Abstract

Genome editing-mediated cell transplantations: Clinical, ethical and legal issues

Tetsuya Ishii, Professor, Hokkaido University

Genome editing technology, represented by CRISPR/Cas9, facilitates somatic and germline gene modifications. The introduction of site-directed nucleases efficiently disrupts endogenous genes, repairs mutations and adds exogenous genes at target sites in the human genome, potentially providing a myriad of medical benefits. However, there are some issues that should be addressed in genome editing-mediated cell transplantations. Autologous ex vivo somatic editing therapy and in vitro germline editing medicine are herein discussed from clinical, ethical and legal points of view. With regard to legal aspect, there is no significant problem in ex vivo somatic editing. By contrast, in vitro germline genome editing faces a de facto global consensus that no human germline should be genetically modified for reproductive purposes. However, current regulatory landscape (39 countries) suggests that human germline gene modification is not completely prohibited worldwide. Informed consent is obtainable from patients who will receive edited somatic cells. In the case of germline editing, the consent can be obtained from prospective parents. In doing so, the risk of off-target effects will be included in the explanation prior to obtaining informed consent. This presentation will discuss possible responses to upcoming clinical, ethical and legal issues raised by genome editing-mediated cell transplantation.