Geoff Cross 0770 2278385 geoffcross@video4medics.com

ESGLD Virtual Summer Meeting 2021 – Programme

Please note ALL times are UK time (BST)

Day 1  Monday 28th June

Welcome

12pm (BST)/1pm (CEST)     Welcome introduction - Brian Bigger - ESGLD Chairman, UoM, Manchester, UK

Mechanisms of LSDs

Session 1 (Chair Eeva-Liisa Eskelinen)

12.10pm (BST)/1.10pm (CEST)   Invited speakerCarmine Settembre - TIGEM, Naples, Italy - Selective autophagy dysfunctions in lysosomal storage disorders.

12.45pm (BST)/1.45pm (CEST)   Ulrich Matzner - Deletion of fatty acid amide hydrolase reduces lyso-sulfatide levels but aggravates the disease phenotype of a mouse model of metachromatic leukodystrophy

1.05pm (BST)/2.05pm (CEST)     Alessia Calcagni - CLN3 protein is at the crossroad between the biosynthetic/secretory compartment and lysosomes

1.25pm (BST)/2.25pm (CEST)     Coffee break

Session 2 (Chair Fran Platt)

1.45pm (BST)/2.45pm (CEST)     Invited speaker Judith Klumperman - UMC Utrecht, Utrecht, Netherlands - The role of the HOPS complex in lysosomal biogenesis and diseases

2.20pm (BST)/3.20pm (CEST)     Oriana Mandolfo - Systemic immune challenges exacerbate inflammation and cognitive decline in a mouse model of MPSIIIA

2.40pm (BST)/3.40pm (CEST)     Lea Vidatic - BACE1 inhibition ameliorates neuroinflammation in organotypic brain slices of Niemann-Pick type C disease murine model

3pm (BST)/4pm (CEST)  Coffee break

Session 3 (Chair Paul Saftig)

3.20pm (BST)/4.20pm (CEST)     Invited speaker - Christian Haas - LMU Munich DZNE, Munich, Germany - Lysosomal dysfunction in microglia drives neuronal TDP-43 pathology in PGRN related FTLD

3.55pm (BST)/4.55pm (CEST)     Antonietta TaralloCorrection of oxidative stress enhances enzyme replacement therapy in Pompe disease

4.15pm (BST)/5.15pm (CEST)     José Clemente-Ramos - Using S. pombe to understand the pathogenesis of CLN3 disease

4.35pm (BST)/5.35pm (CEST)     Group discussion on Mechanisms of LSDs (Chaired by Paul Saftig and Including Invited speakers from the day)

4.55pm (BST)/5.55pm (CEST)   Day 1 main conference closes

5.00pm-5.30pm (BST)/6.00pm-6.30pm (CEST) Day 1 - Zoom Drinks Reception
(Everyone Welcome - Bring Your Own Beer!)
Meeting ID will follow

Day 2   Tuesday 29th June

Lysosomal Function

Session 1 (Chair Andrea Ballabio)

12pm (BST)/1pm (CEST)   Keynote speakerJuan Bonifacino - NIH, Bethesda, USA

Molecular mechanisms of lysosome positioning

Bright ideas/flash talks

12.35pm (BST)/1.35pm (CEST)   S.L.M. in 't GroenUncovering pathological mechanisms in Pompe disease using a 3D-skeletal-muscle-on-a-chip system.

12.40pm (BST)/1.40pm (CEST)    Mara Reichmann - Loss of TMEM55B leads to subfertility in male mice

12.45pm (BST)/1.45pm (CEST)   Florian Bleimbaum - Co-expression network analysis identifies NHLRC3 as a new lysosomal protein to control endothelial cell homeostasis

12.50pm (BST)/1.50pm (CEST)   Tereza Andreou - Brain-targeted Haematopoietic Stem Cell Gene Therapy for severe Mucopolysaccharidosis I (Hurler) - does one peptide tag fit all?

12.55pm (BST)/1.55pm (CEST)    Jane Potter - Ex-vivo autologous haematopoietic stem cell gene therapy in mucopolysaccharidosis type IIIA

1pm (BST)/2pm (CEST)               Q&A group discussion (Chair Andrea Ballabio)

1.20pm (BST)/2.20pm (CEST)     Coffee break

Session 2 (Chair Mia Horowitz)

1.40pm (BST)/2.40pm (CEST)     Invited speakerChiara di Malta - TIGEM, Naples, Italy - Transcriptional regulation of mTORC1 signaling in physiology and cancer

2.15pm (BST)/3.15pm (CEST)     M Klussendorf - Cytoplasmic retention of acetylated transcription factor EB

2.35pm (BST)/3.35pm (CEST)     Sonke Rudnik - S-palmitoylation determines TMEM55B-dependent positioning of lysosomes

2.55pm (BST)/3.55pm (CEST)     Coffee break

Session 3 (Chair Thomas Braulke)

3.15pm (BST)/4.15pm (CEST)     Invited speaker - James H. Hurley - University of California, Berkley, USA - Coordination of the Rag GTPase cycle on lysosomes

3.50pm (BST)/4.50pm (CEST)     Dominic Winter - Characterization of Lysosomal Protein Interactions and Structures by Cross Linking Mass Spectrometry

4.10pm (BST)/5.10pm (CEST)     Shroddha Bose - Impact of altered chloride transport on lysosomal morphology and function

4.30pm (BST)/5.30pm (CEST)     Group discussion on lysosomal function (Chaired by Andrea Ballabio and Including Invited speakers from the day)

5.00pm (BST)/6.00pm (CEST)      Day 2 main conference closes

5 – 5.30pm (BST)/6 – 6.30pm (CEST) Day 2 - Zoom Drinks Reception (Everyone Welcome - Bring Your Own Beer!) Meeting ID will follow

Day 3    Wednesday 30th June

Therapies and diagnosis

Session 1 (Chair Francisca Coutinho)

12pm (BST)/1pm (CEST)             Invited speaker - Sandra Alves - Instituto Nacional de Saúde Dr. Ricardo Jorge (INSA), Portugal - Oligonucleotide-based therapies for inherited metabolic diseases: some examples of their application in Lysosomal Storage Disorders.

12.35pm (BST)/1.35pm (CEST)    Xuefang Pan - Oral Glucosamine Ameliorates Aggravated Neurological Phenotype in Mucopolysaccharidosis III Type C Mouse Model Expressing Misfolded HGSNAT Variant

12.55pm (BST)/1.55pm (CEST)    Atze BergsmaIdentification of aberrant splicing events that induce nonsense-mediated decay and development of splice switching antisense oligonucleotides for Pompe disease

1.15pm (BST)/2.15pm (CEST)    Coffee break

 

Session 2 (Chair Pim Pijnappel)

1.35pm (BST)/2.35pm (CEST)      Invited speaker - Federico Mingozzi - Spark Therapeutics, Philadelphia, USA - Targeting the liver to develop in vivo gene therapies for lysosomal storage diseases

2.10pm (BST)/3.10pm (CEST)      Alessandro di Spiezio - Enzyme replacement therapy with pro cathepsin D in NCL disease

2.30pm (BST)/3.30pm (CEST)      Valeria De Pasquale - Targeting heparin sulfate proteoglycans as a novel treatment for neuropathology in Mucopolysaccharidosis IIIB

2.50pm (BST)/3.50pm (CEST)    Coffee break

Session 3 (Chair Brian Bigger)

3.10pm (BST)/4.10pm (CEST)      Invited speaker - Eric Adler - UC San Diego, La Jolla, USA - New Therapy for Danon Disease (TBC)

3.45pm (BST)/4.45pm (CEST)      Shaun Wood - Long-term Outcomes of Brain-targeted Haematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis Type II in Pre-Clinical MPSII Mouse Model

4.05pm (BST)/5.05pm (CEST)      Nicola BrunettiSafety and efficacy of liver-directed AAV-mediated gene therapy for mucopolysaccharidosis type VI

4.25pm (BST)/5.25pm (CEST)      Group discussion on therapies and diagnosis (Chaired by Brian Bigger and Including Invited speakers from the day)

4.45pm (BST)/5.45pm (CEST)    Coffee break

4.55pm (BST)/5.55pm (CEST)      Award for best presentation

5pm(BST)/6pm (CEST)                Award for best flash talk

5.05pm (BST)/6.05pm (CEST)    Concluding remarks

5.10pm (BST)/6.10pm (CEST)      Conference close

Register Now!

Register now for ESGLD 2021 Virtual Summer Meeting. Please note that there is no fee for ESGLD members. Upon receipt of your completed registration form we will confirm that you or your group are ‘paid up’. Upon confirmation of membership status you will then receive an email notification with your Virtual Summer Meeting login details including unique username and password.

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