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Cystic fibrosis: current therapeutic targets and future approaches.

Misbahuddin M RafeeqHussam Aly Sayed Murad
Published in: Journal of translational medicine (2017)
The treatment has a long way to go as most of the existing therapeutics is for older children. Other limiting factors include mutation class, genetic profile, drug interactions, adverse effects, and cost. Novel approaches like gene transfer/gene editing, disease modeling and search for alternative targets are warranted.
Keyphrases
  • cystic fibrosis
  • genome wide
  • copy number
  • young adults
  • physical activity
  • small molecule
  • dna methylation
  • combination therapy
  • replacement therapy