Here’s to Good Science for 2019

Welcome to our final issue of 2018 – a year that will likely be best remembered for the controversy surrounding the birth of the first gene-edited babies – twin sisters, Lulu and Nana – in China. But Jiankui He – the scientist responsible for the “experiment” – will never be mentioned in the same breath as Edward Jenner, Alexander Fleming, or any other pioneering biomedical scientist of the past or present. If the claims made in He’s presentation to the International Human Genome Summit in Hong Kong are true, then his work falls far short of justifying the enormous risk involved – worse still, the radical research may have failed to achieve its basic aim.

He set out to create an HIV-resistant baby by using CRISPR to edit CCR5 – a gene encoding a receptor that plays a major role in the infection process. He told delegates that he chose CCR5 because some people naturally carry a mutation in CCR5 – a deletion known as delta-32 – which inactivates the gene. But according to several experts, the data presented suggest that both of the babies’ cells harbor multiple edited versions of the CCR5 gene – none of which are identical to the delta-32 mutation (1).

Before implanting the embryos, He had no way of knowing for sure if either of the twins would be protected from HIV – and we still don’t. Nor do we know if the new mutant genes will have any deleterious effects, as they have not even been studied in animal models. Despite all the red flags and ethical issues, the Associate Professor from the Southern University of Science and Technology in Shenzhen went ahead with the pregnancy anyway in the hopes of protecting the twins against a treatable disease.

One day (perhaps far in the future), we may be able to eradicate life-threatening diseases using embryonic gene editing, without the risk of off-target effects. And even then the question of whether we should do so is by no means straightforward. But in the present day? As discussed on page 10, He’s work looks destined to set the field backwards, and who knows how far?

What a stark contrast to the amazing work emanating from the cell and gene therapy field as a whole. John Rasko, President of the International Society for Cell and Gene Therapy (ISCT) and one of the authors of our supplement that accompanies this issue, says he will tell his grandchildren how 2017 was the year when the field hit the mainstream. Clearly, it hasn’t been plain sailing (and we present more than a few challenges in our supplement), but with perseverance, good science, and a clear unmet need in mind, it’s entirely possible to achieve great things under the full scrutiny of ethical and regulatory approval. Let’s hope the year 2019 follows the latter example.