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[Respiratory disease in cystic fibrosis: from physiopathology to therapy. Kinesitherapy and pulmonary transplantation excluded].
Rev Mal Respir 1999; 16(4):495-509RM

Abstract

Respiratory disease is the major cause of morbidity and mortality in cystic fibrosis. Cystic fibrosis was long treated in the pediatric setting, but improved survival has led to the implication of adult pneumology in therapeutic management. Since the gene causing cystic fibrosis has been clone, our knowledge of the pathophysiology of the disease has literally exploded, particularly concerning the deleterious consequences of defective expression and distribution of CFTR (cystic fibrosis transmembrane conductance regulator) protein in chronic lung inflammation and infection. This knowledge has led to an optimization of existing therapeutic strategies and to the formulation of hypotheses for the development of new pharmaceutical reagents, allowing an assessment of disease outcome not only in terms of survival but also in terms of quality of life. Early in vivo clinical trials have been encouraging although the efficacy of gene transfer and expression remain modest and the optimal vector remains to be determined. Different potential pharmacological approaches are being studied in order to correct for defective CFTR function at different levels of gene mutation, and to modulate the disorder in transepithelial ionic transfer. One could expect in the near future to see combinations of complementary genotype-specific drugs used for the treatment of cystic fibrosis after patient genotyping to categorize the type of mutation.

Authors+Show Affiliations

Service de Chirurgie Thoracique, CHU Sud Sainte-Marguerite, Marseille.

Pub Type(s)

English Abstract
Journal Article
Review

Language

fre

PubMed ID

10549060

Citation

Reynaud-Gaubert, M. "[Respiratory Disease in Cystic Fibrosis: From Physiopathology to Therapy. Kinesitherapy and Pulmonary Transplantation Excluded]." Revue Des Maladies Respiratoires, vol. 16, no. 4, 1999, pp. 495-509.
Reynaud-Gaubert M. [Respiratory disease in cystic fibrosis: from physiopathology to therapy. Kinesitherapy and pulmonary transplantation excluded]. Rev Mal Respir. 1999;16(4):495-509.
Reynaud-Gaubert, M. (1999). [Respiratory disease in cystic fibrosis: from physiopathology to therapy. Kinesitherapy and pulmonary transplantation excluded]. Revue Des Maladies Respiratoires, 16(4), pp. 495-509.
Reynaud-Gaubert M. [Respiratory Disease in Cystic Fibrosis: From Physiopathology to Therapy. Kinesitherapy and Pulmonary Transplantation Excluded]. Rev Mal Respir. 1999;16(4):495-509. PubMed PMID: 10549060.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - [Respiratory disease in cystic fibrosis: from physiopathology to therapy. Kinesitherapy and pulmonary transplantation excluded]. A1 - Reynaud-Gaubert,M, PY - 1999/11/5/pubmed PY - 1999/11/5/medline PY - 1999/11/5/entrez SP - 495 EP - 509 JF - Revue des maladies respiratoires JO - Rev Mal Respir VL - 16 IS - 4 N2 - Respiratory disease is the major cause of morbidity and mortality in cystic fibrosis. Cystic fibrosis was long treated in the pediatric setting, but improved survival has led to the implication of adult pneumology in therapeutic management. Since the gene causing cystic fibrosis has been clone, our knowledge of the pathophysiology of the disease has literally exploded, particularly concerning the deleterious consequences of defective expression and distribution of CFTR (cystic fibrosis transmembrane conductance regulator) protein in chronic lung inflammation and infection. This knowledge has led to an optimization of existing therapeutic strategies and to the formulation of hypotheses for the development of new pharmaceutical reagents, allowing an assessment of disease outcome not only in terms of survival but also in terms of quality of life. Early in vivo clinical trials have been encouraging although the efficacy of gene transfer and expression remain modest and the optimal vector remains to be determined. Different potential pharmacological approaches are being studied in order to correct for defective CFTR function at different levels of gene mutation, and to modulate the disorder in transepithelial ionic transfer. One could expect in the near future to see combinations of complementary genotype-specific drugs used for the treatment of cystic fibrosis after patient genotyping to categorize the type of mutation. SN - 0761-8425 UR - https://www.unboundmedicine.com/medline/citation/10549060/[Respiratory_disease_in_cystic_fibrosis:_from_physiopathology_to_therapy__Kinesitherapy_and_pulmonary_transplantation_excluded]_ L2 - http://www.em-consulte.com/retrieve/pii/MDOI-RMR-08-1999-16-4-0761-8425-101019-ART64 DB - PRIME DP - Unbound Medicine ER -