A Combination Therapy for Cystic Fibrosis

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.

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...

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...

13 Channel Replacement Therapy for Cystic Fibrosis

Another Beginning for Cystic Fibrosis Therapy.

n engl j med 373;3 nejm.org July 16, 2015 274 whether most or all of these cancers are dependent on CDK4 and CDK6, including those that are not cyclin D1–amplified or have no p16INK4a loss. A molecular analysis of the cyclin D–p16INK4a–RB pathway in breast cancer from the Cancer Genome Atlas supports this hypothesis,6 suggesting that most estrogen-receptor–positive breast cancers have intact RB...