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

Molecular Therapy Advances 2026 Open access

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Authors: T.C. Lund, AO Gupta, S. Kemp, D.J. Loes, D.R. Nascene, PJ Orchard
DOI: 10.1016/j.omta.2026.201702
Cited by: 1
References: 34

Abstract

= 0.0304). These results are the first to report NfL as a prognostic biomarker in response to GT for CALD.

Fields of Study & Subjects

Peroxisome Proliferator-Activated Receptors Alcoholism and Thiamine Deficiency Metabolism and Genetic Disorders

Publication details

Journal / source
Molecular Therapy Advances
Publisher
Elsevier BV
Volume
34
Issue
1
Pages
201702–201702
Publication date
18 February 2026
Language
EN