
"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
Rebecca Ahrens-Nicklas (The Children's Hospital of Philadelphia, USA)
"Correcting the NCLs"
11:20 am - 11:30 am
Discussion
11:30 am - 11:50 am
Lamya Shihabuddan (Sanofi, USA)
"Modulating Glycosphingolipids for GBA-Related Parkinson's Disease"
11:50 am - 12:00 pm
Discussion
12:00 pm - 12:15 pm
Yael Pewzner-Jung (Weizmann Institute of Science, Israel)
"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
Discussion Leaders: Thomas Braulke (University Medical Center Hamburg-Eppendorf, Germany) and Ana Maria Cuervo (Albert Einstein College of Medicine, USA)
7:30 pm - 7:50 pm
Christian Michael Grimm (Ludwig Maximilian University of Munich, Germany)
"Patching the Endo-Lysosome"
7:50 pm - 8:00 pm
Discussion
8:00 pm - 8:20 pm
Yu-Hui Zhang (Huazhong University of Science and Technology, China)
"Cell Permeable Probes for Visualizing Lysosome-Organelle Interactions"
8:20 pm - 8:30 pm
Discussion
8:30 pm - 8:50 pm
Tomas Kirchhausen (Harvard Medical School, USA)
"Imaging the Dynamics of Endocytic Traffic"
8:50 pm - 9:00 pm
Discussion
9:00 pm - 9:15 pm
Kasturi Chakraborty (University of Chicago, USA)
"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
Discussion Leaders: Alessandra D'Azzo (St. Jude Children's Research Hospital, USA) and Frances Platt (University of Oxford, United Kingdom)
9:00 am - 9:20 am
Andrew Lieberman (University of Michigan Medical School, USA)
"Cathepsins and NPC Disease"
9:20 am - 9:30 am
Discussion
9:30 am - 9:50 am
Fenghua Hu (Cornell University, USA)
"Prosaposin and Lysosomal Trafficking in FTLD"
9:50 am - 10:00 am
Discussion
10:00 am - 10:15 am
Elena-Raluca Nicoli (National Human Genome Research Institute, NIH, USA)
"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
Bruno Gasnier (Paris Descartes University, France)
"Cystinosis Through the Lens of a Synthetic Lethal Interaction"
11:20 am - 11:30 am
Discussion
11:30 am - 11:50 am
Jeffrey Medin (Medical College of Wisconsin, USA)
"Acid Ceramidase Deficiency and Farber Disease Models"
11:50 am - 12:00 pm
Discussion
12:00 pm - 12:15 pm
Daesung Shin (University at Buffalo, SUNY, USA)
"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
Discussion Leader: Kai Wang (Children's Hospital of Philadelphia / University of Pennsylvania, USA)
7:30 pm - 7:50 pm
Diego di Bernardo (Telethon Institute of Genetics and Medicine (TIGEM) / University of Naples Federico II, Italy)
"Using Big Data to Advance Therapeutics"
7:50 pm - 8:00 pm
Discussion
8:00 pm - 8:20 pm
Joshua Shulman (Baylor College of Medicine, USA)
"Functional Genomics of the Lysosome in Parkinson's Disease"
8:20 pm - 8:30 pm
Discussion
8:30 pm - 8:45 pm
Nadav Weinstock (University at Buffalo, USA)
"Elucidating Cellular Autonomy in Peripheral Globoid Cell Leukodystrophy"
8:45 pm - 8:50 pm
Discussion
8:50 pm - 9:05 pm
Allison Bradbury (University of Pennsylvania, USA)
"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
Discussion Leaders: Seng Cheng (Pfizer, USA) and William Gahl (National Institutes of Health, USA)
9:00 am - 9:20 am
Sagar Vaidya (Sangamo Therapeutics, Inc, USA)
"Gene Editing for Lysosomal Storage Diseases"
9:20 am - 9:30 am
Discussion
9:30 am - 9:50 am
Emil Kakkis (Ultragenyx, USA)
"Taking It to the Clinic for Rare Diseases"
9:50 am - 10:00 am
Discussion
10:00 am - 10:15 am
Toloo Taghian (University of Massachusetts Medical School, USA)
"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
Daniel Ory (Washington University in St. Louis, USA)
"Clinical Advances in NPC Therapy"
11:20 am - 11:30 am
Discussion
11:30 am - 11:50 am
Marco Sardiello (Baylor College of Medicine, USA)
"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
Jakub Sikora (Charles University in Prague, Czech Republic)
"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
Discussion Leaders: Susan Cotman (Massachusetts General Hospital, USA) and Tony Futerman (Weizmann Institute of Science, Israel)
7:30 pm - 7:50 pm
Xianping Dong (Dalhousie University, Canada)
"TRPML1 and Severe Combined Deficiency Diseases"
7:50 pm - 8:00 pm
Discussion
8:00 pm - 8:20 pm
Haoxing Xu (University of Michigan, USA)
"TRPML1 Manages Lysosome Size"
8:20 pm - 8:30 pm
Discussion
8:30 pm - 8:50 pm
Sandip Patel (University College London, United Kingdom)
"Targeting Endo-Lysosomal Two-Pore Channels in Health and Disease"
8:50 pm - 9:00 pm
Discussion
9:00 pm - 9:15 pm
Diego Medina Sanabria (Telethon Institute of Genetics and Medicine (TIGEM), Italy)
"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