Aerosolized Gene Therapy for Cystic Fibrosis

Plus: Natural Killer Cells Target Lung Cancer

Hi friends 👋🏼,

Is it just me, or is science fiction rapidly becoming science fact? Here’s what we’ve got prepped for you this week:

  • Aerosolized Gene Therapy for Cystic Fibrosis READER REQUEST

  • Natural Killer Cells Target Lung Cancer READER REQUEST

  • Fighting NASH with RNAi Therapy READER REQUEST

  • Dermatomyositis: A New Intervention READER REQUEST

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🌬️ Aerosolized Gene Therapy for Cystic Fibrosis

Cystic fibrosis (CF) is a disease characterized by thick, sticky mucus buildup that progressively damages the respiratory, digestive, and sometimes reproductive systems.

1/ What causes CF? Mutations in the CFTR gene cause CF. The CFTR gene provides instructions for making the CFTR protein, which functions as a gate to move chloride ions into and out of cells. Efficient movement of chloride ions is necessary to produce thin, freely flowing mucus. When mutations occur in CFTR, the chloride ions are not transported effectively, leading to thick, sticky mucus.

2/ CF is currently treated with CFTR modulator therapies. There are 4 FDA-approved CFTR modulator therapies, 3 of which (Trikafta, Symdeko, Orkambi) specifically work in patients with the F508del mutation, the most common mutation causing CF. These modulator therapies hold the CFTR gate open longer so chloride ions can freely move through. However, modulator therapies do not target the underlying genetic cause of CF.

Can we use gene therapy to develop a “one-shot” cure for CF? That’s exactly what scientists are testing! 👇🏼

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