7th September 2022
The Graduate course starts at 2 PM on Wednesday 7th September 2022. The graduate course programme and curriculum can be accessed below.
Graduate Course Programme
The Graduate Course (7th and 8th September 2022) is an opportunity for postdocs, PhD students and clinical fellows who are new to the lysosomal disease field to be taught about the fundamentals of lysosomal diseases by experts who are at the cutting edge of these areas and to have a chance to informally practice presenting their work in a non-challenging environment to their peers. Participants in the graduate course usually go on to attend the workshop afterwards.
This year we have an excellent line-up of teachers who will cover the following themes:
• Lysosome Biogenesis (Thomas Braulke)
• Autophagy (David Rubinsztein)
• HOPS complex (Jan Van Der Beek)
• Channels and Transporters (Bruno Gasnier)
• Membranes (Markus Damme)
• Misfolding in lysosomal diseases (Mia Horowitz)
• Lipid degradation and lipid storage diseases (Hans Aerts)
• Lysosomal Leukodystrophies (Volkmar Gieselmann)
• Neuronal Ceroid Lipofuscinosis (Angela Schulz)
• Mucopolysaccharidoses (Nicole Muschol)
• Current therapies for lysosomal diseases (TBC)
• Novel Therapies for LSDs (Brian Bigger)
Graduate Course Curriculum
- Lysosome Biogenesis/Biosynthesis of enzymes and accessory proteins – Thomas Braulke
Rough ER, N-Glycosylation, Processing, Generation M6P, Phosphotransferase and uncoupling enzyme, sorting from Golgi to lysosome, partial secretion, Proteolytic processing of lysosomal enzymes, Mucolipidosis Type II and III. Structure of 46kda and 300 kDa M6P receptors, IGFII/M6P/GlcNac-P-Man binding domains.
- Autophagy – David Rubinsztein
Regulation of autophagy, selective vs non selective forms of autophagy, termination of autophagy, physiological roles of autophagy during tissues development and maintenance, pharmacological modulation of autophagy.
- HOPS complex – Jan Van Der Beek
The role of HOPS in endolysosomal biogenesis and fusion, structure of HOPS complex, HOPS subunits, regulation and additional functions. Endocytosis, pathway from CCV>EE >LE >Lysosome, APs, Rabs, ESCRT, Corvet, HOPS, MVBs, role of Ubiquitinilation.
- Channels and Transporters – Bruno Gasnier
Lysosomal ion channels and small-molecule transporters, and their integration in cell metabolism and signalling.
- Lipid degradation and lipid storage diseases – Hans Aerts
Sphingolipid metabolism, Ganglioside degradation, enzymes, prosaposin, GM2 activator. Relevant biochemistry of major diseases Gaucher, TS, Fabry, lesser info on NPA, NPB, NPC, Sandhoff.
- Lysosomal Leukodystrophies – Volkmar Gieselmann
Disease pathology, animal models, current and new treatment options. Krabbe, MLD, MSD (relevant parts of Fabry, Gaucher GM gangliosidosis).
- Misfolding in lysosomal diseases – Mia Horowitz
misfolding in lysosomal diseases, its impact on LSDs, how can it be validated in animal models, relevant therapies – chaperones, and its importance in the association between a LSD and a neurological disease like Gaucher disease and Parkinson disease.
- Neuronal Ceroid Lipofuscinosis – Angela Schulz
Batten disease, disease pathology, animal models, current and new treatment options.
- Mucopolysaccharidoses – Nicole Muschol
Heparan-, Dermatan-, Chondroitin-, Keratan sulfate degradation pathways. Mucopolysaccharidoses, from biochemical defects to phenotype(s).
- Lysosomal Membrane proteins – Markus Damme
Lysosomal membrane proteins in general, LAMPs, LIMP, Proton pump, (storage) diseases caused by transporter deficiencies, NPC1, lysosomal membrane proteins and their interaction with cytosolic components.
- Current therapies for LSDs – Simon Jones
Overview of ERT, SRT and chaperone treatments approved and used for many lysosomal diseases.
Overview of the use of HSCT in MPSI and beyond and HSC gene therapy in MLD.
- Novel Therapies for LSDs– Brian Bigger
Overview of therapies in development – What targets should we aim for,
What are limitations of existing therapies, Routes of intervention, Brain targeted ERT,
Gene therapy, next generation of brain and bone targeted HSC gene therapy,
other novel therapies, Combination therapy.
ESGLD Graduate Course
Please note ALL times are UK time (BST)
Day 1 of Graduate Course
Wednesday 7th September
12:30 – 14:00 Registration and Lunch – Lancaster University Management School Atrium
ALL Graduate course lectures will take place in Management School Lecture Theatre 14
14:00 – 14:15 Introductory Remarks – Brian Bigger
14:15 – 15:00 Lysosome Biogenesis/Biosynthesis of enzymes – Thomas Braulke
15:00 – 15:45 Autophagy – David Rubinsztein
15:45 – 16:30 HOPS complex – Jan van der Beek
16:30 – 17:00 Coffee Break and Refreshments - Management School Atrium
17:00 – 17:45 Channels and Transporters – Bruno Gasnier
17:45 – 18:30 Lipid degradation and lipid storage diseases – Hans Aerts
18:30 – 19:15 Lysosomal Leukodystrophies – Volkmar Gieselmann
19:15 – 19:30 Designation of flash-talk discussion leaders - Management School Atrium
20:00 DINNER - Marketplace
Students will need to prepare their flash talks over dinner
Day 2 of Graduate Course Thursday 8th September
07:00 – 08:30 Breakfast - Marketplace
All Graduate course lectures will take place in Management School Lecture Theatre 14
08:30 – 09:15 Misfolding in lysosomal diseases – Mia Horowitz
09:15 – 10:00 Neuronal Ceroid Lipofuscinosis – Angela Schulz
10:00 – 10:45 Mucopolysaccharidoses – Nicole Muschol
10:45 – 11:15 Coffee Break and Refreshments - Management School Atrium
11:15 – 12:00 Lysosomal Membrane proteins – Markus Damme
12:00 – 12:45 Current therapies for LSDs – Simon Jones
12:45 – 13:30 Novel Therapies for LSDs– Brian Bigger
13:30 – 14:30 Lunch - Management School Atrium
14:30 – 17:00 FLASH TALKS BY GRADUATE STUDENTS AND POSTDOCS
Management School Lecture Theatre 14
Graduate course students will be asked to prepare 1-3 slides (4-6 minute talk)
on a current scientific topic over dinner on 7/09/2022.
Flash talks will be delivered during this session
Workshop only Single room £650
Workshop only Twin room £550
Partner rate (Twin or double room)
no attendance to talks £450
Workshop only Student rate £300
Graduate Course £100
A limited number of travel grants, valid for up to 300 EUR per person, is available for members who do not receive sufficient support from their institutions. Please send a request for support directly to Pim Pijnappel by e-mail firstname.lastname@example.org
The European Study Group on Lysosomal Diseases would like to thank all our sponsors for their support of the 24th ESGLD Workshop and Graduate Course.
For further information regarding 24th ESGLD Workshop and Graduate Course, 2022 Lancaster please contact:
Dr Tereza Andreou
School of Biological Sciences
Faculty of Biology, Medicine and Health
3.721 Stopford Building
The University of Manchester