Development of Lipid Nanoparticle Reagents for Functional Genomics in Difficult-to-Transfect Cells In Vitro

The objective of this project is to develop lipid nanoparticle (LNP) reagents for the delivery of nucleic acids to turn off, or turn on target genes in “hard-to-transfect” neurons and stem cells in vitro and in vivo. A recent report (BCC Research, April 2011) observed that “51% of researchers employ cell-based techniques to perform transfection routinely. Although transfection techniques have been available for many years ….this procedure faces challenges such as the efficiency of gene introduction and its toxicity in cells.” With an estimated market of $1.9 billion by 2016 this project will help explore high value market niche that is poised for substantial growth. It will leverage clinical grade, proprietary LNP reagents, a novel proprietary microfluidic-based manufacturing LNP technology, and a unique mechanism of action that maximizes LNP potency by combining broad expertise in technology development and commercialization from the University of British Columbia, and Precision NanoSystems Inc. (PNI).

Faculty Supervisor:

Dr. Marcel Bally

Student:

Gesine Heuck

Partner:

Precision NanoSystems Inc.

Discipline:

Pharmacy / Pharmacology

Sector:

Nanotechnologies

University:

University of British Columbia

Program:

Elevate

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