@ShorterLab

Countering deleterious phase transitions in ALS/FTD, PD, AD, and related disorders @Penn @PennMedicine @bb_upenn @BMBGGUPENN @PGGUPenn @PennNGG @CAMBUpenn

University of Pennsylvania
Joined January 2012
Biased stochastic motor dynamics drive bidirectional, processive translocation by the AAA+ disaggregase Hsp104: biorxiv.org/content/10.64898… – at Millbourne, PA
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A long time in the making, our latest work on large-scale membrane MD simulations is finally out! We show that LC3 forms nanoclusters on autophagosomes, with implications for how they bind cargo and move!! A wonderful collaboration with Nitin Ram’s lab who spearheaded experimental work at IIT Kanpur, and great help from Anirban Banerjee on the xenophagy experiments. Big congratulations to our first authors of the study Saman and Deepak and to the entire team! And the icing on the cake is: @TheHurleyLab commentary on our work! See thread below for our key findings.
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A targeted treatment for a rare genetic form of ALS has met its main goal in a Phase 3 trial. Ulefnersen is designed to reduce production of the FUS protein, which can build up in motor neurons in people with FUS-ALS. In the FUSION study, the therapy performed significantly better than placebo on a combined measure that included function and survival. Detailed results have not yet been released, but the companies are now discussing potential expedited approval pathways with regulators. Read more: bit.ly/4d3Oq2e #ALS #ALSTreatment #ALSResearch #ALSNewsToday #Bionews
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Happy to share the revised and now published in Cell Systems version of our paper, where we explore why dCas9-KRAB(KOX1) protein levels drop so drastically after neuronal differentiation in WTC11 and KOL2.1J iPSC lines. Paper: doi.org/10.1016/j.cels.2026.…
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Home Fly Eagles Fly 🦅🦅
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Otsuka Announces Transformative Phase 3 FUSION Results for Ulefnersen, Bringing the FUS-ALS Community Closer to a Potential Targeted Treatment: otsuka-us.com/news/otsuka-an…
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