Abstract
Non-viral gene delivery vectors are emerging as a safer alternative to viral vectors. Among natural polymers, chitosan (Ch) is the most studied one, and low molecular weight Ch, specifically, presents a wide range of advantages for non-viral pDNA delivery. It is crucial to determine the best process for the formation of Low Molecular Weight Chitosan (LMWC)-pDNA complexes and to characterize their physicochemical properties to better understand their behavior once the polyplexes are administered. The transfection efficiency of Ch based polyplexes is relatively low. Therefore, it is essential to understand all the transfection process, including the cellular uptake, endosomal escape and nuclear import, together with the parameters involved in the process to improve the design and development of the non-viral vectors. The aim of this review is to describe the formation and characterization of LMWC based polyplexes, the in vitro transfection process and finally, the in vivo applications of LMWC based polyplexes for gene therapy purposes.
Highlights
The success of gene therapy relies on ensuring that the therapeutic gene enters into the target cell and transcribes without being biodegraded
This parameter determines the colloidal stability of the polyplexes aquous suspension and it plays a crucial role in the viability, cellular uptake and transfection efficiency of Low Molecular Weight Chitosan (LMWC)-pDNA polyplexes, as it is widely reported in the literature [30,41]
Our research group has established a molecular weight (Mw) dependent buffering capacity, being significantly higher for oligochitosans compared to high molecular weight chitosans (HMWC), which could explain in part the flattering properties of LMWC
Summary
The success of gene therapy relies on ensuring that the therapeutic gene enters into the target cell and transcribes without being biodegraded. Gene therapy is still dominated by viral vectors, which present higher transfection efficiencies compared to non-viral gene delivery systems Safety issues such as immunogenicity and mutagenicity hinder the clinical applications of the viral gene delivery systems [1,2]. Numerous factors affect the stability and transfection efficiency of Ch based vectors, being the most relevant ones the molecular weight (Mw) and the degree of deacetylation (DDA) of the polymer [17]. Polyplexes based on low molecular weight chitosans (LMWC) (
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