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- W1988984419 abstract "A decade ago it was widely anticipated that cystic fibrosis would be one of the first diseases to be treated by gene therapy. The difficult hurdle of cloning the responsible gene had been accomplished, its function was established and the lung appeared readily accessible for gene replacement. Since the first clinical trials for cystic fibrosis lung disease in the early 1990s it has become increasingly apparent that successful lung-directed gene therapy is significantly more complex than was first envisioned. Numerous obstacles including vector toxicity, inefficient transgene expression and limited vector production have delayed progress. An increased understanding of vector biology and host interaction has led to the development of novel strategies to enhance the efficiency and selectivity of gene delivery to the lung. Although significant challenges remain, there is now a realistic prospect of a clinically effective treatment in the next 10years." @default.
- W1988984419 created "2016-06-24" @default.
- W1988984419 creator A5039017673 @default.
- W1988984419 creator A5077624043 @default.
- W1988984419 date "2005-03-01" @default.
- W1988984419 modified "2023-10-14" @default.
- W1988984419 title "Progress towards gene therapy for cystic fibrosis" @default.
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- W1988984419 doi "https://doi.org/10.1517/17425247.2.2.269" @default.
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