Title:Advances in Targeted Gene Delivery
Volume: 16
Issue: 7
Author(s): Anjuman A. Begum, Istvan Toth, Waleed M. Hussein and Peter M. Moyle*
Affiliation:
- School of Pharmacy, The University of Queensland, Woolloongabba, 4102,Australia
Keywords:
Gene therapy, gene delivery, targeted delivery, vectors, cell surface receptors, targeting moieties.
Abstract: Gene therapy has the potential to treat both acquired and inherited genetic diseases. Generally,
two types of gene delivery vectors are used - viral vectors and non-viral vectors. Non-viral gene delivery
systems have attracted significant interest (e.g. 115 gene therapies approved for clinical trials in 2018;
clinicaltrials.gov) due to their lower toxicity, lack of immunogenicity and ease of production compared
to viral vectors. To achieve the goal of maximal therapeutic efficacy with minimal adverse effects, the
cell-specific targeting of non-viral gene delivery systems has attracted research interest. Targeting
through cell surface receptors; the enhanced permeability and retention effect, or pH differences are
potential means to target genes to specific organs, tissues, or cells. As for targeting moieties, receptorspecific
ligand peptides, antibodies, aptamers and affibodies have been incorporated into synthetic nonviral
gene delivery vectors to fulfill the requirement of active targeting. This review provides an overview
of different potential targets and targeting moieties to target specific gene delivery systems.
