Evidence map›Paper›PMID 31293082›Full record

ArticleJournal of cellular and molecular medicine2019

Mannose 6-phosphonate labelling: A key for processing the therapeutic enzyme in Pompe disease.

Anastasia Godefroy, Morgane Daurat, Afitz Da Silva, Ilaria Basile, Khaled El Cheikh, Catherine Caillaud, Sabrina Sacconi, Benedikt Schoser, Henry-Vincent Charbonné, Magali Gary-Bobo and 3 more

Open access · goldAbstract read
In one paragraph

Article in Journal of cellular and molecular medicine, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact, top 90% 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

2 citing papers in PubMed, 4 citations in OpenAlex.

  1. Article
  2. Article
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

13 authors at 4 institutions in 2 countries.

Anastasia GodefroyIBMM, CNRS, ENSCM, University of Montpellier, Montpellier, France.
Morgane DauratIBMM, CNRS, ENSCM, University of Montpellier, Montpellier, France.
Afitz Da SilvaIBMM, CNRS, ENSCM, University of Montpellier, Montpellier, France.
Ilaria BasileNanoMedSyn, Montpellier, France.
Khaled El CheikhNanoMedSyn, Montpellier, France.
Catherine CaillaudBiochimie Métabolique et Protéique, AH-HP, Hôpital Necker Enfants-Malades and Inserm U1151, Institut Necker Enfants Malades, Université Paris-Descartes, Paris, France.
Sabrina SacconiService Système Nerveux Périphérique, Muscle et SLA, Centre Hospitalier Universitaire de Nice, Centre National de la Recherche Scientifique, Institut National de la Santé et de la Recherche Médicale, Institute for Research on Cancer and Aging of Nice, Université Côte d'Azur, Nice, France.
Benedikt SchoserDepartment of Neurology, Friedrich-Baur-Institute, Ludwig-Maximilians University Munich, Munich, Germany.
Henry-Vincent CharbonnéNanoMedSyn, Montpellier, France.
Magali Gary-BoboIBMM, CNRS, ENSCM, University of Montpellier, Montpellier, France.
Alain MorèreIBMM, CNRS, ENSCM, University of Montpellier, Montpellier, France.ORCID 0000-0002-3269-5172
Marcel GarciaIBMM, CNRS, ENSCM, University of Montpellier, Montpellier, France.
Marie MaynadierNanoMedSyn, Montpellier, France.
École Nationale Supérieure de Chimie de Montpellier · FRFriedrich Baur Stiftung · DEHôpital Necker-Enfants Malades · FRUniversité Côte d'Azur · FR

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

In the search of a better enzyme therapy in Pompe disease, the conjugation of mannose 6-phosphonates to the recombinant enzyme appeared as an enhancer of its efficacy. Here, we demonstrated that the increased efficacy of the conjugated enzyme is partly due to a higher intracellular maturation because of its insensitiveness to acid phosphatases during the routing to lysosomes.

Indexed as

AdultAnimalsCells, CulturedDisease Models, AnimalGlycogen Storage Disease Type IIHumansLysosomesMannoseMiceMuscle, SkeletalMyoblastsOrganophosphonatesMannoseOrganophosphonatesacid phosphatasesacid α-glucosidaseenzyme replacement therapyintracellular processinglysosomal storage diseasemannose 6-phosphate receptor

Identifiers

PMID31293082
PMCPMC6714136
OpenAlexW2956355544

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.