Evidence map›Paper›PMID 40950145›Full record

ArticlebioRxiv : the preprint server for biology2025

Broad brain biodistribution conferred by an AAV to restore TDP-43 function mitigates Frontotemporal Demenia-like deficits.

Tianyu Cao, Rashmi Thapa, Rongsong Liu, Aswathy Peethambaran Mallika, Meghraj Singh Baghel, Yijun Wei, Irika R Sinha, Grace D Burns, Xinrui Wen, Bo Pang and 4 more

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

14 authors.

Tianyu CaoDepartment of Pathology, The Johns Hopkins University School of Medicine.ORCID 0000-0002-5299-847X
Rashmi ThapaDepartment of Zoology and Physiology, University of Wyoming.
Rongsong LiuDepartment of Mathematics and Statistics, University of Wyoming.
Aswathy Peethambaran MallikaDepartment of Pathology, The Johns Hopkins University School of Medicine.ORCID 0000-0003-0303-5156
Meghraj Singh BaghelDepartment of Pathology, The Johns Hopkins University School of Medicine.ORCID 0000-0003-3988-6438
Yijun WeiDepartment of Pathology, The Johns Hopkins University School of Medicine.ORCID 0009-0002-3656-9998
Irika R SinhaDepartment of Pathology, The Johns Hopkins University School of Medicine.ORCID 0000-0001-6440-7019
Grace D BurnsDepartment of Pathology, The Johns Hopkins University School of Medicine.ORCID 0000-0001-8691-779X
Xinrui WenDepartment of Pathology, The Johns Hopkins University School of Medicine.ORCID 0000-0003-2667-2983
Bo PangDepartment of Pathology, The Johns Hopkins University School of Medicine.
Jonathan P LingDepartment of Pathology, The Johns Hopkins University School of Medicine.ORCID 0000-0003-1927-9729
Da-Ting LinDepartment Intramural Research Program, National Institute on Drug Abuse.
Yun LiDepartment of Zoology and Physiology, University of Wyoming.
Philip C WongDepartment of Pathology, The Johns Hopkins University School of Medicine.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
Loss of TDP-43 disrupts the prefrontal neural activity and circuitry: relevance for TDP-43 linked ADRDR01NS129878 · NINDS · UNIVERSITY OF WYOMING · PI YUN LI, PHILIP C WONG · 2025 to 2026
$1.5M
Functional Validation of TDP-43 splicing repression for frontotemporal degenerationUG3NS115608 · NINDS · JOHNS HOPKINS UNIVERSITY · PI WONG, PHILIP C · 2019 to 2019
$1.5M
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 R01 NS129878NINDS NIH HHS R33 NS115161NINDS NIH HHS UG3 NS115608
6 · The paper itself

Abstract

TDP-43 dysfunction is an early pathogenic determinant of frontotemporal lobar degeneration with TDP-43 pathology (FTLD-TDP), a devastating disorder currently without effective therapy. Here, we exploit a blood-brain-barrier (BBB)-permeable AAV (AAV-PHP.eB) that confers broad brain biodistribution to restore TDP-43 function in a TDP-43 deficient model (

Indexed as

AAVAD-TDPbiodistributioncryptic exonsforebrain neuronFTLD-TDPgene therapyhyperactivityLATEmemory deficitmouse modelsplicing repressorTDP-43 dysfunction

Identifiers

PMID40950145
PMCPMC12424654

What Socratic 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 Socratic graph.