Endosomal disruptors in non-viral gene delivery

Minchin, Rodney F. and Yang, Shu (2010) Endosomal disruptors in non-viral gene delivery. Expert Opinion On Drug Delivery, 7 3: 331-339. doi:10.1517/17425240903512931

Author Minchin, Rodney F.
Yang, Shu
Title Endosomal disruptors in non-viral gene delivery
Journal name Expert Opinion On Drug Delivery   Check publisher's open access policy
ISSN 1742-5247
Publication date 2010-03-01
Sub-type Critical review of research, literature review, critical commentary
DOI 10.1517/17425240903512931
Open Access Status Not yet assessed
Volume 7
Issue 3
Start page 331
End page 339
Total pages 9
Place of publication London, U.K.
Publisher Informa Healthcare
Language eng
Subject C1
920110 Inherited Diseases (incl. Gene Therapy)
0604 Genetics
Formatted abstract
Importance of the field: Non-viral gene delivery for the treatment of genetic and non-genetic diseases has been under investigation for several decades, but there has been very little application in patients because of poor gene expression and toxicity.
Areas covered in this review: As gene delivery almost invariably involves endocytosis, many of its limitations are related to compartmentalisation of the transgene within the endosomes. Gene expression enhancers have become an essential part of manipulating endosomal release, as well as protecting transgene from intracellular degradation. However, disruption of the endosomes can also release proteases that have been shown to activate apoptotic pathways.
What the reader will gain: An understanding of the role that endosomal release plays in the toxicity of gene delivery vehicles will help identify new approaches to minimise adverse effects while enhancing non-viral gene expression.
Take home message: The future of non-viral gene therapy needs to identify new approaches that limit endosome-induced toxicity while enhancing expression so that a pharmacological response can be reliably observed in vivo.
© 2010 Informa UK Ltd
Keyword Gene delivery
Expression enhancers
Q-Index Code C1
Q-Index Status Confirmed Code
Institutional Status UQ

Document type: Journal Article
Sub-type: Critical review of research, literature review, critical commentary
Collections: Official 2011 Collection
School of Biomedical Sciences Publications
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Citation counts: TR Web of Science Citation Count  Cited 19 times in Thomson Reuters Web of Science Article | Citations
Scopus Citation Count Cited 18 times in Scopus Article | Citations
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Created: Sun, 11 Apr 2010, 10:06:12 EST