On 7 October 2020, the Nobel Prize in Chemistry has been awarded jointly to Emmanuelle Charpentier and Jennifer A. Doudna "for the development of a method for genome editing". This is the first time a Nobel Prize has been awarded to scientists for their work in CRISPR!
Over the past decade, the CRISPR-Cas system has transformed our ability to manipulate specific DNA and RNA in living cells of diverse species. The discovery and its subsequent application in clinics has bridged the gap between fundamental science and translational medicine. Scientists from all over the world have carried out numerous studies on CRISPR, and within just a decade, their work has expanded from basic research to actual application in human clinical trials. The 2020 Nobel Prize, to Emmanuelle Charpentier and Jennifer A. Doudna, is not only a recognition of the laureates’ achievements, but also a recognition of the achievements pertaining to the whole field. It reflects the confidence of the Nobel committee in future applications of gene editing technology, which may have far-reaching significance.
The Nobel Prize comes just in time!
On March 2020, an adult with congenital blindness became the first person to receive an in vivo CRISPR-based therapy, in the clinical trial sponsored by Editas Medicine and Allergan. In recent years, the practice of using CRISPR to make edits in somatic cells for purposes of treating genetically inherited diseases is growing rapidly and a few programs are already under clinical trials.
Meanwhile, strict and effective supervision are urgently needed.
The impacts of gene editing are widespread on humans and many other species, and even the entire environment. Particularly, the rapid development of gene editing technology and the He Jiankui event has aroused worldwide concern and discussion regarding gene editing in human germ cells. Strict and effective supervision of gene editing is urgently needed.
For some parents with serious genetic disorders, gene editing technology could help them have a healthy and biologically related child without passing down any genetic diseases. But as of today, it is still too risky to use any kind of gene editing technique in embryos which are destined for implantation, as claimed by an international high-profile commission panel. The commission convened by the National Academy of Sciences (NAS) and the UK Royal Society, released a much-anticipated 200-page report on Heritable Human Genome Editing (HHGE). The report charts a course for limited use of HHGE to help couples have a healthy, biologically related child, once several important criteria have been met. As Rick Lifton, Rockefeller University president and HHGE commission co-chair said:
"Any country considering HHGE should have mechanisms in place to ensure competent regulatory bodies to establish the conditions are met. If not, no clinical use of HHGE should go ahead... Decisions should be made with a full international assessment on safety and efficacy, and each country “should be transparent about what’s going on in their country.”
The World Health Organization has called another international commission and will issue a companion report focusing on the ethical and societal issues surrounding HHGE in the next few months.
Besides the ethical and social issues, some technical challenges relating to the use of the CRISPR system in vivo treatment still linger. The possibility of human immune responses to its bacterial components, multiplex gene editing with CRISPR, and the delivery of CRISPR components into cells are just a few examples.
We have faith that the gene editing techniques represented by CRISPR, including zinc finger nucleases and TALEN nucleases, will be more widely used in medicine and agriculture. Thus, it is becoming more urgent to regulate and supervise the application of gene editing technology in human cell genome editing.
National Academy of Medicine, National Academy of Sciences, and the Royal Society. 2020. Heritable Human Genome Editing. https://doi.org/10.17226/25665.