Rothstein Lab

Exploratory assessment of phosphorylated TDP-43 immunoreactivity in minor salivary glands of patients with ALS link.springer.com/article/10.118…

Characterizing TDP-43 involvement in vascular dementia alz-journals.onlinelibrary.wiley.com/doi/10.1002/al…

Amisodin alleviates TDP-43-associated ALS pathology by inhibiting the trimeric SOD1 researchsquare.com/article/rs-869…

C9orf72-derived dipeptide repeat proteins poly-PR disrupt membrane excitability and synaptic function in cortical neurons sciencedirect.com/science/articl…

C9orf72 hexanucleotide repeat RNA drives transcriptional dysregulation through genome-wide DNA:RNA hybrid G-quadruplexes cell.com/neuron/abstrac…

Impaired nucleocytoplasmic transport in SOD1-mediated ALS link.springer.com/article/10.118…

Novel extracellular vesicle release pathway facilitated by toxic superoxide dismutase 1 oligomers sciencedirect.com/science/articl…

Blocking RAN translation without altering repeat RNAs rescues C9ORF72-related ALS and FTD phenotypes science.org/doi/10.1126/sc…

TDP-43 dysfunction leads to the accumulation of cryptic transposable element-derived exons, crypTEs, in iPSC derived neurons and ALS/FTD patient tissues biorxiv.org/content/10.648…

Quantifying multimodal longitudinal brain changes in presymptomatic C9orf72 disease alz-journals.onlinelibrary.wiley.com/doi/10.1002/al…

MicroRNA profiling in post-mortem spinal cord of C9ORF72-related ALS patients reveals molecular pathways involved in motor neuron degeneration frontiersin.org/journals/neuro…

Co-aggregation of annexin A11 and TDP-43 in FTLD/MND with primary lateral sclerosis phenotype link.springer.com/article/10.118…

Calcineurin depletion coincides with phosphorylated TDP-43 deposition in a mouse model of ALS/FTLD-TDP link.springer.com/article/10.118…

Revisiting oligodendrocytes in amyotrophic lateral sclerosis using human multicellular stem cell models cell.com/trends/cell-bi…

Tofersen, SOD1, and the Treatability of Amyotrophic Lateral Sclerosis jamanetwork.com/journals/jaman…

Long-Term Tofersen in SOD1 Amyotrophic Lateral Sclerosis jamanetwork.com/journals/jaman…

Lysosomal escape and TMEM106B fibrillar core determine TDP-43 seeding outcomes biorxiv.org/content/10.648…

CRISPR screens in human neural organoids and assembloids nature.com/articles/s4159…

Last week, we held the 36th #alsmndsymp in San Diego. The essence of the International Symposium is to bring the global community together and drive #MND #ALS research forward. 🌍 Read more about this year's symposium on our blog ⬇️ mndresearch.blog/2025/12/12/bri…

LRRC59 cooperates with nuclear transporters to restrain the nuclear envelope repair machinery and safeguard genome integrity nature.com/articles/s4146…