Abstract
Gene therapy is a promising therapeutic modality for the treatment of genetic disorders. Gene therapy has been able to correct many of the genetic diseases at the root of their cause by systematizing genetic information that encodes for all functions of every cell in our body. Recent studies have identified novel molecular targets for genetic disorders that can be used to deliver gene to the specific site. Gene therapy applications require safe and efficient method for gene transfer. Over the last decade, non-viral and viral gene therapy approaches have been tested in preclinical studies and human clinical trials. Gene delivery via conventional means by using viral vectors has several undesirable side effects such as insertion of mutational viral gene into the host genome and overwhelming immune and inflammatory responses. As compared to viral vectors, non-viral vehicles has received great attention due to their several favorable properties, including low toxicity and immunogenicty, resistance to nuclease, and their high affinity for DNA targets. Here, we describe how non-viral gene-transfer vehicles have been used and can be modified to target specific tissues for gene therapy. This review focuses on existing and emerging patents on non-viral based genetic engineering strategies for the delivery of therapeutic molecules or several approaches for genetic disorder treatment.
Keywords: Gene therapy, non-viral gene delivery, liposomes, polymers, poly-L-lysine, polyethylenimine, gene gun, dendrimer, sonoporosis, electroporation
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