@Rothstein_Lab

Dr. Jeffrey Rothstein is a professor of neurology and neuroscience @HopkinsMedicine and is the founding director of @packardcenter. (trainee run)

 Baltimore, MD
rothsteinlab.org
Joined April 2023
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Rothstein Lab retweeted
Suleyman Can Akerman@CanAkermanSep 3
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
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TDP-43-related cryptic splicing reflects pathological heterogeneity in amyotrophic lateral sclerosis link.springer.com/article/10…

TDP-43-related cryptic splicing reflects pathological heterogeneity in amyotrophic lateral sclerosis

Acta Neuropathologica Communications - Amyotrophic lateral sclerosis (ALS) involves TAR DNA-binding protein 43 (TDP-43) dysfunction, leading to cryptic splicing, including UNC13A cryptic exon...

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

Selective Brain-Penetrant TTBK1 Inhibitors Modulate TDP-43 Pathology and Rescue Cognitive Deficits...

Abstract. Transactive response DNA-binding protein of 43 kDa (TDP-43) is a pathological hallmark of neurodegenerative disorders, including amyotrophic late

pubs.acs.org
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EPHA2/CD44-directed trafficking enhances endosomal leakiness and antisense therapy delivery rupress.org/jcb/article/225/…

EPHA2/CD44-directed trafficking enhances endosomal leakiness and antisense therapy delivery

A scavenger receptor (CD44) and a receptor tyrosine kinase (EPHA2) join forces to mediate internalization of therapeutic ASOs into endosomes, which are the

rupress.org
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Rothstein Lab retweeted
Suleyman Can Akerman@CanAkermanAug 7
Multi-omics of C9orf72 spinal cord organoids reveal convergent molecular signatures and targets researchsquare.com/article/r…

Multi-omics of C9orf72 spinal cord organoids reveal convergent molecular signatures and targets

Background: amyotrophic lateral sclerosis is the most common adult-onset motor neuron disease, characterised by the progressive degeneration of upper and lower motor neurons in the brain and spinal...

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

Impaired Motor Function in a Zebrafish Stathmin‐2 Knockout Model

Stathmin-2 (STMN2) is a microtubule-associated protein that plays a role in the stability of microtubules in axons of the nervous system of animals. In this study, we generated a novel zebrafish...

onlinelibrary.wiley.com
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Rothstein Lab retweeted
ShorterLab@ShorterLabAug 6
α-Synuclein blocks endoplasmic reticulum co-translational protein translocation early in Parkinson’s disease: nature.com/articles/s41467-0…

α-Synuclein blocks endoplasmic reticulum co-translational protein translocation early in Parkinso...

Nature Communications - Proteomic and transcriptomic profiling on hiPSC-derived midbrain dopaminergic neurons with pathological α-synuclein burden reveals defective Sec61A interactions and...

nature.com
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Astrocyte dysfunction distinguishes monozygotic twin C9orf72 expansion carriers discordant for amyotrophic lateral sclerosis biorxiv.org/content/10.64898…

Astrocyte dysfunction distinguishes monozygotic twin C9orf72 expansion carriers discordant for...

Hexanucleotide repeat expansions in C9orf72 are the most common genetic cause of amyotrophic lateral sclerosis, yet many carriers remain asymptomatic for decades or never develop disease. This...

biorxiv.org
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Deep axonal proteomics of human iPSC-derived neurons by microfluidic separation and DIA-MS biorxiv.org/content/10.64898…
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Schwann Cell–Specific TDP-43 Rescue Improves Peripheral Nerve Myelin Pathology Without Altering Motor Behaviour in a Mouse Model of ALS biorxiv.org/content/10.64898…

Schwann Cell–Specific TDP-43 Rescue Improves Peripheral Nerve Myelin Pathology Without Altering...

Amyotrophic lateral sclerosis (ALS) is a terminal disease caused by motor neuron loss. Schwann cells, the myelinating cells of the peripheral nervous system, metabolically and structurally support...

biorxiv.org
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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/10.64898…

ESCRT Machinery Dysfunction in Motor Neurone Disease: TSG101, CHMP2B, and VPS4a Differentially...

Motor Neurone Disease (MND) is characterised by progressive degeneration of upper and lower motor neurons, accompanied by cytoplasmic mislocalisation and hyperphosphorylation of TDP-43 — hallmarks...

biorxiv.org
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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/s43587-0…

Therapeutic targeting of the conserved region within the low-complexity domain of TDP-43 is...

Nature Aging - Gao et al. identify a toxicity-driving region within TDP-43 and show that a brain-penetrant small molecule targeting this region improves mitochondrial function, reduces...

nature.com
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Striatal neuron dysfunction in C9ORF72-FTD/ALS is driven by AIS and potassium channel dysregulation sciencedirect.com/science/ar…
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STMN2 protein depletion via translation deficits and stress granules in amyotrophic lateral sclerosis academic.oup.com/brain/advan…

STMN2 protein depletion via translation deficits and stress granules in amyotrophic lateral...

Ellis et al. explore how the ALS-associated protein STMN2 is regulated during neuronal stress. They show that cellular stress reduces STMN2 levels through

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

An open-label Phase 2a study of fasudil in amyotrophic lateral sclerosis: safety and exploratory...

The primary objective was to assess the safety of oral fasudil in amyotrophic lateral sclerosis (ALS) patients. Changes in serum neurofilament light (NfL) levels and the ratio of phosphorylated to ...

tandfonline.com
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Mitochondria directly interact with the nuclear pore complex nature.com/articles/s41586-0…

Mitochondria directly interact with the nuclear pore complex

Nature - Mitochondria interact directly with the nuclear pore complex via VDAC1–RANBP2 binding to sustain nuclear ATP levels.

nature.com
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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/nene20…). Thank you to the organizers for putting together such a nice program, and grateful for all the feedback and discussion!
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Rothstein Lab retweeted
Charlotte Fare@charlotte_fareJun 13
TDP-43 self-assembly is regulated by its disordered NLS-region biorxiv.org/content/10.64898…

TDP-43 self-assembly is regulated by its disordered NLS-region

Cytosolic inclusions of the RNA-binding protein TDP-43 are a pathological hallmark of several neurodegenerative diseases, such as amyotrophic lateral sclerosis and frontotemporal dementia. Cellular...

biorxiv.org
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Downregulation of miRNAs Accompanies Increased HERV-K (HML-2) Expression in Amyotrophic Lateral Sclerosis link.springer.com/article/10…

Downregulation of miRNAs Accompanies Increased HERV-K (HML-2) Expression in Amyotrophic Lateral...

Molecular Neurobiology - Amyotrophic lateral sclerosis (ALS) is a rapidly progressing neurodegenerative disease with limited treatments. Evidence suggests that reactivation of the HERV-K (HML-2)...

link.springer.com
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Development and characterization of 3D spinal cord organoids to advance the study of amyotrophic lateral sclerosis link.springer.com/article/10…

Development and characterization of 3D spinal cord organoids to advance the study of amyotrophic...

Molecular Biomedicine -

link.springer.com
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