Neuroimmunology in globoid cell leukodystrophy: A comprehensive review including treatments, models, and neuroimmune mechanisms underlying neuropathology.

Pearl A Sutter, Zaenab Dhari, Stephen J Crocker

Abstract readReview

In one paragraph

Review in Journal of neuroimmunology, 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

3 authors.

Pearl A SutterDepartment of Neuroscience, University of Connecticut School of Medicine, Farmington, CT 06032, USA.

Zaenab DhariDepartment of Neuroscience, University of Connecticut School of Medicine, Farmington, CT 06032, USA; Mandell Center for Multiple Sclerosis, Mount Sinai Rehabilitation Hospital, Trinity Health of New England, Hartford, CT 06105, USA; Departemnt of Rehabilitative Medicine, Frank H. Netter MD School of Medicine, North Haven, CT 06473, USA.

Stephen J CrockerDepartment of Neuroscience, University of Connecticut School of Medicine, Farmington, CT 06032, USA; Department of Immunology, University of Connecticut School of Medicine, Farmington, CT 06032, USA. Electronic address: crocker@uchc.edu.

Funding

Role of CD8 T cell-mediated Pathology in Globoid Cell LeukodystrophyR01NS131327 · NINDS · UNIVERSITY OF CONNECTICUT SCH OF MED/DNT · PI Stephen J Crocker · 2023 to 2026

$2.0M

The role of microglia as antigen presenting cells in Globoid Cell LeukodystrophyF30NS129238 · NINDS · UNIVERSITY OF CONNECTICUT SCH OF MED/DNT · PI SUTTER, PEARL A · 2022 to 2025

$195k

NINDS NIH HHS F30 NS129238NINDS NIH HHS R01 NS131327

6 · The paper itself

Abstract

Globoid cell leukodystrophy (GLD), or Krabbe's disease, is a fatal genetic demyelinating disease of the central nervous system (CNS) caused by loss-of-function mutations in galactosylceramidase (GALC). As a result of the loss of GALC enzymatic activity, there is an accumulation of a toxic lipid called galactosylsphingosine, or psychosine. Current treatments have focused on restoring GALC function as a means to reduce psychosine accumulation, which show promise, however, still have limited success at improving behavioral or cognitive deficits in infants with GLD. Recent studies have discovered a role for T cells in GLD, indicating that there is a previously understudied role for the adaptive immune system as a contributing factor to GLD pathophysiology. This review aims to provide a comprehensive discussion of the current field of GLD research including treatment advances and GLD pathophysiology, with a focus on the role of neuroimmunological mechanisms contributing to GLD.

Indexed as

Leukodystrophy, Globoid CellNeuroimmunomodulationAnimalsDisease Models, AnimalGalactosylceramidaseHumansGalactosylceramidaseCD8+ T cellsDemyelinationGamma delta T cellsGloboid cell leukodystrophyInflammationKrabbe's diseaseNeuroimmunology

Identifiers

PMID40058166

DOI10.1016/j.jneuroim.2025.578573

PMCPMC13347690

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