Great to be part of #ALSNexus26 last week! I presented our work on modulating extracellular vesicles to inhibit dipeptide repeat propagation in C9-ALS, both in the Emerging Science session and as a poster.

Great discussions across the ALS community. #ALS

TDP-43-related cryptic splicing reflects pathological heterogeneity in amyotrophic lateral sclerosis
link.springer.com/article/10.118…

TTBK1 Inhibitors Modulate TDP-43 Pathology and Rescue Cognitive Deficits in a Mouse Model of TDP-43 Proteinopathy
pubs.acs.org/jmcmar/article…

EPHA2/CD44-directed trafficking enhances endosomal leakiness and antisense therapy delivery
rupress.org/jcb/article/22…

Multi-omics of C9orf72 spinal cord organoids reveal convergent molecular signatures and targets
researchsquare.com/article/rs-100…

Impaired Motor Function in a Zebrafish Stathmin-2 Knockout Model
onlinelibrary.wiley.com/doi/10.1002/dn…

α-Synuclein blocks endoplasmic reticulum co-translational protein translocation early in Parkinson’s disease: nature.com/articles/s4146…

Astrocyte dysfunction distinguishes monozygotic twin C9orf72 expansion carriers discordant for amyotrophic lateral sclerosis
biorxiv.org/content/10.648…

Deep axonal proteomics of human iPSC-derived neurons by microfluidic separation and DIA-MS
biorxiv.org/content/10.648…

Schwann Cell–Specific TDP-43 Rescue Improves Peripheral Nerve Myelin Pathology Without Altering Motor Behaviour in a Mouse Model of ALS
biorxiv.org/content/10.648…

Intersting preprint: blocking VPS4a could keep TDP-43 out of exosomes without stopping exosome production. That would suggest that one might be able to block the neuron-to-neuron spread of pathology without shutting down normal vesicle trafficking.
biorxiv.org/content/10.648…

Therapeutic targeting of the conserved region within the low-complexity domain of TDP-43 is neuroprotective and extends survival in amyotrophic lateral sclerosis mice
nature.com/articles/s4358…

Striatal neuron dysfunction in C9ORF72-FTD/ALS is driven by AIS and potassium channel dysregulation
sciencedirect.com/science/articl…

STMN2 protein depletion via translation deficits and stress granules in amyotrophic lateral sclerosis
academic.oup.com/brain/advance-…

An open-label Phase 2a study of fasudil in amyotrophic lateral sclerosis: safety and exploratory endpoints
tandfonline.com/doi/10.1080/21…

Mitochondria directly interact with the nuclear pore complex
nature.com/articles/s4158…

Excited to have shared our work on Nucleoporin210 in a tauopathy model at the Northeast Nuclear Envelope 2026 meeting at Yale (sites.google.com/view/nene2026/…).

Thank you to the organizers for putting together such a nice program, and grateful for all the feedback and discussion!

TDP-43 self-assembly is regulated by its disordered NLS-region biorxiv.org/content/10.648…

Downregulation of miRNAs Accompanies Increased HERV-K (HML-2) Expression in Amyotrophic Lateral Sclerosis
link.springer.com/article/10.100…

Development and characterization of 3D spinal cord organoids to advance the study of amyotrophic lateral sclerosis
link.springer.com/article/10.118…