The better edge of the CRISPR blade

Abstract

The 3-year jail sentence handed down on Dec 30, to Jiankui He, who created gene-edited babies using CRISPR, provides some closure on this case of ethical and medical misconduct. In the He case, human genome editing captured the media's full attention to the detriment of public and regulatory understanding. But human genome editing comes in two forms: heritable germline editing (in sperms, eggs, or embryos), and non-heritable somatic modifications (in all other cells). The fears around CRISPR-edited designer babies risk detracting attention from somatic cell editing and its potential useful clinical applications. Because germline editing results in genetic changes that are passed on to future generations, it is presently off limits to clinical researchers. In 2019, the WHO expert advisory committee on governance and oversight of human genome editing advised against any clinical work on human germline editing until all technical and ethical implications have been properly considered. By contrast, human somatic cell editing is familiar to the medical research community and regulatory agencies—in fact, it is fundamental to cellular and gene therapies, many of which are clinically approved. Among the fast-evolving genome editing techniques, CRISPR stands out for its precision. CRISPR is being tested in many trials to treat diseases ranging from sickle cell anaemia to congenital blindness. Whether it becomes an approved gene therapy hinges partly on the outcomes of these trials. The clinical benefits of CRISPR remain to be proven, but for many otherwise untreatable conditions, it is still one of the most exciting and promising techniques being developed. We shouldn't lose sight of this potential. What is needed are initiatives to develop global standards for governance and oversight of this important field. In August, for example, WHO launched a global registry of clinical trials on human genome editing. It is imperative that regulatory agencies worldwide monitor the evolution of genome editing technologies and provide clear guidance and pathways to facilitate their advancement from discovery to the clinic.