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Current Gene Therapy

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ISSN (Print): 1566-5232
ISSN (Online): 1875-5631

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Review Article

Current Landscape and Emerging Opportunities of Gene Therapy with Non-viral Episomal Vectors

Author(s): Safir Ullah Khan*, Munir Ullah Khan, Muhammad Imran Khan, Fadia Kalsoom and Aqeela Zahra

Volume 23, Issue 2, 2023

Published on: 27 October, 2022

Page: [135 - 147] Pages: 13

DOI: 10.2174/1566523222666221004100858

Price: $65

Abstract

Gene therapy has proven to be extremely beneficial in the management of a wide range of genetic disorders for which there are currently no or few effective treatments. Gene transfer vectors are very significant in the field of gene therapy. It is possible to attach a non-viral attachment vector to the donor cell chromosome instead of integrating it, eliminating the negative consequences of both viral and integrated vectors. It is a safe and optimal express vector for gene therapy because it does not cause any adverse effects. However, the modest cloning rate, low expression, and low clone number make it unsuitable for use in gene therapy. Since the first generation of non-viral attachment episomal vectors was constructed, various steps have been taken to regulate their expression and stability, such as truncating the MAR element, lowering the amount of CpG motifs, choosing appropriate promoters and utilizing regulatory elements. This increases the transfection effectiveness of the non-viral attachment vector while also causing it to express at a high level and maintain a high level of stability. A vector is a genetic construct commonly employed in gene therapy to treat various systemic disorders. This article examines the progress made in the development of various optimization tactics for nonviral attachment vectors and the future applications of these vectors in gene therapy.

Keywords: Attachment carrier, non-viral vector, carrier optimization, gene therapy, Parkinson's disease, toxicity, immune response, efficacy.

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