Evidence map›Paper›PMID 41030970›Full record

ArticlebioRxiv : the preprint server for biology2025

Symptomatic treatment by a BBB-permeable AAV engineered to restore TDP-43 function slows motor neuron disease and prevents paralysis.

Aswathy Peethambaran Mallika, Jessica G Yu, Opal Sitzman, Meghraj Singh Baghel, Shruti Renganathan, Irika R Sinha, Tatiana Melnikova, Jonathan P Ling, Philip C Wong

Abstract readPreprint
PubMed Full text at PMC Publisher
In one paragraph

Article in bioRxiv : the preprint server for biology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

9 authors.

Aswathy Peethambaran MallikaDepartment of Pathology, Johns Hopkins Medicine, Baltimore, Maryland, USA.ORCID 0000-0003-0303-5156
Jessica G YuDepartment of Pathology, Johns Hopkins Medicine, Baltimore, Maryland, USA.ORCID 0009-0001-8806-5356
Opal SitzmanDepartment of Pathology, Johns Hopkins Medicine, Baltimore, Maryland, USA.
Meghraj Singh BaghelDepartment of Pathology, Johns Hopkins Medicine, Baltimore, Maryland, USA.ORCID 0000-0003-3988-6438
Shruti RenganathanDepartment of Pathology, Johns Hopkins Medicine, Baltimore, Maryland, USA.ORCID 0009-0008-6079-1138
Irika R SinhaDepartment of Pathology, Johns Hopkins Medicine, Baltimore, Maryland, USA.ORCID 0000-0001-6440-7019
Tatiana MelnikovaDepartment of Pathology, Johns Hopkins Medicine, Baltimore, Maryland, USA.
Jonathan P LingDepartment of Pathology, Johns Hopkins Medicine, Baltimore, Maryland, USA.ORCID 0000-0003-1927-9729
Philip C WongDepartment of Pathology, Johns Hopkins Medicine, Baltimore, Maryland, USA.ORCID 0000-0001-8162-3274

Funding

TDP-43 Proteinopathy in ALS-FTD: Mechanism, Target Validation and BiomarkerR01NS095969 · NINDS · JOHNS HOPKINS UNIVERSITY · PI PHILIP C WONG · 2016 to 2026
$2.4M
Generation and characterization of a mouse model exhibiting beta-amyloidosis and tauopathy with nuclear depletion of TDP-43R33NS115161 · NINDS · JOHNS HOPKINS UNIVERSITY · PI WONG, PHILIP C · 2022 to 2023
$1.3M
NINDS NIH HHS R01 NS095969NINDS NIH HHS R33 NS115161
6 · The paper itself

Abstract

TAR DNA-binding protein 43kDa (TDP-43) dysfunction is an early pathogenic mechanism that underlies amyotrophic lateral sclerosis (ALS), a devastating neurodegenerative disorder that lacks disease modifying therapies. We previously developed a mouse model in which TDP-43 is selectively deleted from motor neurons (

Indexed as

Amyotrophic Lateral SclerosisBlood-brain-barrier permeable AAVCryptic exonsGene therapyMotor neuronSplicing repressorSymptomatic treatmentTDP-43 autoregulatory elementTDP-43 dysfunction

Identifiers

PMID41030970
PMCPMC12478394

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

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 OpenQuestion graph.