Cribbs Adam P, Perera Sumeth M W
Computational Genomics and Training Centre (CGAT), MRC Weatherall Institute of Molecular Medicine, University of Oxford, John Radcliffe Hospital, Headington, Oxford, UK.
Campion Hall, Oxford, UK.
Yale J Biol Med. 2017 Dec 19;90(4):625-634. eCollection 2017 Dec.
Since its emergence in 2012, the genome editing technique known as CRISPR-Cas9 and its scientific use have rapidly expanded globally within a very short period of time. The technique consists of using an RNA guide molecule to bind to complementary DNA sequences, which simultaneously recruits the endonuclease Cas9 to introduce double-stranded breaks in the target DNA. The resulting double-stranded break is then repaired, allowing modification or removal of specific DNA bases. The technique has gained momentum in the laboratory because it is cheap, quick, and easy to use. Moreover, it is also being applied to generate more complex animal model systems. Such use of genome editing has proven to be highly effective and warrants a potential therapy for both genetic and non-genetic diseases. Although genome editing has the potential to be a transformative therapy for patients it is still in its infancy. Consequently, the legal and ethical frameworks are yet to be fully discussed and will be an increasingly important topic as the technology moves towards more contentious issues such as modification of the germline. Here, we review a number of scientific and ethical issues which may potentially influence the development of both the technology and its use in the clinical setting.
自2012年出现以来，被称为CRISPR-Cas9的基因组编辑技术及其科学应用在极短时间内就在全球迅速扩展。该技术利用一种RNA引导分子与互补DNA序列结合，同时招募核酸内切酶Cas9在目标DNA中引入双链断裂。随后修复产生的双链断裂，从而实现特定DNA碱基的修饰或去除。这项技术在实验室中发展迅速，因为它成本低廉、操作快速且简便。此外，它还被用于构建更复杂的动物模型系统。基因组编辑的这种应用已被证明非常有效，有望成为治疗遗传疾病和非遗传疾病的潜在疗法。尽管基因组编辑有可能成为一种变革性的治疗方法，但它仍处于起步阶段。因此，法律和伦理框架尚未得到充分讨论，随着该技术朝着诸如生殖系修饰等更具争议性的问题发展，这将成为一个日益重要的话题。在此，我们回顾一些可能会影响该技术发展及其在临床应用的科学和伦理问题。
