Mucolytic Therapy in Cystic Fibrosis

Highly effective mucolytic therapy in cystic fibrosis and bronchiolitis

Gene therapy in cystic fibrosis.

Theoretically, cystic fibrosis transmembrane conductance regulator (CFTR) gene replacement during the neonatal period can decrease morbidity and mortality from cystic fibrosis (CF). In vivo gene transfers have been accomplished in CF patients. Choice of vector, mode of delivery to airways, translocation of genetic information, and sufficient expression level of the normalized CFTR gene are issu...

Aerosol therapy in cystic fibrosis.

An aerosol is defined as a suspension of liquid or solid particles in air or oxygen, and the process of forming an aerosol is called nebulization. Therapeutically an aerosol may be inhaled and may, but does not necessarily, contain mucolytic, therapeutic, or antibiotic agents. Common usage of aerosol in medicine usually means intermittent inhalation by face mask or oral tube. Mist tents are aer...

In vitro assessment of combined antibiotic and mucolytic treatment for Pseudomonas aeruginosa infection in cystic fibrosis.

The minimal inhibitory concentration of azlocillin for Pseudomonas aeruginosa is appreciably reduced when combined with the mucolytic agent mesna (Mistabron) because of an independent bacteriostatic effect of mesna. Bactericidal activity of azlocillin is unaltered by mesna. Mesna inhalations alone or combined with azlocillin may benefit cystic fibrosis patients with pseudomonas lung infections.

Barriers and recent advances in non-viral vectors targeting the lungs for cystic fibrosis gene therapy

Cystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in CFTR genes that affect chloride ion channel. The CF is a good nominee for gene therapy as the asymptomatic carriers are phenotypically normal, and the desired cells are accessible for vector delivery. Gene therapy shows promising effects involving the correction of gene or replacement of the mutant gene with the func...