Translation of Angiotensin-Converting Enzyme 2 upon Liver- and Lung-Targeted Delivery of Optimized Chemically Modified mRNA

نویسندگان

  • Eva Schrom
  • Maja Huber
  • Manish Aneja
  • Christian Dohmen
  • Daniela Emrich
  • Johannes Geiger
  • Günther Hasenpusch
  • Annika Herrmann-Janson
  • Verena Kretzschmann
  • Olga Mykhailyk
  • Tamara Pasewald
  • Prajakta Oak
  • Anne Hilgendorff
  • Dirk Wohlleber
  • Heinz-Gerd Hoymann
  • Dirk Schaudien
  • Christian Plank
  • Carsten Rudolph
  • Rebekka Kubisch-Dohmen
چکیده

Changes in lifestyle and environmental conditions give rise to an increasing prevalence of liver and lung fibrosis, and both have a poor prognosis. Promising results have been reported for recombinant angiotensin-converting enzyme 2 (ACE2) protein administration in experimental liver and lung fibrosis. However, the full potential of ACE2 may be achieved by localized translation of a membrane-anchored form. For this purpose, we advanced the latest RNA technology for liver- and lung-targeted ACE2 translation. We demonstrated in vitro that transfection with ACE2 chemically modified messenger RNA (cmRNA) leads to robust translation of fully matured, membrane-anchored ACE2 protein. In a second step, we designed eight modified ACE2 cmRNA sequences and identified a lead sequence for in vivo application. Finally, formulation of this ACE2 cmRNA in tailor-made lipidoid nanoparticles and in lipid nanoparticles led to liver- and lung-targeted translation of significant amounts of ACE2 protein, respectively. In summary, we provide evidence that RNA transcript therapy (RTT) is a promising approach for ACE2-based treatment of liver and lung fibrosis to be tested in fibrotic disease models.

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عنوان ژورنال:

دوره 7  شماره 

صفحات  -

تاریخ انتشار 2017