[Perspective of use of rhDNase and new therapeutics for cystic fibrosis].
The prognosis of cystic fibrosis has dramatically improved during the last three decades. This improvement was due to optimization of symptomatic treatment: antibiotics to combat infection, nutritional support, emphasis on bronchial drainage by physiotherapy and aerosols. The discovery of the gene responsible for the disease and the encoded protein opens the way to a specific and curative approach based upon the knowledge of the intrinsic properties of the protein, and its role in ionic flux. Recently, owing to the development of bioengineering, a recombinant human DNase has become available for clinical use. When aerosolized in the patients, it leads to deep changes in rheological parameters of the secretions that facilitate bronchial drainage and clearing of the airways. Gene therapy, the ultimate expression of these advances, will soon enter the era of clinical applications. Unsolved methodological and ethical issues still preclude immediate use in the patients. The results of transplantation surgery have also dramatically improved since the beginning. Indications are more precisely defined and restricted to the more advanced forms of the disease, according to severe criteria of inclusion.
Navarro
J
J
Service de gastro-entérologie, hôpital Robert-Debré, Paris, France.
Munck
A
A
D000280:Administration, Inhalation; D003550:Cystic Fibrosis / Q000188:drug therapy* / Q000628:therapy; D003850:Deoxyribonuclease I / Q000008:administration & dosage / Q000627:therapeutic use*; D005100:Expectorants / Q000008:administration & dosage / Q000627:therapeutic use*; D006801:Humans; D011994:Recombinant Proteins / Q000008:administration & dosage / Q000627:therapeutic use
D005100:Expectorants; D011994:Recombinant Proteins; C085693:DNASE1 protein, human; D003850:Deoxyribonuclease I
Perspectives d'utilisation de la rhDNase et thérapeutiques nouvelles de la mucoviscidose.