Abstract            Volume:8  Issue-5  Year-2020         Original Research Articles

Online ISSN : 2347 - 3215 Issues : 12 per year Publisher : Excellent Publishers Email : editorijcret@gmail.com

Gene therapy is a new modality with the potential for treating or preventing a variety of inherited or acquired diseases. For this purpose, there are a wide variety of gene delivery methods including viral and non-viral vectors. Commonly used viral methods included: retrovirus; adenovirus and lentiviral systems are suitable for gene therapeutic approaches which are based on permanent expression of the therapeutic gene. Non-viral methods included physical forces and chemical compounds to transfer DNA into a cell. Non-viral vectors are far less efficient than viral vectors, but they have advantages due to their low immunogenicity and their large capacity for therapeutic DNA. To improve the function of non-viral vectors, the addition of viral functions such as receptor mediated uptake and nuclear translocation of DNA may finally lead to the development of an artificial virus. The current literature review sets out many different models that are currently being investigated to bridge from studies in cell lines through towards clinical reality. Animal models include horse, dog, sheep, and Non-human primate models (macaque, common marmoset, owl monkey) are used for preclinical gene vector safety and efficacy trials to bridge the gap prior to clinical studies for the safety development of clinically effective delivery systems of DNA and RNA technologies. The major challenges have been delivery of DNA to the target cells and duration of expression. Ethical and moral issues implicit in gene therapy have drawn notice from several governmental and religious organizations.

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