Evidence map›Paper›PMID 35636725›Full record

ReviewDrug discovery today2022

The pathogenesis of, and pharmacological treatment for, Canavan disease.

Huijun Wei, John R Moffett, Man Amanat, Ali Fatemi, Takashi Tsukamoto, Aryan M Namboodiri, Barbara S Slusher

Open access · greenAbstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Drug discovery today, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.

0numbers the graph read from it
0cells of the map it votes in
18citing papers in PubMed
3.3field-weighted citation impact, top 7% 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

18 citing papers in PubMed, 25 citations in OpenAlex.

  1. Review
  2. The Emerging Role of N-Acetylaspartate in Cancer.International journal of molecular sciences · 2026
    Review
  3. Article
  4. Article
  5. Discovery ofACS medicinal chemistry letters · 2026
    Article
  6. Article
  7. Article
  8. Much ado about NAA-thing.Nature metabolism · 2025
    Article
  9. Article
  10. Article
  11. Homozygous Paternally InheritedMolecular syndromology · 2024
    Article
  12. Review
  13. Article
  14. Review
  15. Article
  16. Review
  17. Article
  18. 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

7 authors at 4 institutions in 1 country.

Huijun WeiJohns Hopkins Drug Discovery, Johns Hopkins University School of Medicine, 855 N. Wolfe Street, Baltimore, MD 21205, USA; Department of Neurology, Johns Hopkins University School of Medicine, 855 N. Wolfe Street, Baltimore, MD 21205, USA; Department of Pharmacology and Molecular Science, Johns Hopkins University School of Medicine, 855 N. Wolfe Street, Baltimore, MD 21205, USA.
John R MoffettDepartment of Anatomy, Physiology and Genetics and Neuroscience Program, Uniformed Services University of the Health Sciences, 4301 Jones Bridge Rd., Bethesda, MD 20814, USA. Electronic address: john.moffett@usuhs.edu.
Man AmanatKennedy Krieger Institute, Baltimore, MD 21205, USA.
Ali FatemiDepartment of Neurology, Johns Hopkins University School of Medicine, 855 N. Wolfe Street, Baltimore, MD 21205, USA; Department of Behavioral Science, Johns Hopkins University School of Medicine, 855 N. Wolfe Street, Baltimore, MD 21205, USA; Department of Pediatrics, Johns Hopkins University School of Medicine, 855 N. Wolfe Street, Baltimore, MD 21205, USA; Kennedy Krieger Institute, Baltimore, MD 21205, USA.
Takashi TsukamotoJohns Hopkins Drug Discovery, Johns Hopkins University School of Medicine, 855 N. Wolfe Street, Baltimore, MD 21205, USA; Department of Neurology, Johns Hopkins University School of Medicine, 855 N. Wolfe Street, Baltimore, MD 21205, USA.
Aryan M NamboodiriDepartment of Anatomy, Physiology and Genetics and Neuroscience Program, Uniformed Services University of the Health Sciences, 4301 Jones Bridge Rd., Bethesda, MD 20814, USA.
Barbara S SlusherJohns Hopkins Drug Discovery, Johns Hopkins University School of Medicine, 855 N. Wolfe Street, Baltimore, MD 21205, USA; Department of Neurology, Johns Hopkins University School of Medicine, 855 N. Wolfe Street, Baltimore, MD 21205, USA; Department of Pharmacology and Molecular Science, Johns Hopkins University School of Medicine, 855 N. Wolfe Street, Baltimore, MD 21205, USA; Department of Oncology, Johns Hopkins University School of Medicine, 855 N. Wolfe Street, Baltimore, MD 21205, USA; Department of Medicine, Johns Hopkins University School of Medicine, 855 N. Wolfe Street, Baltimore, MD 21205, USA; Department of Psychiatry, Johns Hopkins University School of Medicine, 855 N. Wolfe Street, Baltimore, MD 21205, USA. Electronic address: bslusher@jhmi.edu.
Johns Hopkins University · USUniformed Services University of the Health Sciences · USJohns Hopkins Medicine · USKennedy Krieger Institute · US

Funding

High throughput screen for discovery of N-acetyltransferase 8 Like (NAT8L) inhibitorsR61NS119659 · NINDS · JOHNS HOPKINS UNIVERSITY · PI SLUSHER, BARBARA STAUCH, TSUKAMOTO, TAKASHI · 2021 to 2022
$813k
Development of NAT8L inhibitors for the Treatment of Canavan DiseaseR61NS136652 · NINDS · JOHNS HOPKINS UNIVERSITY · PI SLUSHER, BARBARA STAUCH, TSUKAMOTO, TAKASHI · 2024 to 2025
$790k
CANAVAN DISEASE PATHOGENESIS AND TREATMENTR01NS039387 · NINDS · HENRY M. JACKSON FDN FOR THE ADV MIL/MED · PI NAMBOODIRI, ARYAN MANGALAM · 2000 to 2002
$534k
Acetate Supplementation as a therapeutic strategy for Canavan diseaseR21NS084206 · NINDS · HENRY M. JACKSON FDN FOR THE ADV MIL/MED · PI NAMBOODIRI, ARYAN MANGALAM · 2014 to 2015
$418k
High Throughput Screen for Discovery of N-Acetyltransferase 8 Like (NAT8L) InhibitorsR33NS119659 · NINDS · JOHNS HOPKINS UNIVERSITY · PI SLUSHER, BARBARA STAUCH, TSUKAMOTO, TAKASHI · 2023 to 2023
$409k
Canavan Disease: Pathogenesis and TreatmentR56NS039387 · NINDS · HENRY M. JACKSON FDN FOR THE ADV MIL/MED · PI NAMBOODIRI, ARYAN MANGALAM · 2007 to 2008
$346k
NINDS NIH HHS R01 NS039387NINDS NIH HHS R21 NS084206NINDS NIH HHS R33 NS119659NINDS NIH HHS R56 NS039387NINDS NIH HHS R61 NS119659NINDS NIH HHS R61 NS136652
6 · The paper itself

Abstract

Canavan disease (CD) is an inherited leukodystrophy resulting from mutations in the gene encoding aspartoacylase (ASPA). ASPA is highly expressed in oligodendrocytes and catalyzes the cleavage of N-acetylaspartate (NAA) to produce aspartate and acetate. In this review, we examine the pathologies and clinical presentation in CD, the metabolism and transportation of NAA in the brain, and the hypothetical mechanisms whereby ASPA deficiency results in dysmyelination and a failure of normal brain development. We also discuss therapeutic options that could be used for the treatment of CD.

Indexed as

Canavan DiseaseAmidohydrolasesAnimalsBrainDisease Models, AnimalOligodendrogliaAmidohydrolasesAspartate N-acetyltransferase (Asp-NAT)Aspartoacylase (ASPA)Canavan disease (CD)N-acetylaspartate (NAA)NaDC3

Identifiers

PMID35636725
PMCPMC11806932
OpenAlexW4281692218

What OpenQuestion holds

Textmetadata
LicenceTDM
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.