Genetic medicines for CF: Hype versus reality.

@article{Alton2016GeneticMF,
  title={Genetic medicines for CF: Hype versus reality.},
  author={Eric Alton and A. C. Boyd and Jane C Davies and Deborah Rebecca Gill and Uta Griesenbach and Patrick T Harrison and Noreen Henig and Tracy E. Higgins and Stephen C. Hyde and J. Alastair Innes and Michael S D Korman},
  journal={Pediatric pulmonology},
  year={2016},
  volume={51 S44},
  pages={S5-S17}
}
Since identification of the CFTR gene over 25 years ago, gene therapy for cystic fibrosis (CF) has been actively developed. More recently gene therapy has been joined by other forms of "genetic medicines" including mRNA delivery, as well as genome editing and mRNA repair-based strategies. Proof-of-concept that gene therapy can stabilize the progression of CF lung disease has recently been established in a Phase IIb trial. An early phase study to assess the safety and explore efficacy of CFTR… CONTINUE READING
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