Advances in Targeted Gene Delivery.

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.

journal_name

Curr Drug Deliv

journal_title

Current drug delivery

authors

Begum AA,Toth I,Hussein WM,Moyle PM

doi

10.2174/1567201816666190529072914

subject

Has Abstract

pub_date

2019-01-01 00:00:00

pages

588-608

issue

7

eissn

1567-2018

issn

1875-5704

pii

CDD-EPUB-98645

journal_volume

16

pub_type

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