Non-viral nanovectors for gene delivery: factors that govern successful therapeutics.
BACKGROUND
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.


METHODS
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.


RESULTS
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.


CONCLUSIONS
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.
Viola
Joana R
JR
Karolinska University Hospital Huddinge, Karolinska Institute, Clinical Research Center, Department of Laboratory Medicine, Novum, Hälsovägen 7, 141 57 Huddinge, Sweden. Joana.Viola@ki.se
El-Andaloussi
Samir
S
Oprea
Iulian I
II
Smith
C I Edvard
CI
D018014:Gene Transfer Techniques; D005822:Genetic Vectors; D036103:Nanotechnology