Features of Congenital Arthrogryposis Due to Abnormalities in Collagen Homeostasis, a Scoping Review.

collagen congenital arthrogryposis contractures genotype phenotype

Journal

International journal of molecular sciences
ISSN: 1422-0067
Titre abrégé: Int J Mol Sci
Pays: Switzerland
ID NLM: 101092791

Informations de publication

Date de publication:
31 Aug 2023
Historique:
received: 15 07 2023
revised: 30 08 2023
accepted: 30 08 2023
medline: 11 9 2023
pubmed: 9 9 2023
entrez: 9 9 2023
Statut: epublish

Résumé

Congenital arthrogryposis (CA) refers to the presence of multiple contractures at birth. It is a feature of several inherited syndromes, notable amongst them are disorders of collagen formation. This review aims to characterize disorders that directly or indirectly impact collagen structure and function leading to CA in search for common phenotypic or pathophysiological features, possible genotype-phenotype correlation, and potential novel treatment approaches based on a better understanding of the underlying pathomechanism. Nine genes, corresponding to five clinical phenotypes, were identified after a literature search. The most notable trend was the extreme phenotype variability. Clinical features across all syndromes ranged from subtle with minimal congenital contractures, to severe with multiple congenital contractures and extra-articular features including skin, respiratory, or other manifestations. Five of the identified genes were involved in the function of the Lysyl Hydroxylase 2 or 3 enzymes, which enable the hydroxylation and/or glycosylation of lysyl residues to allow the formation of the collagen superstructure. Whilst current treatment approaches are post-natal surgical correction, there are also potential in-utero therapies being developed. Cyclosporin A showed promise in treating collagen VI disorders although there is an associated risk of immunosuppression. The treatments that could be in the clinical trials soon are the splice correction therapies in collagen VI-related disorders.

Identifiants

pubmed: 37686358
pii: ijms241713545
doi: 10.3390/ijms241713545
pmc: PMC10487887
pii:
doi:

Substances chimiques

Collagen 9007-34-5

Types de publication

Journal Article Review

Langues

eng

Sous-ensembles de citation

IM

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Auteurs

Sarah MacKenzie Picker (SM)

Northumbria Healthcare Trust, North Shields NE29 8NH, UK.

George Parker (G)

Newcastle University Medical School, Newcastle NE2 4HH, UK.

Paul Gissen (P)

National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London, London WC1N 1EH, UK.
Genetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.

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