Essais thérapeutiques dans la dystrophie musculaire de Duchenne: entre espoirs et désespoirs [Therapeutic trials for Duchenne muscular dystrophy: between hopes and disappointments].

Détails

ID Serval
serval:BIB_94BE4DD1A486
Type
Article: article d'un périodique ou d'un magazine.
Collection
Publications
Titre
Essais thérapeutiques dans la dystrophie musculaire de Duchenne: entre espoirs et désespoirs [Therapeutic trials for Duchenne muscular dystrophy: between hopes and disappointments].
Périodique
Revue Médicale Suisse
Auteur(s)
Bloetzer C., Fluss J., Jeannet P.Y.
ISSN
1660-9379 (Print)
ISSN-L
1660-9379
Statut éditorial
Publié
Date de publication
2012
Volume
8
Numéro
329
Pages
409-412
Langue
français
Notes
Publication types: English Abstract ; Journal Article Publication Status: ppublish
Résumé
Duchenne muscular dystrophy is an X-linked progressive muscle disease. Since the discovery of the dystrophin gene responsible for the condition, various therapeutic strategies have been elaborated. In this paper we introduce three of them, which are well into clinical trials. The first is based on the ability to read through premature stop codons, the second is based on the technique of exon skipping. Both strategies are examples of "personalized medicines", tailored for specific mutation types. The third approach is a pharmacological one, potentially useful for all Duchenne patients, regardless of their mutation type. These first clinical trials raise many questions for researchers as well as for patients and their families, some of which are discussed.
Mots-clé
Child, Gene Therapy, Humans, Muscular Dystrophy, Duchenne/drug therapy, Muscular Dystrophy, Duchenne/genetics
Pubmed
Création de la notice
06/06/2012 19:05
Dernière modification de la notice
03/03/2018 19:38
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