Abstract
Approximately 4000 diseases are associated with malfunctioning genes in a particular cell type. Gene-based therapy provides a platform to modify the disease-causing genes expression at the cellular level to treat pathological conditions. However, gene delivery is challenging as these therapeutic genes need to overcome several physiological and intracellular barriers in order, to reach the target cells. Over the years, efforts have been dedicated to develop efficient gene delivery vectors to overcome these systemic barriers. Chitosan, a versatile polysaccharide, is an attractive non-viral vector material for gene delivery mainly due to its cationic nature, biodegradability and biocompatibility. The present review discusses the design factors that are critical for efficient gene delivery/transfection and highlights the recent progress of gene therapy using chitosan-based carriers.
Highlights
Gene therapy has grown very rapidly because of its tremendous therapeutic potential to cure numerous genetic diseases by the insertion of new genes (DNA and RNA) into target cells with expression of the transgene
We summarise current progress of chitosan-based formulations for gene delivery, discuss advantages and disadvantages of latest chitosan nanoparticle formulations and introduce the emergence of a new generation of gene delivery carriers
After cellular uptake by endocytosis, these vectors need to escape from endosomes and release interact with negatively charged nucleic acids resulting in the spontaneous formation of into the cytoplasm
Summary
Gene therapy has grown very rapidly because of its tremendous therapeutic potential to cure numerous genetic diseases by the insertion of new genes (DNA and RNA) into target cells with expression of the transgene. Non-viral vectors have a superior safety profile with low immune response, the possibility of facile large-scale production, high loading efficiency for bioactives, and unrestricted gene materials size [5]. Their ability to enter cells and gene transfection efficiencies are lower than that of viral vectors. 17.7% of gene therapy clinical trials use naked DNA because it offers a much safer option than viral vectors This approach lacks target specificity, shows low transfection efficiency and fast degradation. Design Criteria and Requirements for Gene Delivery Using Chitosan-Based Carriers
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