Evidence map›Paper›PMID 36568275›Full record

ArticleFrontiers in molecular neuroscience2022

Dual-function AAV gene therapy reverses late-stage Canavan disease pathology in mice.

Dominik Fröhlich, Elizabeth Kalotay, Georg von Jonquieres, Andre Bongers, Brendan Lee, Alexandra K Suchowerska, Gary D Housley, Matthias Klugmann

Open access · goldAbstract read
In one paragraph

Article in Frontiers in molecular neuroscience, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.

0numbers the graph read from it
0cells of the map it votes in
12citing papers in PubMed
2.8field-weighted citation impact, top 9% of its field
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

12 citing papers in PubMed, 17 citations in OpenAlex.

  1. Article
  2. Article
  3. Discovery ofACS medicinal chemistry letters · 2026
    Article
  4. Current clinical applications of AAV-mediated gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  5. Review
  6. Homozygous Paternally InheritedMolecular syndromology · 2024
    Article
  7. Review
  8. Review
  9. tRNA therapeutics for genetic diseases.Nature reviews. Drug discovery · 2024
    Review
  10. Leukodystrophy Imaging: Insights for Diagnostic Dilemmas.Medical sciences (Basel, Switzerland) · 2024
    Review
  11. Review
  12. 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

8 authors at 1 institution in 2 countries.

Dominik FröhlichTranslational Neuroscience Facility and Department of Physiology, School of Biomedical Sciences, University of New South Wales, Sydney, NSW, Australia.
Elizabeth KalotayTranslational Neuroscience Facility and Department of Physiology, School of Biomedical Sciences, University of New South Wales, Sydney, NSW, Australia.
Georg von JonquieresTranslational Neuroscience Facility and Department of Physiology, School of Biomedical Sciences, University of New South Wales, Sydney, NSW, Australia.
Andre BongersBiological Resources Imaging Laboratory, University of New South Wales, Sydney, NSW, Australia.
Brendan LeeBiological Resources Imaging Laboratory, University of New South Wales, Sydney, NSW, Australia.
Alexandra K SuchowerskaTranslational Neuroscience Facility and Department of Physiology, School of Biomedical Sciences, University of New South Wales, Sydney, NSW, Australia.
Gary D HousleyTranslational Neuroscience Facility and Department of Physiology, School of Biomedical Sciences, University of New South Wales, Sydney, NSW, Australia.
Matthias KlugmannTranslational Neuroscience Facility and Department of Physiology, School of Biomedical Sciences, University of New South Wales, Sydney, NSW, Australia.
UNSW Sydney · AU

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The leukodystrophy Canavan disease is a fatal white matter disorder caused by loss-of-function mutations of the aspartoacylase-encoding

Indexed as

adeno-associated virusASPAaspartoacylaseCanavan diseasegene therapyleukodystrophyNAA (N-acetyl-L-aspartate)NAT8L

Identifiers

PMID36568275
PMCPMC9772617
OpenAlexW4311853198

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.