ESGLD
@ESGLD_tweets
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ESGLD: European study group on lysosomal diseases
Joined May 2021
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 @ !!! Please share and see you in Berlin 😊
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RT @LysoProteomics: Happy to announce the first speaker of our new webinar series of the @ESGLD_tweets: Andrea Ballabio. This series of we….
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RT @ESGLD_tweets: Happy to announce our 2024 ESGLD workshop which will be a joint symposium with the DFG FOR2625! Registration will open 31….
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RT @LysoProteomics: Registration is open NOW for our joint symposium on lysosomes and autophagy by the DFG research unit FOR2625 and the ES….
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And here a glimpse of what you can expect, the list of currently confirmed speakers.
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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.
And the winner of the EURORDIS Photo Award 2023 is. 'Raising Rare', photographed by Lindsay Norman from the USA. 📸. Blakely was born at 26 weeks. Two weeks later she was given a terminal diagnosis of Mucolipidosis type 2. She is 2 years old in this photo. #EURORDISAwards2023
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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. 👏🏻.
Great to see @Tigem_Telethon researchers including Fabio Dell'Aquila @D91Fabio and @cristinasorr participating at the @ESGLD_tweets congress at @LancasterUni!
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Happy to have you and learn more on your exciting results 👏🏻.
Proud to work at the @HCS_Tigem with @Anto_Capuozzo and Giuseppina Grieco, both selected to present their last results studying mucopolysaccharidosis type IIIA and Mucolipidosis type IV in the 24th #Esgldworkshop. Thanks, @Tigem_Telethon, @Telethonitalia, and @CureSanfilippoF
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We're back for our final #ESGDLWorkshop day with an exciting session on Lysosomal Pathology 🧐.
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🧐 Still on IDS-fusion peptides for BBB-crossing in MPS type II, it's time for us to hear @SRWood1989 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 💉🐁
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🧐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❕
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🧐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❔❕. 💊🐁🔍
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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. 🧫🧬➡️💉🐁
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🧐 We're starting this afternoon with a keynote lecture on the transport of biotherapeutics across the BBB by 👩🏻⚕️Annie Arguello (@Denalitx). And isn't that the journey we all wish to travel❔❕
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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 🧫🔍
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RT @D91Fabio: @ESGLD_tweets It was a great pleasure! Very interesting lectures and talks #lysosomes #therapies #MPS.
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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❕
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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 ❕👏🏻
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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❕
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🤔 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. 🐁🔍
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