New therapeutic developments in cystic fibrosis

被引：0

作者：

Bui, S. ^{[1
]}

Macey, J. ^{[2
]}

Fayon, M. ^{[3
]}

Bihouee, T. ^{[4
]}

Burgel, P. -R. ^{[5
]}

Colomb, V. ^{[6
]}

Corvol, H. ^{[7
]}

Durieu, I. ^{[8
]}

Hubert, D. ^{[5
]}

Marguet, C. ^{[9
]}

Mas, E. ^{[10
]}

Munck, A. ^{[11
]}

Murris-Espin, M. ^{[12
]}

Reix, R. ^{[13
]}

Sermet-Gaudelus, I. ^{[14
]}

机构：

[1] CHU Bordeaux Pellegrin, Ctr Invest Clin CIC 1401, CRCM Pediat, Pl Amelie Raba Leon, F-33076 Bordeaux, France

[2] Hop Haut Eveque, CHU Bordeaux Pellegrin, CRCM Adulte, Ave Magellan, F-33076 Bordeaux, France

[3] Univ Bordeaux, Ctr Rech Cardiothorac Bordeaux, U1045, CIC 1401, Pl Amelie Raba Leon, F-33000 Bordeaux, France

[4] Hop Mere Enfant, CRCM Pediat, 7 Quaff Moncousu, F-44093 Nantes, France

[5] Hop Cochin, CRCM Adultes, 27 Rue Faubourg St Jacques, F-75679 Paris, France

[6] Assoc Vaincre Mucoviscidose, 181 Rue Tolbiac, F-75013 Paris, France

[7] Hop Armand Trousseau, CRCM Pediat, 26 Av Dr Arnold Netter, F-75571 Paris, France

[8] Ctr Hosp Lyon Sud, CRCM Adultes, 165 Chemin Grand Revoyet, F-69495 Pierre Benite, France

[9] Hop Charles Nicolle, CRCM Pediat, 1 Rue Germont, F-76031 Rouen, France

[10] Hop Enfants, CRCM Pediat, 330 Av Grande Bretagne, F-31059 Toulouse, France

[11] Hop Robert Debre, CRCM Pediat, 48 Blvd Serurier, F-75019 Paris, France

[12] Hop Larrey, CRCM Adulte, 24 Chemin Pourvouville, F-31059 Toulouse, France

[13] Hop Debrousse, CRCM Pediat, 29 rue Sceurs Bouvier, F-69322 Lyon, France

[14] Hop Necker Enfants Malad, CRCM Pediat, 149 Rue Sevres, F-75743 Paris, France

来源：

ARCHIVES DE PEDIATRIE | 2016年 / 23卷

关键词：

G551D MUTATION; PHE508DEL CFTR; DOUBLE-BLIND; LUMACAFTOR-IVACAFTOR; CONTROLLED-TRIAL; EFFICACY; SAFETY; POTENTIATOR; ATALUREN; CHLORIDE;

D O I：

暂无

中图分类号：

R72 [儿科学];

学科分类号：

100202 ;

摘要：

Since the discovery of chloride secretion by the Cystic Fibrosis Transport regulator CFTR in 1983, and CFTR gene in 1989, knowledge about CFTR synthesis, maturation, intracellular transfer and function has dramatically expanded. These discoveries have led to the distribution of CF mutations into 6 classes with different pathophysiological mechanisms. In this article we will explore the state of art on CFTR synthesis and its chloride secretion function. We will then explore the consequences of the 6 classes of mutations on CFTR protein function and we will describe the new therapeutic developments aiming at correcting these defects.

引用

页码：S47 / S53

页数：7

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