AAV gene therapy for cystic fibrosis: current barriers and recent developments

摘要

IntroductionSince the CF gene was discovered in 1989, researchers have worked to develop a gene therapy. One of the most promising and enduring vectors is AAV, which has been shown to be safe. In particular, several clinical trials have been conducted with AAV serotype 2. All of them detected viral genomes, but identification of mRNA transduction was not consistent; clinical outcomes in Phase II studies were also inconsistent. The lack of a positive outcome has been attributed to a less-than-efficient viral infection by AAV2, a weak transgene promoter and the host immune response to the vector.