Titre

Neonatal treatment with recombinant ectodysplasin prevents respiratory disease in dogs with X-linked ectodermal dysplasia.

Type
article
Institution
UNIL/CHUV/Unisanté + institutions partenaires
Périodique
Auteur(s)
Mauldin, E.A.
Auteure/Auteur
Gaide, O.
Auteure/Auteur
Schneider, P.
Auteure/Auteur
Casal, M.L.
Auteure/Auteur
Liens vers les personnes
Liens vers les unités
ISSN
1552-4833[electronic]
Statut éditorial
Publié
Date de publication
2009
Volume
149A
Numéro
9
Première page
2045
Dernière page/numéro d’article
2049
Peer-reviewed
Oui
Langue
anglais
Résumé
Patients with defective ectodysplasin A (EDA) have X-linked hypohidrotic ectodermal dysplasia (XLHED; OMIM#305100), a condition comprising hypotrichosis, inability to sweat, abnormal teeth, and frequent pulmonary infections. The XLHED dogs show the same clinical signs as humans with the disorder, including frequent respiratory infections that can be fatal. The respiratory disease in humans and dogs is thought to be due to the absence of tracheal and bronchial glands which are a vital part of the mucociliary clearance mechanism. In our XLHED model, the genetically missing EDA was replaced by postnatal intravenous administration of recombinant EDA resulting in long-term, durable corrective effect on adult, permanent dentition. After treatment with EDA, significant correction of the missing tracheal and bronchial glands was achieved in those dogs that received higher doses of EDA. Moreover, successful treatment resulted in the presence of esophageal glands, improved mucociliary clearance, and the absence of respiratory infection. These results demonstrate that a short-term treatment at a neonatal age with a recombinant protein can reverse a developmental disease and result in vastly improved quality of life.
PID Serval
serval:BIB_578EA09B6AF8
DOI
PMID
WOS
Permalien
Date de création
2009-10-07T14:33:59.560Z
Date de création dans IRIS
2025-05-20T14:41:46Z
Fichier(s)
En cours de chargement...
Vignette d'image
Nom

BIB_578EA09B6AF8.P001.pdf

Version du manuscrit

preprint

Taille

786.36 KB

Format

Adobe PDF

PID Serval

serval:BIB_578EA09B6AF8.P001

URN

urn:nbn:ch:serval-BIB_578EA09B6AF80

Somme de contrôle

(MD5):cf6c4b46b8045004a824cb5377756b68