Evidence map›Paper›PMID 34571934›Full record

ArticleCells2021

Substrate Reduction Therapy Reverses Mitochondrial, mTOR, and Autophagy Alterations in a Cell Model of Gaucher Disease.

Yanyan Peng, Benjamin Liou, Yi Lin, Venette Fannin, Wujuan Zhang, Ricardo A Feldman, Kenneth D R Setchell, Gregory A Grabowski, Ying Sun

Open access · goldAbstract read
In one paragraph

Article in Cells, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.

0numbers the graph read from it
0cells of the map it votes in
15citing papers in PubMed
1.3field-weighted citation impact, top 20% 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

15 citing papers in PubMed, 19 citations in OpenAlex.

  1. Article
  2. Article
  3. Article
  4. Review
  5. Article
  6. Article
  7. iPSC-derived neural precursor cells engineeringMolecular therapy. Methods & clinical development · 2023
    Article
  8. Article
  9. Review
  10. PGRN deficiency exacerbates, whereas a brain penetrant PGRN derivative protects,Proceedings of the National Academy of Sciences of the United States of America · 2023
    Article
  11. Article
  12. Review
  13. Article
  14. Article
  15. Neuronopathic Gaucher disease: Beyond lysosomal dysfunction.Frontiers in molecular neuroscience · 2022
    Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

9 authors at 2 institutions in 1 country.

Yanyan PengDivision of Human Genetics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA.
Benjamin LiouDivision of Human Genetics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA.
Yi LinDivision of Human Genetics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA.ORCID 0000-0001-8209-1185
Venette FanninDivision of Human Genetics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA.
Wujuan ZhangDepartment of Pathology, Clinical Mass Spectrometry Laboratory, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA.
Ricardo A FeldmanDepartment of Microbiology and Immunology, University of Maryland School of Medicine, Baltimore, MD 21201, USA.
Kenneth D R SetchellDepartment of Pathology, Clinical Mass Spectrometry Laboratory, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA.
Gregory A GrabowskiDivision of Human Genetics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA.
Ying SunDivision of Human Genetics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA.
Cincinnati Children's Hospital Medical Center · USUniversity of Maryland, Baltimore · US

Funding

The Role of PGRN Growth Factor in Osteoarthritis. - Renewal - 1 - Revision - 2R01AR061484 · NIAMS · NEW YORK UNIVERSITY SCHOOL OF MEDICINE · PI LIU, CHUANJU · 2011 to 2020
$4.8M
Progranulin: A Novel Gene in Gaucher DiseasesR01NS103931 · NINDS · YALE UNIVERSITY · PI Chuanju Liu, Ying Sun · 2017 to 2026
$4.3M
Gaucher disease:Treatment of neurodegenerative diseaseR01NS086134 · NINDS · CINCINNATI CHILDRENS HOSP MED CTR · PI PAN, DAO · 2013 to 2017
$2.1M
The Development of Small Molecule Inhibitors for Gaucher Disease Type 3UH2NS092981 · NINDS · UNIVERSITY OF MICHIGAN AT ANN ARBOR · PI LARSEN, SCOTT D, SHAYMAN, JAMES ALAN · 2015 to 2015
$631k
A new mouse model to study GBA1 mutation-associated diseases with multiple organs involvementR21OD033660 · OD · CINCINNATI CHILDRENS HOSP MED CTR · PI LIU, CHUANJU, SUN, YING · 2022 to 2023
$462k
Brain organoid modeling of Gaucher disease for identification of pathogenic pathways and therapy developmentR21HD102788 · NICHD · CINCINNATI CHILDRENS HOSP MED CTR · PI SUN, YING · 2020 to 2021
$437k
NIAMS NIH HHS R01 AR061484NICHD NIH HHS R21 HD102788NIH HHS R21 OD033660NINDS NIH HHS R01 NS086134NINDS NIH HHS R01 NS103931NINDS NIH HHS UH2 NS092981
6 · The paper itself

Abstract

Substrate reduction therapy (SRT) in clinic adequately manages the visceral manifestations in Gaucher disease (GD) but has no direct effect on brain disease. To understand the molecular basis of SRT in GD treatment, we evaluated the efficacy and underlying mechanism of SRT in an immortalized neuronal cell line derived from a

Indexed as

AutophagyAnimalsGaucher DiseaseGlucosylceramidaseGlycoside Hydrolase InhibitorsLysosomesMechanistic Target of Rapamycin Complex 1MiceMice, Inbred C57BLMice, KnockoutMitochondriaNeuronsTOR Serine-Threonine KinasesGlucosylceramidaseGlycoside Hydrolase InhibitorsMechanistic Target of Rapamycin Complex 1mTOR protein, mouseTOR Serine-Threonine Kinasescell biologyglucosylceramideglucosylsphingosinelysosomal storage disordersneurons

Identifiers

PMID34571934
PMCPMC8466461
OpenAlexW3198299654

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.