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Elevated plasma and CSF neurofilament light chain concentrations are stabilized in response to mutant huntingtin lowering in the brains of Huntington’s disease mice

Abstract Background Therapeutic approaches aimed at lowering toxic mutant huntingtin (mHTT) levels in the brain can reverse disease phenotypes in animal models of Huntington's disease (HD) and are currently being evaluated in clinical trials. Sensitive and dynamic response biomarkers are needed to a...

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Principais autores: Nicholas S. Caron, Lauren M. Byrne, Fanny L. Lemarié, Jeffrey N. Bone, Amirah E.-E. Aly, Seunghyun Ko, Christine Anderson, Lorenzo L. Casal, Austin M. Hill, David J. Hawellek, Peter McColgan, Edward J. Wild, Blair R. Leavitt, Michael R. Hayden
Format: Artigo
Sprog:Inglês
Udgivet: BMC 2024-10-01
Serier:Translational Neurodegeneration
Fag:
Neurofilament light chain
Response biomarker
Huntington's disease
Huntingtin lowering
Antisense oligonucleotide
Cerebrospinal fluid
Online adgang:https://doi.org/10.1186/s40035-024-00443-8
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