Gene regulations and delivery vectors for treatment of cancer

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Journal of Pharmaceutical Investigation (2020) 50:309–326 https://doi.org/10.1007/s40005-020-00484-x

REVIEW

Gene regulations and delivery vectors for treatment of cancer Ming Chen1 · Yu‑Xin Ren1 · Ying Xie1 · Wan‑Liang Lu1 Received: 12 March 2020 / Accepted: 13 April 2020 / Published online: 19 April 2020 © The Korean Society of Pharmaceutical Sciences and Technology 2020

Abstract Background  Resistant residual cancer and cancer stem cells after comprehensive treatment often result in the recurrence of cancer. The refractory nature of cancer is an important scientific problem to be solved. Area covered  An emerging gene regulation strategy of cancer is involved in the cross field of chemistry, genomics, bioinformatics and pharmaceutical sciences, which could offer a possible solution to a curative strategy of cancer. Expert opinion  In this review, we dealt with the advances and applications of major gene regulation strategies, including DNA modification, gene editing and RNA regulations. Moreover, we also analyzed the representative transfection carriers for delivering the genetic material into cancer cells, including viral and non-viral vectors. Herein, we conclude that the gene regulation by a safe and high efficient transfection vector would be promising to provide a new treatment strategy for fully recovery of cancer disease. Keywords  DNA modification · Gene editing · RNA regulation · Gene delivery · Cancer

Introduction Treatment of cancer covers a comprehensive strategy, mainly involving chemotherapy, surgical therapy, radiotherapy and immunotherapy, among which, chemotherapy plays a fundamental role in sweeping cancer cells during whole treatment procedure. However, resistant residual cancer and cancer stem cells after comprehensive treatment often result in the recurrence of cancer. The refractory nature of cancer remains as unsolved scientific and clinical issues. The achievements in human genome project inspire scientists to continuously investigate new strategies by biological approaches, in which gene regulation emerges promising prospect to get a curative solution to cancer therapy. Since the human genome sequence and associated identification of approximately 30,000 genes were elucidated in the year 2001 (Lander et al. 2001; Venter et al. 2001), there have many new treatment methods been developed in the Ming Chen and Yu-Xin Ren have contribute to this work equally. * Wan‑Liang Lu [email protected] 1



State Key Laboratory of Natural and Biomimetic Drugs, Beijing Key Laboratory of Molecular Pharmaceutics and New Drug System, and School of Pharmaceutical Sciences, Peking University, Beijing 100191, China

field of gene therapy. Based on genomic knowledge, gene regulation is used as a possible measure which is performed by putting corrective genetic material into cells to treat a variety of diseases, including cancers (Dunbar et al. 2018). Gene regulation enables to correct the mutated genes of cancer cells, and promote the differentiation of cancer stem cells. Accordingly, gene regulation has the