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Inhaled gene therapy of preclinical muco-obstructive lung diseases by nanoparticles capable of breaching the airway mucus barrier.

Namho KimGijung KwakJason RodriguezAlessandra Livraghi-ButricoXinyuan ZuoValentina SimonEric HanSiddharth Kaup ShenoyNikhil PandeyMarina MazurSusan E BirketAnthony KimSteven M RoweRichard BoucherJustin HanesJung Soo Suk
Published in: Thorax (2021)
DNA-MPP constitutes one of the rare gene delivery systems providing therapeutically meaningful gene transfer efficacy in highly relevant in vivo and in vitro models of muco-obstructive lung diseases due to its unique ability to efficiently penetrate airway mucus.
Keyphrases
  • gene therapy
  • genome wide
  • copy number
  • genome wide identification
  • cystic fibrosis
  • cell free
  • single molecule
  • stem cells
  • bone marrow
  • mesenchymal stem cells
  • nucleic acid
  • genome wide analysis