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In vivo genome editing by non-viral gene delivery

SIP005
  • Project Leaders: Shyh-Dar Li, Colin Ross
  • Institutions: University of British Columbia (UBC)
  • Budget: $249361
  • Program/Competition: Sector Innovation Program
  • Genome Centre(s): Genome British Columbia
  • Fiscal Year: 2017
  • Status: Closed

Genome sequencing has aided our ability to understand and diagnose genetic diseases and cancer. However, of the estimated 6,000 human genetic diseases, less than 5% have approved treatments. Furthermore, these treatments are often of limited therapeutic benefit to patients, and can sometimes cause adverse drug reactions, highlighting the urgent need for improvement of therapeutic efficacy, specificity, and safety.  This research team at UBCaims to develop an optimized genome editing therapy that will be delivered to target cells using customized lipid nanoparticle (LNP) formulations. They will also establish a platform to further optimize novel LNPs for effective and safe delivery of therapeutic genome editing components. The long-term goal of this research is to bring genome-targeted therapeutics into mainstream healthcare, and make precision genome-editing broadly applicable to many genetic diseases, including cancer. Building upon the established expertise in nanomedicines, this new program is poised to establish BC as a world leader in the emerging field of therapeutic genome editing.