亨廷顿舞蹈病患者脑脊液中前强啡肽衍生肽水平降低。

Cerebrospinal Fluid Levels of Prodynorphin-Derived Peptides are Decreased in Huntington's Disease.

作者信息

Al Shweiki Mhd Rami, Oeckl Patrick, Pachollek Adrian, Steinacker Petra, Barschke Peggy, Halbgebauer Steffen, Anderl-Straub Sarah, Lewerenz Jan, Ludolph Albert C, Bernhard Landwehrmeyer Georg, Otto Markus

机构信息

Department of Neurology, Ulm University Hospital, Ulm, Germany.

German Center for Neurodegenerative Diseases, Ulm, Germany.

出版信息

Mov Disord. 2021 Feb;36(2):492-497. doi: 10.1002/mds.28300. Epub 2020 Oct 2.

DOI:10.1002/mds.28300

PMID:33006791

Abstract

RESULTS

Huntington's disease (HD) is a devastating neurodegenerative disorder characterized by a selective loss of striatal medium spiny projection neurons (MSNs). Prodynorphin (PDYN) is enriched in a subpopulation of striatal MSNs. Postmortem brains of HD patients and rodent models have been demonstrated to have reduced levels of PDYN transcripts and the neuropeptide dynorphin.

RESULTS

Given the unmet need for novel pharmacodynamic HD biomarkers in the context of experimental huntingtin (htt)-lowering therapies, we investigated the levels of PDYN-derived peptides and neurofilament light (NfL) chain in the cerebrospinal fluid (CSF) from HD patients (n = 16), matched controls (n = 55), and patients with other neurodegenerative disorders (n = 70).

RESULTS

PDYN-derived peptide levels were found to be substantially decreased in HD patients (P < 0.0001 in comparison to controls), whereas the NfL levels were elevated in all neurodegenerative disorders.

CONCLUSIONS

Our study suggests decreased PDYN-derived peptide levels in the CSF as a more specific biomarker for HD in comparison to NfL. © 2020 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.

摘要