Evidence map›Paper›PMID 35929194›Full record

ArticleEMBO molecular medicine2022

TPC2 rescues lysosomal storage in mucolipidosis type IV, Niemann-Pick type C1, and Batten disease.

Anna Scotto Rosato, Einar K Krogsaeter, Dawid Jaślan, Carla Abrahamian, Sandro Montefusco, Chiara Soldati, Barbara Spix, Maria Teresa Pizzo, Giuseppina Grieco, Julia Böck and 20 more

Open access · goldAbstract read
In one paragraph

Article in EMBO molecular medicine, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 36 papers.

0numbers the graph read from it
0cells of the map it votes in
36citing papers in PubMed
5.9field-weighted citation impact, top 3% 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

36 citing papers in PubMed, 39 citations in OpenAlex.

  1. Allosteric switching of CaThe EMBO journal · 2026
    Article
  2. Article
  3. Article
  4. TPC1-dependent control of endosomal pH and transferrin uptake determines cellular iron status.Proceedings of the National Academy of Sciences of the United States of America · 2026
    Article
  5. Article
  6. Reversible synaptic deficits in early-stage batten disease.Journal of translational medicine · 2026
    Article
  7. Article
  8. Review
  9. Review
  10. Review
  11. Review
  12. Article
  13. Article
  14. Article
  15. Article
  16. Lysosomes' fallback strategies: more than just survival or death.Frontiers in cell and developmental biology · 2025
    Review
  17. Review
  18. Review
  19. Article
  20. 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

30 authors at 10 institutions in 4 countries.

Anna Scotto Rosato *Faculty of Medicine, Walther Straub Institute of Pharmacology and Toxicology, Ludwig-Maximilians-Universität, Munich, Germany.
Einar K Krogsaeter *Faculty of Medicine, Walther Straub Institute of Pharmacology and Toxicology, Ludwig-Maximilians-Universität, Munich, Germany.ORCID 0000-0001-8232-5498
Dawid JaślanFaculty of Medicine, Walther Straub Institute of Pharmacology and Toxicology, Ludwig-Maximilians-Universität, Munich, Germany.ORCID 0000-0002-0685-7633
Carla AbrahamianFaculty of Medicine, Walther Straub Institute of Pharmacology and Toxicology, Ludwig-Maximilians-Universität, Munich, Germany.ORCID 0000-0002-0425-8004
Sandro MontefuscoTelethon Institute of Genetics and Medicine, Naples, Italy.
Chiara SoldatiTelethon Institute of Genetics and Medicine, Naples, Italy.
Barbara SpixFaculty of Medicine, Walther Straub Institute of Pharmacology and Toxicology, Ludwig-Maximilians-Universität, Munich, Germany.
Maria Teresa PizzoTelethon Institute of Genetics and Medicine, Naples, Italy.
Giuseppina GriecoTelethon Institute of Genetics and Medicine, Naples, Italy.
Julia BöckFaculty of Medicine, Walther Straub Institute of Pharmacology and Toxicology, Ludwig-Maximilians-Universität, Munich, Germany.
Amanda WyattExperimental Pharmacology, Center for Molecular Signaling (PZMS), Saarland University School of Medicine, Homburg, Germany.ORCID 0000-0003-1107-3857
Daniela WünkhausEvotec AG, Hamburg, Germany.
Marcel PassonFaculty of Medicine, Walther Straub Institute of Pharmacology and Toxicology, Ludwig-Maximilians-Universität, Munich, Germany.ORCID 0000-0002-9776-4992
Marc StieglitzDepartment of Pharmacy, Center for Drug Research, Ludwig-Maximilians-Universität, Munich, Germany.
Marco KellerDepartment of Pharmacy, Center for Drug Research, Ludwig-Maximilians-Universität, Munich, Germany.ORCID 0000-0003-4792-3980
Guido HermeyCenter for Molecular Neurobiology Hamburg (ZMNH), Institute of Molecular and Cellular Cognition, UKE, Hamburg, Germany.ORCID 0000-0003-4762-5262
Sandra MarkmannEvotec AG, Hamburg, Germany.
Doris Gruber-SchoffneggerEvotec AG, Hamburg, Germany.
Susan CotmanDepartment of Neurology, Center for Genomic Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.ORCID 0000-0003-3114-0543
Ludger JohannesCellular and Chemical Biology Department, Institut Curie, U1143 INSERM, UMR3666 CNRS, PSL Research University, Paris, France.ORCID 0000-0002-2168-0004
Dennis CrusiusInstitute for Stroke and Dementia Research (ISD), Ludwig-Maximilians-University (LMU) Hospital, Munich, Germany.
Ulrich BoehmExperimental Pharmacology, Center for Molecular Signaling (PZMS), Saarland University School of Medicine, Homburg, Germany.ORCID 0000-0003-2436-6907
Christian Wahl-SchottInstitute for Neurophysiology, Hannover Medical School, Hannover, Germany.
Martin BielDepartment of Pharmacy, Center for Drug Research, Ludwig-Maximilians-Universität, Munich, Germany.
Franz BracherDepartment of Pharmacy, Center for Drug Research, Ludwig-Maximilians-Universität, Munich, Germany.
Elvira De LeonibusTelethon Institute of Genetics and Medicine, Naples, Italy.
Elena PolishchukTelethon Institute of Genetics and Medicine, Naples, Italy.
Diego L MedinaTelethon Institute of Genetics and Medicine, Naples, Italy.ORCID 0000-0002-7347-2645
Dominik PaquetInstitute for Stroke and Dementia Research (ISD), Ludwig-Maximilians-University (LMU) Hospital, Munich, Germany.ORCID 0000-0003-2065-1639
Christian GrimmFaculty of Medicine, Walther Straub Institute of Pharmacology and Toxicology, Ludwig-Maximilians-Universität, Munich, Germany.ORCID 0000-0002-0177-5559
Ludwig-Maximilians-Universität München · DETelethon Institute Of Genetics And Medicine · ITEvotec (Germany) · DESaarland University · DECentre National de la Recherche Scientifique · FRFederico II University Hospital · ITHarvard University · USMedizinische Hochschule Hannover · DEMunich Cluster for Systems Neurology · DEUniversität Hamburg · DE

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Lysosomes are cell organelles that degrade macromolecules to recycle their components. If lysosomal degradative function is impaired, e.g., due to mutations in lysosomal enzymes or membrane proteins, lysosomal storage diseases (LSDs) can develop. LSDs manifest often with neurodegenerative symptoms, typically starting in early childhood, and going along with a strongly reduced life expectancy and quality of life. We show here that small molecule activation of the Ca

Indexed as

Lysosomal Storage DiseasesMucolipidosesNeuronal Ceroid-LipofuscinosesAnimalsChild, PreschoolHumansLysosomesMiceQuality of LifeBattenMLIVNPC1TPC2TRPML

Identifiers

PMID35929194
PMCPMC9449600
OpenAlexW4289861451

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.