CSF and plasma neuro-biomarker dynamics after gene therapy for cerebral adrenoleukodystrophy.

T C Lund, A O Gupta, S Kemp, D J Loes, D R Nascene, P J Orchard

Abstract read

In one paragraph

Article in Molecular therapy. Advances, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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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

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3 · Its place in the literature

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0 citing papers in PubMed.

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4 · The record

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5 · Who and what money

Authors and funding

6 authors.

T C LundDepartment of Pediatrics, Division of Blood and Marrow Transplantation & Cellular Therapy, University of Minnesota Medical School, Minneapolis, MN 55455, USA.

A O GuptaDepartment of Pediatrics, Division of Blood and Marrow Transplantation & Cellular Therapy, University of Minnesota Medical School, Minneapolis, MN 55455, USA.

S KempLaboratory Genetic Metabolic Diseases, Department of Laboratory Medicine, Amsterdam UMC Location University of Amsterdam, Amsterdam Neuroscience, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam, the Netherlands.

D J LoesDepartment of Diagnostic Radiology, University of Minnesota Medical Center, Minneapolis, MN 55455, USA.

D R NasceneDepartment of Diagnostic Radiology, University of Minnesota Medical Center, Minneapolis, MN 55455, USA.

P J OrchardDepartment of Pediatrics, Division of Blood and Marrow Transplantation & Cellular Therapy, University of Minnesota Medical School, Minneapolis, MN 55455, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Cerebral adrenoleukodystrophy (CALD) is a progressive, immune-inflammatory neurologic disease that can be arrested through allogeneic or gene-corrected hematopoietic cell transplant. Biomarkers of disease during transplant have not been explored. We assessed 20 exploratory biomarkers, consisting of the matrix metalloproteinase (MMP) family, vascular factors, neuroinflammation, and lipids, to determine changes from baseline pre-transplant to 2 years after gene therapy (GT). We found the most robust changes with the plasma neuroinflammatory markers-neurofilament light chain (NfL) and glial fibrillary acidic protein (GFAP). Pre-GT plasma NfL had the strongest correlation with disease burden quantified on MRI by Loes score (R

Indexed as

adrenoleukodystrophybiomarkersgene therapyneurofilament light chaintransplant

Identifiers

PMID42137293

DOI10.1016/j.omta.2026.201702

PMCPMC13148947

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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.