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Extracellular vesicles as biomarkers of disease progression and therapeutic response in patients with spinal muscular atrophy.

Overview

Authors: Kathy L. Poulin1, Charlotte A. René1,2,3, Ian C. Smith4, Panayiotis O. Vacratsis5, Dylan Burger6,7,8, Hugh J. McMillan9, Rashmi Kothary1,3,8,10, Theodore J. Perkins1,2,3, Jodi Warman-Chardon3,4,8,10,11,12, Robin J. Parks1,2,3,10
ORCID iDs: Robin J. Parks
  1. Regenerative Medicine Program, Ottawa Hospital Research Institute, Ottawa, ON K1H 8L6, Canada
  2. Department of Biochemistry, Microbiology and Immunology, University of Ottawa, Ottawa, ON K1H 8M5, Canada
  3. Centre for Neuromuscular Disease, University of Ottawa, Ottawa, ON K1H 8M5, Canada
  4. NeuroMuscular Centre, The Ottawa Hospital, Ottawa, ON K1Y4E9, Canada
  5. Department of Chemistry and Biochemistry, University of Windsor, Windsor, ON N9B3P4, Canada
  6. Inflammation and Chronic Disease Program, Kidney Research Centre, Ottawa Hospital Research Institute, Ottawa, ON K1H 8L6, Canada
  7. School of Pharmaceutical Sciences, University of Ottawa, Ottawa, ON K1H 8M5, Canada
  8. Department of Cellular and Molecular Medicine, University of Ottawa, Ottawa, ON K1H 8M5, Canada
  9. Department of Pediatrics, Children’s Hospital of Eastern Ontario and University of Ottawa, Ottawa, ON K1H 8L1, Canada
  10. Department of Medicine, The Ottawa Hospital and University of Ottawa, Ottawa, ON K1H 8L6, Canada
  11. Neuroscience Program, Ottawa Hospital Research Institute, Ottawa, ON K1H 8L6, Canada
  12. Department of Genetics, Children’s Hospital of Eastern Ontario, Ottawa, ON K1H 8L1, Canada
Institutions: Ottawa Hospital Research Institute (Canada); University of Ottawa (Canada); Ottawa Hospital (Canada); University of Windsor (Canada); Children's Hospital of Eastern Ontario (Canada)
Journal: Molecular therapy. Advances, volume 34, issue 2, article 201757
Dates: received 26 September 2025; accepted 8 May 2026; published online 13 May 2026
Type: Research article · Language: English
License: CC BY-NC-ND
Identifiers: DOI 10.1016/j.omta.2026.201757 · PMID 42232219 · PMCID PMC13224364 · OpenAlex W7161116725
Open access: hybrid, a free copy (OpenAlex)
Status: code on request
Categories: genetics / omics (modality), human (organism), other condition (population)
Keywords: spinal muscular atrophy, extracellular vesicle, nusinersen, proteomics, plasma, cerebrospinal fluid
Topic: Neurogenetic and Muscular Disorders Research (Genetics, Medicine), according to OpenAlex
Funding: Canadian Institutes of Health Research (PJT-178120, NHG-107767, MOP-136898, PYY-175428); Cure SMA/Cure SMA Canada; Queen Elizabeth II Graduate Scholarship in Science and Technology from the Ontario Provincial Government (Canada)
Citations: not cited yet (Europe PMC); 129 references in the paper

Abstract

The abstract is not reproduced here: the paper's license (CC BY-NC-ND) does not allow it. Read it in the paper, at the publisher or on Europe PMC.

Code

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The paper's code and data availability statement is in the Data section.

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Data

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Code and data availability statement

The paper has a code and data availability statement. Its license (CC BY-NC-ND) does not allow reproducing it here; in short, from what the harvester recognized in it:

  • it says that the data are available on request
  • it says that the code is available on request

Read it in the paper: doi.org/10.1016/j.omta.2026.201757.

Versions

The history of this record: each version stored by the harvester or made by a correction of its authors or of the maintainers of its code, and what changed in its facts. The texts of the paper (its abstract, its availability statements) are not part of it; versions that changed only those are not listed.

Version 2, 28 September 2026

  • Authors: added Robin J. Parks (0000-0002-4959-2233); removed Robin J. Parks

Version 1, 28 September 2026: the first record

Recorded: type, language, journal, volume, issue, pages, dates, 10 authors, 6 keywords, 3 funders, 129 references.

Cite

This paper

Poulin, K. L., René, C. A., Smith, I. C., Vacratsis, P. O., Burger, D., McMillan, H. J., Kothary, R., Perkins, T. J., Warman-Chardon, J., & Parks, R. J. (2026). Extracellular vesicles as biomarkers of disease progression and therapeutic response in patients with spinal muscular atrophy. Molecular therapy. Advances, 34(2), 201757. https://doi.org/10.1016/j.omta.2026.201757

BibTeX

@article{poulin2026extracellular,
author = {Poulin, Kathy L. and René, Charlotte A. and Smith, Ian C. and Vacratsis, Panayiotis O. and Burger, Dylan and McMillan, Hugh J. and Kothary, Rashmi and Perkins, Theodore J. and Warman-Chardon, Jodi and Parks, Robin J.},
title = {{Extracellular vesicles as biomarkers of disease progression and therapeutic response in patients with spinal muscular atrophy}},
journal = {Molecular therapy. Advances},
year = {2026},
month = may,
volume = {34},
number = {2},
pages = {201757},
publisher = {American Society of Gene \& Cell Therapy},
issn = {3117-387X},
doi = {10.1016/j.omta.2026.201757},
url = {https://doi.org/10.1016/j.omta.2026.201757},
pmid = {42232219},
pmcid = {PMC13224364}
}

RIS

TY - JOUR
AU - Poulin, Kathy L.
AU - René, Charlotte A.
AU - Smith, Ian C.
AU - Vacratsis, Panayiotis O.
AU - Burger, Dylan
AU - McMillan, Hugh J.
AU - Kothary, Rashmi
AU - Perkins, Theodore J.
AU - Warman-Chardon, Jodi
AU - Parks, Robin J.
TI - Extracellular vesicles as biomarkers of disease progression and therapeutic response in patients with spinal muscular atrophy
T2 - Molecular therapy. Advances
J2 - Mol Ther Adv
PY - 2026
DA - 2026/05/13
VL - 34
IS - 2
SP - 201757
SN - 3117-387X
PB - American Society of Gene & Cell Therapy
DO - 10.1016/j.omta.2026.201757
UR - https://doi.org/10.1016/j.omta.2026.201757
LA - en
ER -

CSL-JSON

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