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

Author:

Khan Safir Ullah1ORCID,Khan Munir Ullah2,Khan Muhammad Imran34,Kalsoom Fadia4,Zahra Aqeela5

Affiliation:

1. Hefei National Laboratory for Physical Sciences at the Microscale, School of Life Sciences, University of Science and Technology of China, Hefei 230027, People’s Republic of China

2. MOE Key Laboratory of Macromolecular Synthesis and Functionalization, International Research Center for X Polymers, Department of Polymer Science and Engineering, Zhejiang University, Hangzhou, 310027 China

3. School of Life Sciences and Medicine, University of Science and Technology of China,Hefei 230027,People’s Republic of China

4. Department of Pathology, District Headquarters Hospital Jhang 35200, Punjab Province, Islamic Republic of Pakistan

5. Department of Family and Community Medicine. College of Medicine, University of Ha’il, Ha’il 81451, Saudi Arabia

Abstract

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.

Publisher

Bentham Science Publishers Ltd.

Subject

Genetics (clinical),Drug Discovery,Genetics,Molecular Biology,Molecular Medicine

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