Saturday
1:00 pm - 5:00 pm
Arrival and Check-in
3:00 pm - 3:15 pm
Introductory Comments by GRC Staff / Welcome and Introduction from the Chairs
3:15 pm - 4:00 pm
Keynote Session: A Personal Journey Through Lysosomal Diseases - Career in Science and How to Become a Successful Researcher
Discussion Leader: Indranil Basak (University of Otago, New Zealand)
3:15 pm - 3:20 pm
Introduction by Discussion Leader
3:20 pm - 3:55 pm
Frances Platt (University of Oxford, United Kingdom)
"From Caterpillars to Lysosomal Diseases, a Scientific Journey"
3:55 pm - 4:00 pm
Discussion
4:00 pm - 5:30 pm
Poster Session
5:30 pm - 7:30 pm
Pathological Mechanisms of Lysosomal Diseases
Discussion Leader: Claudia Parra-Ruiz (Pontificia Universidad Católica de Chile, Chile)
5:30 pm - 5:45 pm
Neuza Domingues (MIA-Portugal - Multidisciplinary Institute of Ageing - University of Coimbra, Portugal)
"Unveiling the Molecular Mechanisms of Batten Disease in the Absence of CLN3"
5:45 pm - 5:50 pm
Discussion
5:50 pm - 6:05 pm
Yuzhe Weng (University of Oxford, United Kingdom)
"The Role of the NPC Pathway in Salmonella Infection"
6:05 pm - 6:10 pm
Discussion
6:10 pm - 6:25 pm
Isidoro Cobo (University California San Diego, United States)
"Molecular Mechanisms of Increased Gout in Patients With Clonal Hematopoiesis by DNMT3A Mutations and Gout"
6:25 pm - 6:30 pm
Discussion
6:30 pm - 6:45 pm
Leigh Fremuth (St. Jude Children's Research Hospital, United States)
"Neu1 Modulates Trem2 Signaling and Microglial Function"
6:45 pm - 6:50 pm
Discussion
6:50 pm - 7:05 pm
Francesca Filippini (Yale University, United States)
"Progranulin and Prosaposin Require LRP10, a Parkinson’s Disease Gene, for Their Efficient Trafficking to Lysosomes"
7:05 pm - 7:10 pm
Discussion
7:10 pm - 7:25 pm
Ellen Hertz (National Institutes of Health, United States)
"Multiomic Analysis of iPSC-derived Neuronal Model from Sib Pairs With Gaucher Disease Discordant for Parkinson Disease"
7:25 pm - 7:30 pm
Discussion
8:00 pm - 9:00 pm
Dinner
Sunday
7:30 am - 8:30 am
Breakfast
9:00 am - 11:00 am
Novel Models and Therapeutic Strategies for Lysosomal Diseases
Discussion Leader: Mahin Hossain (National Institutes of Health, United States)
9:00 am - 9:15 am
Jillian Gallagher (University of Massachusetts Medical School, United States)
"From Mouse to Sheep: Generating a Sheep Model and Developing a Gene Therapy for Sialidosis"
9:15 am - 9:20 am
Discussion
9:20 am - 9:35 am
Amanda Gross (Auburn University, United States)
"Assessing the Effect of Dosage and Injection Route on AAV Gene Therapy in The Treatment of Feline GM2 Gangliosidosis"
9:35 am - 9:40 am
Discussion
9:40 am - 9:55 am
Ruth Azaria (University of Michigan - Ann Arbor, United States)
"Developing New Models to Test Proteostatic Therapies for Niemann-Pick type C Disease"
9:55 am - 10:00 am
Discussion
10:00 am - 10:15 am
Jessica Root (Emory University, United States)
"Investigating the Ability of Granulin Subunits to Rescue Lysosome Dysfunction, Inflammation, and Disease-like Pathology in a Mouse Model of Progranulin Deficiency"
10:15 am - 10:20 am
Discussion
10:20 am - 10:35 am
Adele Correia (University of Michigan, United States)
"Humanized I1061T-NPC1 Mice as a New Model of Niemann-Pick Type C Disease"
10:35 am - 10:40 am
Discussion
10:40 am - 10:55 am
Sarah Hurt (Washington University in St. Louis, United States)
"Elucidating Expression of a Corrective Enzyme for MPS I Through Administration of an Adenoviral Vector Targeted to Endothelial Cells"
10:55 am - 11:00 am
Discussion
11:00 am - 12:30 pm
Poster Session
Coffee will be served in the poster area from 11:00 am - 11:30 am
12:30 pm - 1:30 pm
Lunch
1:30 pm - 2:30 pm
Mentorship Component: Different Career Paths in Science: Prospectives from Academia and Industry
Discussion Leader: Chloe Christensen (CHOC Children's Research Institute, United States)
1:30 pm - 2:30 pm
Panel Discussion
Science Work Experiences in Academia and Industry
Kerri Kinghorn (University College London, United Kingdom)
Rizwana Islam (Takeda, United States)
Dominic Winter (University of Bonn, Institute for Biochemistry and Molecular Biology, Germany)
Rose Sheridan (Freeline Therapeutics, United Kingdom)
2:30 pm - 3:00 pm
Evaluation Period
Complete the GRS Evaluation Forms; Election of Future Chair(s)
3:00 pm
Seminar Concludes
