🤔 Now it's time for a drug repositioning study: Nicolina Cristina Sorrentino is showing us her team's promising results on the use of a well-known antidepressant to ameliorate the systemic pathology in a mouse model of MSD. 🐁🔍

We're back from our coffee break and ready to learn more on yet another attempt to ameliorate brain pathology in #lysosomalstoragediseases: the development of an IGF2-tagged enzyme for Pompe disease 💉🐁 Great set of data, Fabio Catalano❕

And now Fabio Dell'Aquila brings us an exciting proof of concept on CRISPR/Cas9-triggered homology-independent targeted integration in vivo, in a mouse model og MPS VI. 💠🧬➡️💉🐁 Great results, Fabio! Keep on the good work ❕👏🏻

ESGLD It was a great pleasure! Very interesting lectures and talks #lysosomes #therapies #MPS

Now moving into the clinic, guided by the hand of Charlotte Aries, who's showing us some nice & moving results on the use of Ambroxol both as a monotherapy and as a combination one in real-life Gaucher disease type2 patients 👏🏻 💊👶🏻➡️👩🏻‍⚕️🩺 Unbelievable results, congratulations❕

Time to close this morning's excellent session, with a talk on disease modeling. Let's listen to Silvija Tokic telling us all about her novel cell model for Fabry disease: primary endothelial colony forming cells 🧫🔍

🧐 We're starting this afternoon with a keynote lecture on the transport of biotherapeutics across the BBB by 👩🏻‍⚕️Annie Arguello (Denali Therapeutics). And isn't that the journey we all wish to travel❔❕

Back to our selected oral communications on #therapies, with Oriana Mandolfo, who will tell us all about her assessments on the efficacy of an innovative iPSC-based gene therapy approach for MPS IIIA. 🧫🧬➡️💉🐁

🧐Time to have yet another look over Fluoxetine's potential for drug repositioning in #lysosomalstoragediseases. 🤔 Who would have thought this long-known antidepressant would have a role to play on the search of a treatment for MPS IIIA❔❕ 💊🐁🔍

🧐Back from coffee break with yet another attempt to further improve BBB-crossing through the generation & evaluation of novel IDS fusion peptides for MPS II. Really nice effort and promising results, Rachel Searle❕

🧐 Still on IDS-fusion peptides for BBB-crossing in MPS type II, it's time for us to hear Shaun Wood on his in vivo results using two different peptides fused to IDS and delivered via HSCGT: 1️⃣ the rabies virus glycoprotein & 2️⃣ the herpes virus glycoprotein-derived peptide 💉🐁

We're back for our final #ESGDLWorkshop day with an exciting session on Lysosomal Pathology 🧐

Happy to have you and learn more on your exciting results 👏🏻

Glad to have all these young and talented researchers attending. As usual, Tigem Telethon was well represented in our meeting, with a number exciting & innovative works all all-things-lysosomes. 👏🏻

We're quickly approaching the #RareDiseaseDay and this seems like the perfect time to share this year's winner of the Eurordis Photo Award, which depicts an #LSD Patient/Family. ⬇️ So meet 'Raising Rare', a touching depiction of a ML II-affected child with her caring mother.

Happy to announce our 2024 ESGLD workshop which will be a joint symposium with the DFG FOR2625! Registration will open 31st of January but pre-registration is already open @ lysosomes2024.de !!! Please share and see you in Berlin 😊

Registration is open NOW for our joint symposium on lysosomes and autophagy by the DFG research unit FOR2625 and the ESGLD in Berlin, Germany in June. Don't wait too long as the number of participants is limited... Please share and re-tweet! See you in Berlin 🥳 #lysosomes

Happy to announce the first speaker of our new webinar series of the ESGLD: Andrea Ballabio. This series of webinars will feature prominent scientists in lysosome research, so if you are interested in lysosomes and their roles in human disease, don't miss it!