Abstract
The method of adding multiple copies of transgenes, which was adopted in early gene therapy, has had a variety of adverse effects as well as a problem of controlling experimental outcomes. The development of site-specific nucleases, specifically CRISPR-Cas9, has brought new promise to the field but has also given rise to ethical dilemmas. Pre-clinical research has been conducted to correct mutated genes by establishing induced pluripotent stem cells, and primary clinical research has been carried out on delivering gene editing tools and transplanting corrected cells to patients. Germline editing requires strict guidelines because it leads to permanent genetic change that will affect future generations. This article discusses how to avoid therapeutic misconceptions during translation from pre-clinical to primary clinical research, how to estimate the social risks and benefits often neglected in the risk/benefit analysis, and how to decide the specificity of gene targeting effects. In addition, this article addresses the status of embryos, a topic that is largely ignored in germline editing research. As gene editing technology advances, the legislation and regulations governing clinical practice based on conventional gene therapeutic research should be reconsidered.
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