Evidence map›Paper›PMID 35886995›Full record

ArticleInternational journal of molecular sciences2022

The Capillary Morphogenesis Gene 2 Triggers the Intracellular Hallmarks of Collagen VI-Related Muscular Dystrophy.

Enrico Castroflorio, Ana Joaquina Pérez Berná, Arístides López-Márquez, Carmen Badosa, Pablo Loza-Alvarez, Mónica Roldán, Cecilia Jiménez-Mallebrera

Open access · goldAbstract read
In one paragraph

Article in International journal of molecular sciences, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed
1.4field-weighted citation impact, top 22% 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

9 citing papers in PubMed, 13 citations in OpenAlex.

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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

7 authors at 5 institutions in 1 country.

Enrico CastroflorioICFO-The Institute of Photonic Sciences, The Barcelona Institute of Science and Technology, 08860 Castelldefels, Spain.ORCID 0000-0002-6953-5420
Ana Joaquina Pérez BernáALBA Synchrotron Light Source, 08290 Cerdanyola del Vallès, Spain.
Arístides López-MárquezLaboratorio de Investigación Aplicada en Enfermedades Neuromusculares, Unidad de Patología Neuromuscular, Servicio de Neuropediatría, Institut de Recerca Sant Joan de Déu, 08950 Esplugues de Llobregat, Spain.ORCID 0000-0002-0124-3261
Carmen BadosaLaboratorio de Investigación Aplicada en Enfermedades Neuromusculares, Unidad de Patología Neuromuscular, Servicio de Neuropediatría, Institut de Recerca Sant Joan de Déu, 08950 Esplugues de Llobregat, Spain.
Pablo Loza-AlvarezICFO-The Institute of Photonic Sciences, The Barcelona Institute of Science and Technology, 08860 Castelldefels, Spain.ORCID 0000-0002-3129-1213
Mónica RoldánInstitut de Recerca Sant Joan de Déu, 08950 Esplugues de Llobregat, Spain.ORCID 0000-0002-9530-6234
Cecilia Jiménez-MallebreraLaboratorio de Investigación Aplicada en Enfermedades Neuromusculares, Unidad de Patología Neuromuscular, Servicio de Neuropediatría, Institut de Recerca Sant Joan de Déu, 08950 Esplugues de Llobregat, Spain.ORCID 0000-0001-8203-7103
Centre for Biomedical Network Research on Rare Diseases · ESInstitute of Photonic Sciences · ESALBA Synchrotron (Spain) · ESHospital Sant Joan de Déu Barcelona · ESSant Joan de Déu Research Foundation · ES

Funding

Agency for Management of Univrsity and Research Grants 2020 BP 0017Instituto de Salud Carlos III P119/0122Laser-lab Europe 871124Ministry of Economy, Industry and Competitiveness CEX0219-000910-S [MCIN/AEI/10]Spanish Centre for Biomedical Network Research on Rare Diseases ACCI2018 18-02
6 · The paper itself

Abstract

Collagen VI-related disorders (COL6-RD) represent a severe form of congenital disease for which there is no treatment. Dominant-negative pathogenic variants in the genes encoding α chains of collagen VI are the main cause of COL6-RD. Here we report that patient-derived fibroblasts carrying a common single nucleotide variant mutation are unable to build the extracellular collagen VI network. This correlates with the intracellular accumulation of endosomes and lysosomes triggered by the increased phosphorylation of the collagen VI receptor CMG2. Notably, using a CRISPR-Cas9 gene-editing tool to silence the dominant-negative mutation in patients' cells, we rescued the normal extracellular collagen VI network, CMG2 phosphorylation levels, and the accumulation of endosomes and lysosomes. Our findings reveal an unanticipated role of CMG2 in regulating endosomal and lysosomal homeostasis and suggest that mutated collagen VI dysregulates the intracellular environment in fibroblasts in collagen VI-related muscular dystrophy.

Indexed as

Collagen Type VIMuscular DystrophiesReceptors, PeptideExtracellular MatrixHumansMorphogenesisMutationANTXR2 protein, humanCollagen Type VIReceptors, PeptideCMG2collagen VImuscular dystrophypatient-derived fibroblastssuper-resolution microscopy

Identifiers

PMID35886995
PMCPMC9322809
OpenAlexW4285044094

What Socratic 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 Socratic graph.