Evidence map›Paper›PMID 41078870›Full record

ArticleMolecular therapy. Methods & clinical development2025

Biodistribution of AAV1, AAV5, AAV9, and AAVDJ serotypes after intra-cisterna magna delivery in non-human primates.

Takuro Okai, Sho Sato, Hironobu Yasuno, Miyu Nakayama, Syunsuke Yamamoto, Sebastian Sjöqvist, Kentaro Otake, Masato Nakashima, Mugdha Deshpande, Elizabeth Galbreath and 3 more

Abstract read
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Intra-CNS AAV9-Molecular therapy. Advances · 2026
    Article
  2. Article
  3. Article
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

13 authors.

Takuro OkaiNeuroscience Drug Discovery Unit, Research, Takeda Pharmaceutical Company Limited, Fujisawa 251-8555, Japan.
Sho SatoCenter of Excellence for Drug Metabolism, Pharmacokinetics and Modeling, Preclinical and Translational Sciences, Research, Takeda Pharmaceutical Company Limited, Fujisawa 251-8555, Japan.
Hironobu YasunoDrug Safety Research and Evaluation, Preclinical and Translational Sciences, Research, Takeda Pharmaceutical Company Limited, Fujisawa 251-8555, Japan.
Miyu NakayamaCenter of Excellence for Drug Metabolism, Pharmacokinetics and Modeling, Preclinical and Translational Sciences, Research, Takeda Pharmaceutical Company Limited, Fujisawa 251-8555, Japan.
Syunsuke YamamotoCenter of Excellence for Drug Metabolism, Pharmacokinetics and Modeling, Preclinical and Translational Sciences, Research, Takeda Pharmaceutical Company Limited, Fujisawa 251-8555, Japan.
Sebastian SjöqvistNeuroscience Drug Discovery Unit, Research, Takeda Pharmaceutical Company Limited, Fujisawa 251-8555, Japan.
Kentaro OtakeNeuroscience Drug Discovery Unit, Research, Takeda Pharmaceutical Company Limited, Fujisawa 251-8555, Japan.
Masato NakashimaNeuroscience Drug Discovery Unit, Research, Takeda Pharmaceutical Company Limited, Fujisawa 251-8555, Japan.
Mugdha DeshpandeTakeda Development Center Americas, Cambridge, MA 02142, USA.
Elizabeth GalbreathDrug Safety Research and Evaluation, Preclinical and Translational Sciences, Research, Takeda Pharmaceutical Company Limited, Fujisawa 251-8555, Japan.
Jeong-Ho OakDrug Safety Research and Evaluation, Preclinical and Translational Sciences, Research, Takeda Pharmaceutical Company Limited, Fujisawa 251-8555, Japan.
Saku MiyamotoDrug Safety Research and Evaluation, Preclinical and Translational Sciences, Research, Takeda Pharmaceutical Company Limited, Fujisawa 251-8555, Japan.
Gabriele ProetzelNeuroscience Drug Discovery Unit, Research, Takeda Development Center Americas, Inc, Cambridge, MA 02142, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Delivering drugs effectively to the central nervous system (CNS) is a major challenge in drug development, including adeno-associated virus (AAV) gene therapy. The cerebrospinal fluid (CSF) circulates through the ventricular system and the subarachnoid space, surrounding both the brain and spinal cord, making it an attractive target for CNS drug delivery. Here, we compare intra-cisterna magna (ICM) administration of four AAV serotypes, AAV1, AAV5, AAV9, and AAVDJ, carrying

Indexed as

AAVbiodistributionCNSdorsal root ganglionGBA1gene therapyGlucosylceramidase beta 1intra-cisterna magnaNHPnon-human primate

Identifiers

PMID41078870
PMCPMC12509745

What OpenQuestion holds

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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 OpenQuestion graph.