CRISPR screening by AAV episome-sequencing (CrAAVe-seq): a scalable cell-type-specific in vivo platform uncovers neuronal essential genes nature.com/articles/s4159…

Transcriptome-based screening in TARDBP/TDP-43 knock-in motor neurons identifies the NEDD8-activating enzyme inhibitor MLN4924 nature.com/articles/s4159…

Molecular impact of antisense oligonucleotide therapy in C9orf72-associated ALS: Cell cell.com/cell/fulltext/…

Multi-modal comparative phenotyping of knock-in mouse models of frontotemporal dementia/amyotrophic lateral sclerosis journals.biologists.com/dmm/article/18…

Endolysosomal dysfunction impairs proteostasis and induces neurodegeneration in vivo cell.com/iscience/fullt…

Cortical hyperexcitability drives dying forward amyotrophic lateral sclerosis symptoms and pathology in mice sciencedirect.com/science/articl…

TARDBP (TDP-43) Knock-in Zebrafish Display a Late-Onset Motor Phenotype and Loss of Large Spinal Cord Motor Neurons onlinelibrary.wiley.com/doi/10.1002/an…

Multi-modal comparative phenotyping of knock-in mouse models of frontotemporal dementia/amyotrophic lateral sclerosis journals.biologists.com/dmm/article/18…

Metformin decreases RAN proteins, rescues splicing abnormalities and improves behavioral phenotypes in SCA8 BAC mice biorxiv.org/content/10.110…

Neurofilament light chain dynamics in the pre-symptomatic phase of amyotrophic lateral sclerosis: a case report tandfonline.com/doi/abs/10.108…

CSF total tau as a proxy of synaptic degeneration nature.com/articles/s4146…

Decoding ATXN2 Phosphocode: Structural Insights and Therapeutic Opportunities in Disease link.springer.com/article/10.100…

C9orf72 ALS‐causing mutations lead to mislocalization and aggregation of nucleoporin Nup107 into stress granules febs.onlinelibrary.wiley.com/doi/10.1002/18…

Cryptic splicing in synaptic and membrane excitability genes links TDP-43 loss to neuronal dysfunction biorxiv.org/content/10.110….

Generation of Astrocyte-Selective Cre Driver Lines With Distinct Onsets of Recombination Activity During Development onlinelibrary.wiley.com/doi/abs/10.100…

Can non-pharmacological interventions change levels of neurofilament light in older adults at risk of dementia? A secondary analysis of the SCD-Well randomized clinical trial sciencedirect.com/science/articl…

Network dysfunction precedes neurodegeneration in a dox-regulatable TDP-43 mouse model of ALS-FTD. jneurosci.org/content/early/…

HDAC6 and TDP-43 promote autophagy impairment in amyotrophic lateral sclerosis sciencedirect.com/science/articl…

Comparative snRNAseq study of C9orf72, SOD1, and sALS spinal cord biorxiv.org/content/10.110…

Cardiac Troponin T in Tofersen-Treated SOD1 ALS Patients: Beginning to Resolve the Catch-22 of Identifying Treatment Responsive Biomarkers in ALS Drug Development onlinelibrary.wiley.com/doi/abs/10.100…