WS17.04 Developing a non-viral gene therapy strategy for treating lung cystic fibrosis disease

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...

Status of gene therapy for cystic fibrosis lung disease.

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...

Biopolymer-Based Nanoparticles for Cystic Fibrosis Lung Gene Therapy Studies

Lung gene therapy for cystic fibrosis disease has not been successful due to several challenges such as the absence of an appropriate vector. Therefore, optimal delivery of emerging therapeutics to airway epithelial cells demands suitable non-viral systems. In this work, we describe the formulation and the physicochemical investigation of biocompatible and biodegradable polymeric nanoparticles ...

Targeting the Lung: Challenges in Gene Therapy for Cystic Fibrosis

Cystic Fibrosis (CF) is the most common fatal autosomal recessive genetic disease in the Caucasians with a frequency of approximately 1 in 2500 newborns (Cystic Fibrosis Founda‐ tion, http://www.cff.org/). It affects several organs including the lungs, the liver, the pan‐ creas, the sweat glands and the gastrointestinal and reproductive tracts [1]. The most severe complications that finally lea...