Gene therapy: a double-edged sword with great powers
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Gene therapy: a double‑edged sword with great powers Ran Tang1 · Zhigang Xu2,3 Received: 13 April 2020 / Accepted: 11 July 2020 © Springer Science+Business Media, LLC, part of Springer Nature 2020
Abstract Gene therapy is the treatment of a disease through transferring genetic material into cells of the patients. In the recent several years, gene therapy has experienced rapid progress and achieved huge success. Over two dozens of gene therapies have been approved for clinical use by the drug regulatory agencies from different countries. However, concerns about its efficacy and safety have accompanied gene therapy since its birth. In the present manuscript, we first introduce various strategies employed in gene therapy, which includes ex vivo gene delivery v.s. in vivo gene delivery; gene addition v.s. genome editing; inherited disease v.s. acquired disease; and somatic gene therapy v.s. germline gene therapy. Then we discuss the clinical outcomes of some approved gene therapies. We finish our discussion with the safety issues related to gene therapy. We will see that with the technology improvement, somatic gene therapy has been proved to be efficient and safe enough for clinical practice. However, germline gene therapy has important efficiency and safety issues at present, and should not be put into clinical practice before these issues are solved. Keywords Gene therapy · Adeno-associated virus · Genome editing · Cas9
Introduction Gene therapy is the treatment of a disease through transferring genetic material, either DNA or RNA, into cells of the patients. The transferred genetic material acts through one of the three following ways (i) enabling expression of the transferred gene, (ii) inhibiting the expression of a target gene, or (iii) modifying a target gene. The concept of gene therapy was first put forward nearly fifty years ago for treatment of inherited monogenic disorder [1]. Soon after that, its potential application was extended to acquired diseases such as cancer [2]. The possibility of durable cure through a single application of gene delivery or modification makes the concept of gene therapy very attractive. After nearly half-a-century’s intense work, gene therapy has achieved significant success, with over two dozens of gene therapies officially approved as clinically used drugs [3, 4] (Table 1). * Zhigang Xu [email protected] 1
Jilin University School of Law, Changchun 130012, China
2
Shandong University School of Life Sciences, Qingdao 266237, China
3
Shandong Provincial Collaborative Innovation Center of Cell Biology, Shandong Normal University, Jinan 250014, China
They have provided treatments for certain diseases otherwise untreatable or failed in traditional treatments. For example, spinal muscular atrophy (SMA) is a severe childhood monogenic motor neuron disease caused by SMN1 mutation, and only 8% of patients could survive through 20 months without permanent ventilatory support [5]. In contrast, all patients who received gene therapy Zolgensma survived through 20 months of
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