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Current Gene Therapy

Author(s): Natalie J. Holl, Han-Jung Lee and Yue-Wern Huang*

DOI: 10.2174/1566523220666201208092517

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Evolutionary Timeline of Genetic Delivery and Gene Therapy

Page: [89 - 111] Pages: 23

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Abstract

There are more than 3,500 genes that are being linked to hereditary diseases or correlated with an elevated risk of certain illnesses. As an alternative to conventional treatments with small molecule drugs, gene therapy has arisen as an effective treatment with the potential to not just alleviate disease conditions but also cure them completely. In order for these treatment regimens to work, genes or editing tools intended to correct diseased genetic material must be efficiently delivered to target sites. There have been many techniques developed to achieve such a goal. In this article, we systematically review a variety of gene delivery and therapy methods that include physical methods, chemical and biochemical methods, viral methods, and genome editing. We discuss their historical discovery, mechanisms, advantages, limitations, safety, and perspectives.

Keywords: Nonviral, CPPs, viral, ZFNs, TALENs, CRISPR, gene therapy, genetic delivery, genome editing.

Graphical Abstract

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