Skewed X-chromosome inactivation drives the proportion of

Respiratory Tract Diseases X-Linked Genetic Diseases

Journal

Journal of medical genetics
ISSN: 1468-6244
Titre abrégé: J Med Genet
Pays: England
ID NLM: 2985087R

Informations de publication

Date de publication:
26 Feb 2024
Historique:
received: 19 10 2023
accepted: 10 02 2024
medline: 27 2 2024
pubmed: 27 2 2024
entrez: 26 2 2024
Statut: aheadofprint

Résumé

Primary ciliary dyskinesia (PCD) is a rare airway disorder caused by defective motile cilia. Only male patients have been reported with pathogenic mutations in X-linked XCI patterns of six mothers of male patients with The mothers' phenotypes ranged from absence of symptoms to mild/moderate or severe airway phenotypes, closely reflecting their XCI pattern. Analyses of the symptomatic mothers' airway ciliated cells revealed the coexistence of normal cells and cells with immotile cilia lacking dynein arms, whose ratio closely mirrored their XCI pattern. This study highlights the importance of searching for heterozygous pathogenic

Sections du résumé

BACKGROUND BACKGROUND
Primary ciliary dyskinesia (PCD) is a rare airway disorder caused by defective motile cilia. Only male patients have been reported with pathogenic mutations in X-linked
METHODS METHODS
XCI patterns of six mothers of male patients with
RESULTS RESULTS
The mothers' phenotypes ranged from absence of symptoms to mild/moderate or severe airway phenotypes, closely reflecting their XCI pattern. Analyses of the symptomatic mothers' airway ciliated cells revealed the coexistence of normal cells and cells with immotile cilia lacking dynein arms, whose ratio closely mirrored their XCI pattern.
CONCLUSION CONCLUSIONS
This study highlights the importance of searching for heterozygous pathogenic

Identifiants

pubmed: 38408845
pii: jmg-2023-109700
doi: 10.1136/jmg-2023-109700
pii:
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Informations de copyright

© Author(s) (or their employer(s)) 2024. No commercial re-use. See rights and permissions. Published by BMJ.

Déclaration de conflit d'intérêts

Competing interests: None declared.

Auteurs

Lucie Thomas (L)

Childhood Genetic Diseases, Sorbonne Université, Inserm, Hôpital Armand-Trousseau, Paris, F-75012, France.

Laurence Cuisset (L)

Service de Médecine Génomique, Assistance Publique Hôpitaux de Paris (AP-HP), Université de Paris, Hôpital Cochin, Paris, F-75014, France.

Jean-Francois Papon (JF)

Service d'Oto-Rhino-Laryngologie et de Chirurgie Cervico-Faciale, AP-HP, Hôpital Bicêtre, Le Kremlin-Bicêtre, F-94270, France.
Institut Mondor de Recherche Biomédicale, Université Paris-Est Créteil, Inserm U955, CNRS ERL7240, Hôpital Henri-Mondor, Créteil, F-94010, France.

Aline Tamalet (A)

Département de Pneumologie Pédiatrique, Centre National de Référence des Maladies Respiratoires Rares RespiRare, AP-HP, Sorbonne Université, Hôpital Armand-Trousseau Hospital, Paris, F-75012, France.

Isabelle Pin (I)

Pédiatrie, CHU Grenoble Alpes, Grenoble, F-38500, France.

Rola Abou Taam (R)

Service de Pneumologie et Allergologie Pédiatriques, AP-HP, Hôpital Necker-Enfants Malades, Paris, F-75015, France.

Catherine Faucon (C)

Service d'Anatomopathologie, Laboratoire de Microscopie Electronique, Centre Hospitalier Intercommunal de Créteil, Créteil, F-94000, France.

Guy Montantin (G)

Génétique moléculaire, AP-HP, Hôpital Armand-Trousseau, Paris, F-75012, Paris.

Sylvie Tissier (S)

Génétique moléculaire, AP-HP, Hôpital Armand-Trousseau, Paris, F-75012, Paris.

Philippe Duquesnoy (P)

Childhood Genetic Diseases, Sorbonne Université, Inserm, Hôpital Armand-Trousseau, Paris, F-75012, France.

Florence Dastot-Le Moal (F)

Génétique moléculaire, AP-HP, Hôpital Armand-Trousseau, Paris, F-75012, Paris.

Bruno Copin (B)

Childhood Genetic Diseases, Sorbonne Université, Inserm, Hôpital Armand-Trousseau, Paris, F-75012, France.
Génétique moléculaire, AP-HP, Hôpital Armand-Trousseau, Paris, F-75012, Paris.

Nathalie Carion (N)

Service de Médecine Génomique, Assistance Publique Hôpitaux de Paris (AP-HP), Université de Paris, Hôpital Cochin, Paris, F-75014, France.

Bruno Louis (B)

Institut Mondor de Recherche Biomédicale, Université Paris-Est Créteil, Inserm U955, CNRS ERL7240, Hôpital Henri-Mondor, Créteil, F-94010, France.

Sandra Chantot-Bastaraud (S)

Childhood Genetic Diseases, Sorbonne Université, Inserm, Hôpital Armand-Trousseau, Paris, F-75012, France.
Génétique chromosomique, AP-HP, Hôpital Trousseau, Paris, F-75012, France.

Jean-Pierre Siffroi (JP)

Childhood Genetic Diseases, Sorbonne Université, Inserm, Hôpital Armand-Trousseau, Paris, F-75012, France.
Génétique chromosomique, AP-HP, Hôpital Trousseau, Paris, F-75012, France.

Rana Mitri (R)

Service d'Anatomopathologie, Laboratoire de Microscopie Electronique, Centre Hospitalier Intercommunal de Créteil, Créteil, F-94000, France.

André Coste (A)

Institut Mondor de Recherche Biomédicale, Université Paris-Est Créteil, Inserm U955, CNRS ERL7240, Hôpital Henri-Mondor, Créteil, F-94010, France.
Service d'ORL et de Chirurgie Cervico-Faciale, AP-HP, Hôpital Henri-Mondor, Centre Hospitalier Intercommunal de Créteil, Créteil, F-94000, France.

Estelle Escudier (E)

Childhood Genetic Diseases, Sorbonne Université, Inserm, Hôpital Armand-Trousseau, Paris, F-75012, France.
Génétique moléculaire, AP-HP, Hôpital Armand-Trousseau, Paris, F-75012, Paris.

Guillaume Thouvenin (G)

Département de Pneumologie Pédiatrique, Centre National de Référence des Maladies Respiratoires Rares RespiRare, AP-HP, Sorbonne Université, Hôpital Armand-Trousseau Hospital, Paris, F-75012, France.

Serge Amselem (S)

Childhood Genetic Diseases, Sorbonne Université, Inserm, Hôpital Armand-Trousseau, Paris, F-75012, France.
Génétique moléculaire, AP-HP, Hôpital Armand-Trousseau, Paris, F-75012, Paris.

Marie Legendre (M)

Childhood Genetic Diseases, Sorbonne Université, Inserm, Hôpital Armand-Trousseau, Paris, F-75012, France marie.legendre@aphp.fr.
Génétique moléculaire, AP-HP, Hôpital Armand-Trousseau, Paris, F-75012, Paris.

Classifications MeSH