Perlson lab

In our recent paper we show that TDP43 mislocalization and condensates in axons and neuromuscular junction cause Neurodegenration in ALS by inhibiting local synthesis of mitochondrial proteins nature.com/articles/s4146…

Small heat shock proteins HspB1 and HspB5 differentially alter the condensation and aggregation of the TDP‐43 low‐complexity domain - Walker - 2026 - Protein Science - Wiley Online Library onlinelibrary.wiley.com/doi/10.1002/pr…

Mecca et al. investigate the role of muscle stem cells in SMA and show that their depletion disrupts neuromuscular junctions and leads to motor neuron loss in the spinal cord. They conclude that muscle stem cells represent potential therapeutic targets. shorturl.at/vIwEU

Annexin A7 enhances TIA1 axonal trafficking to counteract pathological aggregation in neurons embopress.org/doi/full/10.10…

Neuron subtypes have distinct mitochondrial transport in axons and vulnerability to tau in vivo researchsquare.com/article/rs-762…

Annexin A7 enhances TIA1 axonal trafficking to counteract pathological aggregation in neurons | The EMBO Journal embopress.org/doi/full/10.10…

KIF5A binds RNA to orchestrate synaptic mRNA localization and stress granules in ALS biorxiv.org/content/10.110… #biorxiv_neursci

Cell size-dependent mRNA transcription drives proteome remodeling biorxiv.org/content/10.110…

Read this excellent story from the @jltwiss1 lab. Acetylation of Axonal G3BP1 through ELP3 Accelerates Axon Regeneration biorxiv.org/content/10.110…

O'Brien et al. demonstrate a link between ALS and extreme exercise in males which is potentially mediated via failed mTOR signalling at the neuromuscular junction. tinyurl.com/cajta7y3; tinyurl.com/ymd5c6h4

Drs. Yi Zeng, Aaron Gitler, and colleagues show that loss of TDP-43 from neuronal nuclei of human brain and disease-causing mutations in TDP-43 are associated with widespread changes in alternative polyadenylation. 🧠 @StanfordMed @NatureNeuro | go.nature.com/473g5Oe

Retrotransposons unplugged: Rewiring the nervous system and wreaking havoc: Neuron cell.com/neuron/fulltex…

Synaptic changes contribute to persistent extra-motor behaviour deficits in the rNLS8 TDP-43 mouse model of amyotrophic lateral sclerosis: biorxiv.org/content/10.110…

Our cover art suggestion. Firefighters spraying mir126 balloons to douse the flames- toxic TDP-43 aggregates that damage axons and neuromuscular junction in ALS. Illustration by Maayan Visuals. nature.com/articles/s4159…

By uncovering a new mechanism that drives ALS progression and identifies a potential therapeutic target, we hope this work opens up new avenues for intervention. Grateful to my brilliant team and collaborators for making this possible.

Thrilled to share our new work in Nature Neuroscience! Together with amazing collaborators and led by Ariel Ionescu, we discovered that muscle-derived miR-126 controls local axonal TDP-43 synthesis and protects neuromuscular junctions in ALS. nature.com/articles/s4159…

This reveals a novel muscle-to-motor neuron communication axis, where loss of miR-126 triggers TDP-43 accumulation, NMJ disruption, and motor decline. Restoring miR-126 demonstrated neuroprotective effects in both mouse and human ALS models.

Translation landscape of stress granules | Science Advances science.org/doi/10.1126/sc…

Development of a Human iPSC-Derived “Corticospinal Tract-on-a-Chip” for Neurodegenerative Disease Research biorxiv.org/content/10.110…

The Impact of Methylglyoxal and SOD1 Mutation on TDP-43 Interaction in ALS Proteinopathy biorxiv.org/content/10.110…

Ubiquitin Proteasome System Components, RAD23A and USP13, Modulate TDP-43 Solubility and Neuronal Toxicity: biorxiv.org/content/10.110…