Authors: Viola, Joana R; El-Andaloussi, Samir; Oprea, Iulian I; Smith, CI Edvard

Source: Expert Opinion on Drug Delivery, Volume 7, Number 6, June 2010 , pp. 721-735(15)

Publisher: Informa Healthcare

Abstract:

Importance of the field: Gene therapy is regarded as one of the most promising therapeutic approaches, as it has the potential to treat disorders by correcting malformations at the nucleic acids.

Areas covered in this review: Some of the most recent developments in the process of plasmid DNA vector design and formulation are reviewed with a special focus on: different formulations of nanovectors and a summary of successful cases reported; requirements for systemic administration; and functionalization of the nanocarriers by use of targeting entities.

What the reader will gain: An understanding of the different physiological barriers and a comprehensive review of the recent strategies used to overcome these obstacles. Particular attention is given to formulations for intravenous administration, colloidal stability properties and different targeting entities used.

Take home message: Overall, vector formulation must take into account the administration route and inherent physiological barriers. Critical parameters for the success of pDNA nanovectors are: particles size, colloidal stability of the formulation and interaction between the carrier and plasmid DNA. Highly relevant is the fact that this interaction should be balanced to offer protection to degradation as well as allow dissociation of the therapeutic nucleic acid for obtaining maximal activity.

Keywords: in vivo; intravenous administration; nanoparticles; receptor-mediated uptake

Document Type: Research article

DOI: http://dx.doi.org/10.1517/17425241003716810

Affiliations: 1: Karolinska University Hospital Huddinge, Karolinska Institute, Clinical Research Center, Department of Laboratory Medicine, Novum, Hälsovägen 7, 141 57 Huddinge, Sweden +46 8 585 836 53; +46 8 585 836 50;, Email: Joana.Viola@ki.se

Publication date: 2010-06-01

More about this publication?

• Editorial Board
• Information for Authors
• Subscribe to this Title
• Terms & Conditions
• ingentaconnect is not responsible for the content or availability of external websites

Related content

• In this: publication
• By this: publisher
• In this Subject: Pharmacology
• By this author: Viola, Joana R ;  El-Andaloussi, Samir ;  Oprea, Iulian I ;  Smith, CI Edvard

Website © Publishing Technology. Article copyright remains with the publisher, society or author(s) as specified within the article.

• Terms and conditions
• Privacy policy
• Information for advertisers