In vivo genome editing using nuclease-encoding mRNA corrects SP-B deficiency

Nat Biotechnol. 2015 Jun;33(6):584-6. doi: 10.1038/nbt.3241. Epub 2015 May 18.
No abstract available

Publication types

  • Letter
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Asthma / genetics
  • Asthma / therapy*
  • Chitosan / therapeutic use
  • Disease Models, Animal
  • Humans
  • Mice
  • Mice, Transgenic
  • Nanoparticles / therapeutic use
  • Pulmonary Alveolar Proteinosis / congenital*
  • Pulmonary Alveolar Proteinosis / genetics
  • Pulmonary Alveolar Proteinosis / therapy
  • Pulmonary Surfactant-Associated Protein B / deficiency*
  • Pulmonary Surfactant-Associated Protein B / genetics
  • RNA Editing / genetics*
  • RNA, Messenger / genetics
  • RNA, Messenger / therapeutic use*

Substances

  • Pulmonary Surfactant-Associated Protein B
  • RNA, Messenger
  • Chitosan

Supplementary concepts

  • Congenital Deficiency of Pulmonary Surfactant Protein B