Gene delivery using cationic liposomes

Sarah E. McNeil, Yvonne Perrie

Research output: Contribution to journalArticle

33 Citations (Scopus)

Abstract

The development of cationic liposomes for gene delivery has been ongoing for almost 20 years; however, despite extensive efforts to develop a successful therapeutic agent, there has been limited progress towards generating an effective pharmaceutical product. Since the introduction of N-(1-[2,3-dioleyloxy]propyl)-N,N,N-trimethylammonium chloride, an immense number of different cationic lipids have been synthesised and used to formulate cationic liposome–DNA complexes. Structural modification of the cationic lipids and the addition of components within the delivery system that can facilitate the fusion, cellular uptake and targeting of liposome–DNA complexes have all been used in a bid to enhance their transfection efficiency. Unfortunately, the overall impact of these improvements is still nominal, with the vast majority of clinical trials (∼ 85%) continuing to rely on more potent viral delivery of DNA despite their associated toxicity issues. Key characteristics of the most effective cationic liposomes for the delivery of plasmid DNA (from a consensus of the literature) is identified here and the problems of converting these attributes into an effective pharmaceutical product are outlined.
LanguageEnglish
Pages1371-1382
Number of pages12
JournalExpert Opinion on Therapeutic Patents
Volume16
Issue number10
DOIs
Publication statusPublished - 1 Oct 2006

Fingerprint

Liposomes
Genes
DNA
Lipids
Viral DNA
Pharmaceutical Preparations
Transfection
Plasmids
Clinical Trials
Therapeutics

Keywords

  • cationic lipids
  • cationic liposomes
  • gene delivery
  • gene therapy
  • non-viral delivery system

Cite this

McNeil, Sarah E. ; Perrie, Yvonne. / Gene delivery using cationic liposomes. In: Expert Opinion on Therapeutic Patents. 2006 ; Vol. 16, No. 10. pp. 1371-1382.
@article{bb540f48f99e49948a8ee1512d8c52ad,
title = "Gene delivery using cationic liposomes",
abstract = "The development of cationic liposomes for gene delivery has been ongoing for almost 20 years; however, despite extensive efforts to develop a successful therapeutic agent, there has been limited progress towards generating an effective pharmaceutical product. Since the introduction of N-(1-[2,3-dioleyloxy]propyl)-N,N,N-trimethylammonium chloride, an immense number of different cationic lipids have been synthesised and used to formulate cationic liposome–DNA complexes. Structural modification of the cationic lipids and the addition of components within the delivery system that can facilitate the fusion, cellular uptake and targeting of liposome–DNA complexes have all been used in a bid to enhance their transfection efficiency. Unfortunately, the overall impact of these improvements is still nominal, with the vast majority of clinical trials (∼ 85{\%}) continuing to rely on more potent viral delivery of DNA despite their associated toxicity issues. Key characteristics of the most effective cationic liposomes for the delivery of plasmid DNA (from a consensus of the literature) is identified here and the problems of converting these attributes into an effective pharmaceutical product are outlined.",
keywords = "cationic lipids, cationic liposomes, gene delivery, gene therapy, non-viral delivery system",
author = "McNeil, {Sarah E.} and Yvonne Perrie",
year = "2006",
month = "10",
day = "1",
doi = "10.1517/13543776.16.10.1371",
language = "English",
volume = "16",
pages = "1371--1382",
journal = "Expert Opinion on Therapeutic Patents",
issn = "1354-3776",
number = "10",

}

Gene delivery using cationic liposomes. / McNeil, Sarah E.; Perrie, Yvonne.

In: Expert Opinion on Therapeutic Patents, Vol. 16, No. 10, 01.10.2006, p. 1371-1382.

Research output: Contribution to journalArticle

TY - JOUR

T1 - Gene delivery using cationic liposomes

AU - McNeil, Sarah E.

AU - Perrie, Yvonne

PY - 2006/10/1

Y1 - 2006/10/1

N2 - The development of cationic liposomes for gene delivery has been ongoing for almost 20 years; however, despite extensive efforts to develop a successful therapeutic agent, there has been limited progress towards generating an effective pharmaceutical product. Since the introduction of N-(1-[2,3-dioleyloxy]propyl)-N,N,N-trimethylammonium chloride, an immense number of different cationic lipids have been synthesised and used to formulate cationic liposome–DNA complexes. Structural modification of the cationic lipids and the addition of components within the delivery system that can facilitate the fusion, cellular uptake and targeting of liposome–DNA complexes have all been used in a bid to enhance their transfection efficiency. Unfortunately, the overall impact of these improvements is still nominal, with the vast majority of clinical trials (∼ 85%) continuing to rely on more potent viral delivery of DNA despite their associated toxicity issues. Key characteristics of the most effective cationic liposomes for the delivery of plasmid DNA (from a consensus of the literature) is identified here and the problems of converting these attributes into an effective pharmaceutical product are outlined.

AB - The development of cationic liposomes for gene delivery has been ongoing for almost 20 years; however, despite extensive efforts to develop a successful therapeutic agent, there has been limited progress towards generating an effective pharmaceutical product. Since the introduction of N-(1-[2,3-dioleyloxy]propyl)-N,N,N-trimethylammonium chloride, an immense number of different cationic lipids have been synthesised and used to formulate cationic liposome–DNA complexes. Structural modification of the cationic lipids and the addition of components within the delivery system that can facilitate the fusion, cellular uptake and targeting of liposome–DNA complexes have all been used in a bid to enhance their transfection efficiency. Unfortunately, the overall impact of these improvements is still nominal, with the vast majority of clinical trials (∼ 85%) continuing to rely on more potent viral delivery of DNA despite their associated toxicity issues. Key characteristics of the most effective cationic liposomes for the delivery of plasmid DNA (from a consensus of the literature) is identified here and the problems of converting these attributes into an effective pharmaceutical product are outlined.

KW - cationic lipids

KW - cationic liposomes

KW - gene delivery

KW - gene therapy

KW - non-viral delivery system

UR - http://www.tandfonline.com/doi/full/10.1517/13543776.16.10.1371

U2 - 10.1517/13543776.16.10.1371

DO - 10.1517/13543776.16.10.1371

M3 - Article

VL - 16

SP - 1371

EP - 1382

JO - Expert Opinion on Therapeutic Patents

T2 - Expert Opinion on Therapeutic Patents

JF - Expert Opinion on Therapeutic Patents

SN - 1354-3776

IS - 10

ER -