PXD065345-1

PXD065345 is an original dataset announced via ProteomeXchange.

Title	Neuronal Pentraxin 2 as a Potential Biomarker for Nusinersen Therapy Response in Adults with Spinal Muscular Atrophy: A Pilot Study
Description	This project investigates cerebrospinal fluid (CSF) proteome changes in adults with spinal muscular atrophy (pwSMA) treated with the antisense oligonucleotide nusinersen. A label-free quantitative proteomic analysis was conducted on CSF samples from a subset of pwSMA (n=7) classified as clinical responders or non-responders based on motor function changes under therapy. The aim was to identify proteins differentially abundant between these groups. Candidate biomarkers emerging from this discovery phase—Neuronal Pentraxin 2 (NPTX2), Contactin 5 (CNTN5), and Anthrax Toxin Receptor 1 (ANTXR1)—were selected for follow-up analysis using orthogonal validation methods in an independent cohort. This dataset contains the primary CSF proteomic data used for the identification of potential treatment-associated protein changes in adult SMA.
HostingRepository	PRIDE
AnnounceDate	2025-08-25
AnnouncementXML	Submission_2025-08-25_09:48:10.393.xml
DigitalObjectIdentifier
ReviewLevel	Peer-reviewed dataset
DatasetOrigin	Original dataset
RepositorySupport	Unsupported dataset by repository
PrimarySubmitter	Svenja Neuhoff
SpeciesList	scientific name: Homo sapiens (Human); NCBI TaxID: 9606;
ModificationList	iodoacetamide derivatized residue
Instrument	Q Exactive HF

Revision	Datetime	Status	ChangeLog Entry
0	2025-06-23 09:12:15	ID requested
⏵ 1	2025-08-25 09:48:10	announced

Dataset with its publication pending

submitter keyword: spinal muscular atrophy

biomarker

NPTX2

nusinersen

Prof. Dr. med. Tim Hagenacker
contact affiliation	Department of Neurology and Center for Translational Neuro- and Behavioral Sciences (C-TNBS), University Hospital Essen, Hufelandstr. 55, 45147 Essen, Germany
contact email	tim.hagenacker@uk-essen.de
lab head
Svenja Neuhoff
contact affiliation	University Clinic Essen
contact email	svenja.brakemeier@gmx.de
dataset submitter

Dataset FTP location NOTE: Most web browsers have now discontinued native support for FTP access within the browser window. But you can usually install another FTP app (we recommend FileZilla) and configure your browser to launch the external application when you click on this FTP link. Or otherwise, launch an app that supports FTP (like FileZilla) and use this address: ftp://ftp.pride.ebi.ac.uk/pride/data/archive/2025/08/PXD065345

PRIDE project URI

• PRIDE
  1. PXD065345
     1. Label: PRIDE project
     2. Name: Neuronal Pentraxin 2 as a Potential Biomarker for Nusinersen Therapy Response in Adults with Spinal Muscular Atrophy: A Pilot Study