Evidence map›Paper›PMID 39138578›Full record

ArticleFluids and barriers of the CNS2024

A patient-derived amyotrophic lateral sclerosis blood-brain barrier model for focused ultrasound-mediated anti-TDP-43 antibody delivery.

Joanna M Wasielewska, Juliana C S Chaves, Mauricio Castro Cabral-da-Silva, Martina Pecoraro, Stephani J Viljoen, Tam Hong Nguyen, Vincenzo La Bella, Lotta E Oikari, Lezanne Ooi, Anthony R White

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Fluids and barriers of the CNS, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

10 citing papers in PubMed.

  1. Review
  2. Review
  3. Reconstructing cerebral lymphatic clearance: an emerging target in the Alzheimer's disease therapeutic pipeline.Alzheimer's & dementia : the journal of the Alzheimer's Association · 2026
    Review
  4. Review
  5. Review
  6. Review
  7. Review
  8. Review
  9. Drug repurposing in amyotrophic lateral sclerosis (ALS).Expert opinion on drug discovery · 2025
    Review
  10. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

10 authors.

Joanna M WasielewskaBrain and Mental Health Program, QIMR Berghofer Medical Research Institute, Brisbane, QLD, Australia.
Juliana C S ChavesBrain and Mental Health Program, QIMR Berghofer Medical Research Institute, Brisbane, QLD, Australia.
Mauricio Castro Cabral-da-SilvaMolecular Horizons, School of Chemistry and Molecular Bioscience, University of Wollongong, Northfields Avenue, Wollongong, NSW, Australia.
Martina PecoraroALS Clinical Research Centre and Laboratory of Neurochemistry, Department of Biomedicine, Neurosciences and Advanced Diagnosis, University of Palermo, Palermo, Italy.
Stephani J ViljoenBrain and Mental Health Program, QIMR Berghofer Medical Research Institute, Brisbane, QLD, Australia.
Tam Hong NguyenFlow Cytometry and Imaging Facility, QIMR Berghofer Medical Research Institute, Brisbane, QLD, Australia.
Vincenzo La BellaALS Clinical Research Centre and Laboratory of Neurochemistry, Department of Biomedicine, Neurosciences and Advanced Diagnosis, University of Palermo, Palermo, Italy.
Lotta E OikariBrain and Mental Health Program, QIMR Berghofer Medical Research Institute, Brisbane, QLD, Australia.
Lezanne OoiMolecular Horizons, School of Chemistry and Molecular Bioscience, University of Wollongong, Northfields Avenue, Wollongong, NSW, Australia.
Anthony R WhiteBrain and Mental Health Program, QIMR Berghofer Medical Research Institute, Brisbane, QLD, Australia. tony.white@qimrberghofer.edu.au.

Funding

National Health and Medical Research Council APP1125796 and 1118452QIMR Berghofer Medical Research Institute PhD Top-Up ScholarshipUniversity of Queensland PhD Scholarship
6 · The paper itself

Abstract

backgroundAmyotrophic lateral sclerosis (ALS) is a rapidly progressing neurodegenerative disorder with minimally effective treatment options. An important hurdle in ALS drug development is the non-invasive therapeutic access to the motor cortex currently limited by the presence of the blood-brain barrier (BBB). Focused ultrasound and microbubble (FUS

methodsHere we established the first FUS

resultsGenerated ALS iBECs recapitulated disease-specific hallmarks of BBB pathology, including reduced BBB integrity and permeability, and TDP-43 proteinopathy. The results also identified differences between sporadic ALS and familial (C9orf72 expansion carrying) ALS iBECs reflecting patient heterogeneity associated with disease subgroups. Studies in these models revealed successful ALS iBEC monolayer opening in vitro with no adverse cellular effects of FUS

conclusionsTogether, this study describes the first characterisation of cellular and molecular responses of ALS iBECs to FUS

Indexed as

Amyotrophic Lateral SclerosisBlood-Brain BarrierDNA-Binding ProteinsMicrobubblesAntibodiesCells, CulturedDrug Delivery SystemsEndothelial CellsHumansUltrasonic WavesAntibodiesDNA-Binding ProteinsTARDBP protein, humanAmyotrophic lateral sclerosisAntibodyBlood-brain barrierDrug deliveryFocused ultrasoundIn vitro modelTDP-43

Identifiers

PMID39138578
PMCPMC11323367

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.