Evidence map›Paper›PMID 34613624›Full record

ReviewMovement disorders : official journal of the Movement Disorder Society2021

Evaluation of Strategies for Measuring Lysosomal Glucocerebrosidase Activity.

Daniel Ysselstein, Tiffany J Young, Maria Nguyen, Shalini Padmanabhan, Warren D Hirst, Nicolas Dzamko, Dimitri Krainc

Open access · hybridAbstract readReview
In one paragraph

Review in Movement disorders : official journal of the Movement Disorder Society, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers.

0numbers the graph read from it
0cells of the map it votes in
27citing papers in PubMed
3.6field-weighted citation impact, top 6% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

27 citing papers in PubMed, 39 citations in OpenAlex.

  1. Intra-CNS AAV9-Molecular therapy. Advances · 2026
    Article
  2. Associations of Monocyte Glucocerebrosidase with Cognition and Cholinergic Innervation in GBA1 Parkinson's Disease.Movement disorders : official journal of the Movement Disorder Society · 2026
    Article
  3. Article
  4. Review
  5. Article
  6. Review
  7. A Common PD-RiskbioRxiv : the preprint server for biology · 2025
    Article
  8. Article
  9. Article
  10. Article
  11. Article
  12. Review
  13. Article
  14. New Antibodies to Advance Glucocerebrosidase Research.Journal of Parkinson's disease · 2024
    Article
  15. Review
  16. LRRK2 and Parkinson's disease: from genetics to targeted therapy.Annals of clinical and translational neurology · 2023
    Review
  17. Article
  18. Live cellFrontiers in cellular neuroscience · 2023
    Article
  19. Review
  20. Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

7 authors at 4 institutions in 2 countries.

Daniel YsselsteinVanqua Bio, Chicago, Illinois, USA.ORCID 0000-0003-3423-9653
Tiffany J YoungKen and Ruth Davee Department of Neurology, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.
Maria NguyenVanqua Bio, Chicago, Illinois, USA.
Shalini PadmanabhanThe Michael J. Fox Foundation for Parkinson's Research, New York, New York, USA.ORCID 0000-0002-6933-416X
Warren D HirstNeurodegenerative Diseases Research Unit, Biogen, Cambridge, Massachusetts, USA.ORCID 0000-0003-0389-8891
Nicolas DzamkoBrain and Mind Centre and Faculty of Medicine and Health, School of Medical Sciences, University of Sydney, Camperdown, New South Wales, Australia.
Dimitri KraincKen and Ruth Davee Department of Neurology, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.
Northwestern University · USBiogen (United States) · USMichael J. Fox Foundation · USThe University of Sydney · AU

Funding

Mechanistic Analysis of Genetic Modifiers in Parkinson's DiseaseR35NS122257 · NINDS · NORTHWESTERN UNIVERSITY AT CHICAGO · PI DIMITRI KRAINC · 2021 to 2026
$6.8M
The Role of Lysosomal Glucocerebrosidase in SynucleinopathiesR01NS076054 · NINDS · NORTHWESTERN UNIVERSITY AT CHICAGO · PI KRAINC, DIMITRI · 2011 to 2020
$4.1M
The role of ATP13A2/PARK9 in secretion of exosomes and alpha synucleinR37NS096241 · NINDS · NORTHWESTERN UNIVERSITY AT CHICAGO · PI KRAINC, DIMITRI · 2016 to 2020
$3.1M
NINDS NIH HHS R01 NS076054NINDS NIH HHS R35 NS122257NINDS NIH HHS R37 NS096241
6 · The paper itself

Abstract

Mutations in GBA1, which encode for the protein glucocerebrosidase (GCase), are the most common genetic risk factor for Parkinson's disease and dementia with Lewy bodies. In addition, growing evidence now suggests that the loss of GCase activity is also involved in onset of all forms of Parkinson's disease, dementia with Lewy bodies, and other dementias, such as progranulin-linked frontal temporal dementia. As a result, there is significant interest in developing GCase-targeted therapies that have the potential to stop or slow progression of these diseases. Despite this interest in GCase as a therapeutic target, there is significant inconsistency in the methodology for measuring GCase enzymatic activity in disease-modeling systems and patient populations, which could hinder progress in developing GCase therapies. In this review, we discuss the different strategies that have been developed to assess GCase activity and highlight the specific strengths and weaknesses of these approaches as well as the gaps that remain. We also discuss the current and potential role of these different methodologies in preclinical and clinical development of GCase-targeted therapies. © 2021 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.

Indexed as

GlucosylceramidaseParkinson Diseasealpha-SynucleinHumansLewy BodiesLysosomesMutationalpha-SynucleinGlucosylceramidaseGCaseGCase enzyme activityglucocerebrosidaseParkinson's disease

Identifiers

PMID34613624
PMCPMC8853444
OpenAlexW3202789603

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.