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Current Gene Therapy

Author(s): S.P. Simna and Zongchao Han*

DOI: 10.2174/1566523221666210419090357

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Prospects of Non-Coding Elements in Genomic DNA Based Gene Therapy

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Abstract

Gene therapy has made significant development since the commencement of the first clinical trials a few decades ago and has remained a dynamic area of research regardless of obstacles such as immune response and insertional mutagenesis. Progression in various technologies like next-generation sequencing (NGS) and nanotechnology has established the importance of non-- coding segments of a genome, thereby taking gene therapy to the next level. In this review, we have summarized the importance of non-coding elements, highlighting the advantages of using full- length genomic DNA loci (gDNA) compared to complementary DNA (cDNA) or minigene, currently used in gene therapy. The focus of this review is to provide an overview of the advances and the future of potential use of gDNA loci in gene therapy, expanding the therapeutic repertoire in molecular medicine.

Keywords: Gene therapy, cDNA, genomic DNA, non-coding DNA, gene expression, polygenic diseases.

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