[Future prospects in the management of cystic fibrosis].
Basic and clinical research in cystic fibrosis have led to several new hypothesis to improve the management of the disease. The numerous tracks for new therapies may be explained by the lack of firm patho-physiological explanations for the disease and of knowledge of the best targets to get a significant improvement of the patients. After initial great hopes, there has been important limitations and slow down of gene therapy, imposing to go back to research programs on new vectors. New hopes have arisen with protein therapies, including chaperones molecules that can activate mutated CFTR proteins within the cells. New anti-inflammatory therapies are developed, including proteases inhibitors. The prevention of airway colonisation with Pseudomonas aeruginosa is fundamental and could go through the development of specific vaccines, cellular therapies or molecules directly acting on the virulent factors of the bacteria.
Pin
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Département de pédiatrie, CHU de Grenoble, BP 217, 38043 Grenoble, France. IPin@chu-grenoble.fr
Pilenko
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C
Gout
J P
JP
D000893:Anti-Inflammatory Agents / Q000627:therapeutic use*; D001428:Bacterial Vaccines / Q000627:therapeutic use; D003550:Cystic Fibrosis / Q000150:complications / Q000188:drug therapy* / Q000235:genetics; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics* / Q000502:physiology*; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D009154:Mutation; D011379:Prognosis; D011480:Protease Inhibitors / Q000627:therapeutic use; D011552:Pseudomonas Infections / Q000188:drug therapy / Q000209:etiology; D011550:Pseudomonas aeruginosa / Q000472:pathogenicity; D014774:Virulence
D000893:Anti-Inflammatory Agents; D001428:Bacterial Vaccines; C505032:CFTR protein, human; D011480:Protease Inhibitors; D019005:Cystic Fibrosis Transmembrane Conductance Regulator