Avancées thérapeutiques et évolution vers une médecine personnalisée pour les patients atteints de mucoviscidose

[en] Cystic fibrosis is a genetic disorder responsible for the production of a defective transmembrane protein. In recent years, new protein modulators have been developed. They aim to treat the underlying cause of the disease. The results on the biomarkers of the function of the CFTR protein and on the clinical outcomes are very encouraging. However, there is an individual heterogeneity in the response to modulators within a same genotype. Furthermore, clinical trials focus on the most common mutations in the CFTR gene, in particular DF508. Intestinal organoids, a new model of ex vivo study, could offer a quick approach to increase access to effective treatment for all patients with cystic fibrosis regardless of their CFTR genotype. Organoids could enable personalized treatment of cystic fibrosis.

Disciplines :

Pediatrics

Author, co-author :

BOBOLI, Hedwige ; Centre Hospitalier Universitaire de Liège - CHU > Département de Pédiatrie > Service de pédiatrie

Pirson, Jessica

SEGHAYE, Marie-Christine ; Centre Hospitalier Universitaire de Liège - CHU > Département de Pédiatrie > Service de pédiatrie

KEMPENEERS, Céline ; Centre Hospitalier Universitaire de Liège - CHU > Département de Pédiatrie > Service de pédiatrie

Language :

French

Title :

Avancées thérapeutiques et évolution vers une médecine personnalisée pour les patients atteints de mucoviscidose

Alternative titles :

[en] News drugs and evolution towards personalized treatment for cystic fibrosis

Publication date :

May 2020

Journal title :

Revue Médicale de Liège

ISSN :

0370-629X

eISSN :

2566-1566

Publisher :

Université de Liège. Revue Médicale de Liège, Liège, Belgium

Peer reviewed :

Peer reviewed

Available on ORBi :

since 12 November 2020

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Bibliography

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