Ablating N‐acetylaspartate prevents leukodystrophy in a C anavan disease model
Open accessAbstract
Canavan disease is caused by inactivating ASPA (aspartoacylase) mutations that prevent cleavage of N-acetyl-L-aspartate (NAA), resulting in marked elevations in central nervous system (CNS) NAA and progressively worsening leukodystrophy. We now report that ablating NAA synthesis by constitutive genetic disruption of Nat8l (N-acetyltransferase-8 like) permits normal CNS myelination and prevents leukodystrophy in a murine Canavan disease model.
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
NAA synthase knockdown appears a viable means to rescue gross pathology and vacuolation.
“with NAA synthase knockdown appearing a viable means to rescue this.22,23”
From Preclinical biodistribution, tropism, and efficacy of oligotropic AAV/Olig001 in a mouse model of congenital white matter disease (2021) · cites this paper as [23]
The Results (Aspanur7/nur7/Nat8lKO/KO Mice; page unavailable) report no astroglial vacuolation or demyelination in Canavan-model mice unable to synthesize NAA. But the study used constitutive Nat8l knockout, not knockdown, and showed prevention of pathology rather than rescue of established pathology. The Discussion proposes NAT8L inhibitors as possible adjunctive therapies but does not test them. (LLM, conf=0.88)
Experimental — accuracy not yet measuredChronically elevated NAA in Canavan disease is proposed to directly cause gross pathology and vacuolation.
“Chronically elevated NAA in Canavan disease is proposed to directly cause gross pathology and vacuolation”
From Preclinical biodistribution, tropism, and efficacy of oligotropic AAV/Olig001 in a mouse model of congenital white matter disease (2021) · cites this paper as [23]
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
The citation lists haven’t been fetched yet
References 21 · Cited by 52
- Authors
- Guo, F., Bannerman, P., Mills Ko, E., Miers, L., Xu, J., Burns, T., Li, S., Freeman, E., McDonough, J. A., & Pleasure, D.
- Affiliations
- Shriners Hospitals for Children - Northern California (US)
- Institute for Pediatric Regenerative Medicine University of California, Davis School of Medicine and Shriners Hospitals for Children Northern California Sacramento CA
- Kent State University (US)
- Corresponding author
- Pleasure, David
- Venue
- Annals of Neurology
- Publisher
- Wiley
- Published
- 2015-03-27
- Volume
- 77
- Issue
- 5
- Pages
- 884-888
- ISSN
- 0364-5134 (ISSN-L)
- 1531-8249
- PMC
- PMC11131957
- OpenAlex
- W1955574538
- Cited by
- 52
- References
- 21
- Citations by year
- 2026: 1 · 2024: 3 · 2023: 3 · 2022: 7 · 2021: 6 · 2020: 4 · 2019: 3 · 2018: 3 · 2017: 7 · 2016: 11 · 2015: 4
- FWCI
- 6.89
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Annals of Neurology (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- eScholarship (California Digital Library) (submittedVersion)
- Funders
- Shriners Hospitals for Children
- European Leukodystrophy Association
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Clinical Biochemistry › Metabolism and Genetic Disorders
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Biochemistry › Amino Acid Enzymes and Metabolism
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Biochemical and Molecular Research
- Keywords
- Canavan disease, N-acetylaspartate, aspartoacylase, leukodystrophy, NAT8L, myelination, mouse model
- MeSH terms
- Animals, Aspartic Acid, Disease Models, Animal, Female, Male, Canavan Disease, Mice, Knockout, Mice
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
