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Nanotechnology

Breakthrough in Gene Editing: Pfizer's Research Showcases Efficiency of Hyaluronic Acid-Based Nanoparticles

Strategic Insights

— 1 min read

Researchers at Pfizer have made a significant breakthrough in gene editing, using hyaluronic acid-based nanoparticles to target hepatocytes in mice. The study, published in the journal Nanomedicine, demonstrated the efficiency of these nanoparticles in delivering CRISPR/Cas9 gene editing plasmid and eGFP gene payload to the liver. The findings have promising implications for the development of non-viral biodegradable and biocompatible polymeric delivery systems for gene editing.

Key Takeaways:

  • The study aimed to evaluate hepatocyte-specific gene editing using systemic administration of hyaluronic acid (HA)-based nanoparticles in naive CD-1 mice.
  • Financial support for this research came from Pfizer Inc Centers for Therapeutic Innovation.
  • The research showed that HA-PEI-mannose nanoparticles with differential mannose density (1X and 2X) had increased uptake in both the liver and spleen, with 55-65% uptake by hepatocytes.
  • One of two gRNA targets showed 15% genome editing, and similar results were obtained for all three nanoparticle formulations.
  • The results were encouraging as proof of concept for the development of a non-viral biodegradable and biocompatible polymeric delivery system for gene editing specifically targeting hepatocytes upon systemic administration.
  • The research was conducted by Liliana Wroblewska, Christopher Francis, Pamela Pegman, and Mansoor Amiji, with financial support from Pfizer Inc.
  • The study was published in the journal Nanomedicine, volume 40, issue 1, article number 102488.

Statistics:

  • 55-65% uptake by hepatocytes of HA-PEI-mannose nanoparticles
  • 15% genome editing of one of two gRNA targets
  • 16.2% of cells were GFP positive with HA-PEI-mannose-1X nanoparticles
  • 3 different nanoparticle formulations were used in the study

Sources:

  • Systemic biodistribution and hepatocyte-specific gene editing with CRISPR/Cas9 using hyaluronic acid-based nanoparticles. Nanomedicine, 2021;40:102488.
  • Pfizer Inc., Worldwide Research & Development, Medicinal Sciences, Biomedicine Design, Cambridge, MA, United States.

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