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- W2590980968 endingPage "1081" @default.
- W2590980968 startingPage "1071" @default.
- W2590980968 abstract "Cystic fibrosis (CF) is a monogenic autosomal recessive disorder where the defective gene, the cystic fibrosis transmembrane conductance regulator (CFTR), is well identified. Moreover, the respiratory tract can be targeted through noninvasive aerosolized formulations for inhalation. Therefore, gene therapy is considered a plausible strategy to address this disease. Conventional gene therapy strategies rely on the addition of a correct copy of the CFTR gene into affected cells in order to restore the channel activity. In recent years, genome correction strategies have emerged, such as zinc-finger nucleases, transcription activator-like effector nucleases and clustered regularly interspaced short palindromic repeats associated to Cas9 nucleases. These gene editing tools aim to repair the mutated gene at its original genomic locus with high specificity. Besides, the success of gene therapy critically depends on the nucleic acids carriers. To date, several clinical studies have been carried out to add corrected copies of the CFTR gene into target cells using viral and non-viral vectors, some of them with encouraging results. Regarding genome editing systems, preliminary in vitro studies have been performed in order to repair the CFTR gene. In this review, after briefly introducing the basis of CF, we discuss the up-to-date gene therapy strategies to address the disease. The review focuses on the main factors to take into consideration when developing gene delivery strategies, such as the design of vectors and plasmid DNA, in vitro/in vivo tests, translation to human use, administration methods, manufacturing conditions and regulatory issues." @default.
- W2590980968 created "2017-03-03" @default.
- W2590980968 creator A5052386999 @default.
- W2590980968 creator A5052643404 @default.
- W2590980968 creator A5054254948 @default.
- W2590980968 creator A5070577695 @default.
- W2590980968 date "2017-03-07" @default.
- W2590980968 modified "2023-10-13" @default.
- W2590980968 title "Gene delivery to the lungs: pulmonary gene therapy for cystic fibrosis" @default.
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