Gene therapy and protein therapy of ectodermal dysplasias: a perspective.
Détails
ID Serval
serval:BIB_1C3C9EE587E2
Type
Article: article d'un périodique ou d'un magazine.
Sous-type
Synthèse (review): revue aussi complète que possible des connaissances sur un sujet, rédigée à partir de l'analyse exhaustive des travaux publiés.
Collection
Publications
Institution
Titre
Gene therapy and protein therapy of ectodermal dysplasias: a perspective.
Périodique
American journal of medical genetics. Part A
ISSN
1552-4833 (Electronic)
ISSN-L
1552-4825
Statut éditorial
Publié
Date de publication
09/2009
Peer-reviewed
Oui
Volume
149A
Numéro
9
Pages
2042-2044
Langue
anglais
Notes
Publication types: Journal Article
Publication Status: ppublish
Publication Status: ppublish
Résumé
Ectodermal dysplasias (EDs) form a complex and heterogeneous group of diseases currently defined and classified according to their clinical symptoms. The characterization, for several EDs, of the molecular events underlying their development, not only challenges this classification but also opens the door to new therapeutic options such as gene or protein therapy. This article provides a concise overview of the most recent successes and failures of this new type of treatment and sets in perspective how the specificities of given EDs will influence their feasibility in the near future. It makes the case for the need of new classification of EDs that is based on our most recent knowledge of the molecular basis of these diseases.
Mots-clé
Ectodermal Dysplasia/diagnosis, Ectodermal Dysplasia/genetics, Ectodermal Dysplasia/therapy, Ectodysplasins/genetics, Ectodysplasins/therapeutic use, Genetic Therapy, Humans, Proteins/genetics, Proteins/therapeutic use
Pubmed
Open Access
Oui
Création de la notice
02/11/2017 11:04
Dernière modification de la notice
20/08/2019 12:52