Oxygen therapy for cystic fibrosis

Gene therapy for cystic fibrosis.

Following the cloning of the cystic fibrosis (CF) gene, in vitro studies rapidly established the feasibility of gene therapy for this disease. Unlike ex vivo approaches that have been utilized for other genetic diseases such as adenosine deaminase deficiency, gene therapy for CF will likely require direct in vivo delivery of gene transfer vectors to the airways of patients with CF. Hence, major...

Oxygen saturation in cystic fibrosis.

The availability of non-invasive oxygen saturation (SaO2) measurement could prove to be a useful tool for following up the progress of patients with cystic fibrosis. The present study was undertaken to compare its use with other routine measurements in the clinic. A total of 100 patients with cystic fibrosis were compared with 50 patients with stable asthma. The children were aged between 5 and...

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...

A Combination Therapy for Cystic Fibrosis

The most prevalent form of cystic fibrosis arises from an amino acid deletion in the cystic fibrosis transmembrane conductance regulator, CFTR. A recently approved treatment for individuals homozygous for this mutation combines a chemical corrector, which helps CFTR fold, and a potentiator that increases CFTR channel activity.

13 Channel Replacement Therapy for Cystic Fibrosis