Evidence map›Paper›PMID 42662429›Full record

ArticleMolecular therapy. Advances2026

Biomarker changes in cerebral adrenoleukodystrophy after gene therapy or allogeneic hematopoietic cell transplant.

Troy C Lund, Stephan Kemp, Parmida Alikhani, Annika Willmert, Jennifer Braun, Ashish Gupta, Dan J Loes, David R Nascene, Paul J Orchard

Abstract read
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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
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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

9 authors.

Troy C LundDepartment of Pediatrics, Division of Blood and Marrow Transplantation & Cellular Therapy, University of Minnesota Medical School, Minneapolis, MN 55455, USA.
Stephan KempLaboratory Genetic Metabolic Diseases, Department of Laboratory Medicine, Amsterdam UMC Location University of Amsterdam, Amsterdam Neuroscience, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam 1105 AZ, the Netherlands.
Parmida AlikhaniDepartment of Pediatrics, Division of Blood and Marrow Transplantation & Cellular Therapy, University of Minnesota Medical School, Minneapolis, MN 55455, USA.
Annika WillmertDepartment of Pediatrics, Division of Blood and Marrow Transplantation & Cellular Therapy, University of Minnesota Medical School, Minneapolis, MN 55455, USA.
Jennifer BraunDepartment of Pediatrics, Division of Blood and Marrow Transplantation & Cellular Therapy, University of Minnesota Medical School, Minneapolis, MN 55455, USA.
Ashish GuptaDepartment of Pediatrics, Division of Blood and Marrow Transplantation & Cellular Therapy, University of Minnesota Medical School, Minneapolis, MN 55455, USA.
Dan J LoesDepartment of Diagnostic Radiology, University of Minnesota Medical Center, Minneapolis, MN 55455, USA.
David R NasceneDepartment of Diagnostic Radiology, University of Minnesota Medical Center, Minneapolis, MN 55455, USA.
Paul 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 with autologous hematopoietic cell transplant-based gene therapy (GT) or allogeneic hematopoietic cell transplantation (Allo-HCT). We compared transplant types by evaluating changes in two biomarkers, C26:0-lysophosphatidylcholine (C26:0-lysoPC) and neurofilament light chain (NfL), before and 12 months after therapy. Disease severity was quantified by Loes score on MRI. Baseline C26:0-lysoPC was 489 nmol/L for GT recipients and 487 nmol/L for Allo-HCT recipients; it decreased by 6.7% after GT and 48.4% after Allo-HCT (

Indexed as

adrenoleukodystrophybiomarkersgene therapylysophosphatidylcholineneurofilament light chaintransplant

Identifiers

PMID42662429
PMCPMC13519610

What OpenQuestion holds

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Registered trials

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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.