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Intellia Therapeutics’ Preclinical Data Show Continued Progress in In Vivo Gene Editing With Systemic Lipid Nanoparticle Delivery of CRISPR/Cas9 components

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Additional Information

Additional Relevant Details
In several in vitro and in vivo preclinical studies, the data demonstrated:
  • Editing efficiency in mouse liver of up to approximately 60 percent at the transthyretin (TTR) target site after a single intravenous administration, consistently across different lobes. This resulted in an associated decrease in serum TTR protein levels of up to approximately 80 percent;
  • Dose-dependent editing by LNP delivery;
  • Undetectable Cas9 mRNA and guide RNA (gRNA) in the liver at 72 hours post administration;
  • Repair patterns in mouse liver cells in vivo being best predicted by primary mouse liver cells in vitro.
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Catalyst Date
Occurred on:
Aug 18, 2016
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Related Keywords In Vivo Gene Editing, Crispr/cas9, Lipid Nanoparticle Delivery, Rna, Grna, Liver