CRISPR-CAS9 gene editing complex from Streptococcus pyogenes. The Cas9 nuclease protein (white) uses a guide RNA (blue) sequence to cut DNA (red) at a complementary site. Used in genome engineering and gene therapy. Illustration.

Who stands to benefit from – or lose out on – gene editing? Advances in our ability to tweak the human genome using CRISPR Cas-9 and similar techniques won’t only affect those at risk of genetic disorders. Women and disabled people are likely to be disproportionately affected. And people living in poor countries – who are at the greatest risk of disease – could miss out on the benefits.

These were among the points raised at a summit held by the US National Academies of Science and Medicine’s Committee on Human Gene Editing in Paris today. The committee will draw up a set of recommendations based on the questions and comments raised by the audience – a mix of scientists, ethicists and lawyers.

The CRISPR editing tool offers scientists a way to cut out sections of DNA and potentially replace them with others. Since it took the research world by storm last year, some have suggested it could be used to treat genetic disorders by precisely targeting the responsible genetic mutation. Disorders like Duchenne muscular dystrophy, which causes muscle degeneration, and osteogenica imperfecta, known as brittle bone disease, have been flagged as potential beneficiaries.

But the people who will probably be the first to have their lives altered by gene editing were missing from the discussion, says Jackie Leach Scully, a bioethicist based at Newcastle University in the UK. When researchers focus on correcting genetic mutations, they may be overlooking the individual’s personal experience of disability, she says.


“There are lots of intervening steps in between a person’s genotype and their experience of phenotype,” says Leach Scully.

Spectrum of opinion

Disability exists on a spectrum, and impacts each affected person’s life in a different way. Some will be happy to live with their disability, says Leach Scully. “Disabled and chronically ill people will hold a broad spectrum of opinion.”

Because of all this diversity, disabled people themselves must have a say in any policy recommendations, she says. Some might not like the idea of gene editing, and, at any rate, governments could be doing a lot more to improve the wellbeing of disabled people before planning on editing their genes.

Women also stand to be disproportionately affected by advances in gene editing. For a start, women tend to take on the bulk of caregiving for disabled people, and so will have to deal with the fallout of changes to treatments.

But women are also likely to be the first recipients of gene therapies to improve fertility. Just a couple of months ago, a UK-based team were given the go-ahead to experiment on human embryos, with the aim of identifying genes linked to embryo health, fertility and miscarriage. A team based in Sweden is set to start a similar project. “Both initial research and ultimately treatment involves early embryos or eggs,” says Leach Scully.

All the world

At the same time, people living in poor countries are left out of the conversation. While CRISPR debates over designer babies, cosmetic improvements and super-human enhancements rage on, researchers need to remember that developing countries stand to benefit hugely from gene editing, says Gemma Ortiz Genovese of charity Doctors without Borders, or Medicins Sans Frontieres.

“Based on the presentations [made at the summit], we’re looking at an elitist treatment,” she says. “We [at MSF] want to know how the benefits will be shared equally.”

Ortiz Genovese listed sickle cell disease, malaria and dengue among the diseases that collectively are responsible for hundreds of thousands of deaths per year in the developing world. Some research teams are looking into ways to gene edit mosquitos – which spread dengue and malaria – into oblivion by rendering them unable to reproduce.

“We think it’s important that technologies are managed through governance to ensure that the needs of people in developing countries are met,” she says.

The best way to do this is to provide incentives for companies to invest in the technology, says Soren Holm at the University of Manchester in the UK. “In the past that has been done by shaming them when they do not do it,” he says.

DIY biology

Any policy on human gene editing will also have to account for the DIY biology community – “citizen researchers” who do their own low-budget genetic experiments.

“People build their own hardware and teach themselves genetics,” says Bethan Wolfenden of Bento Bioworks. Wolfenden works with biology hobbyists, providing them with low-cost scientific equipment. “I’ve been approached by a lot of people who are interested in CRISPR and how they might be able to use it,” says Wolfenden.

While some may want to use the technique to improve their health, others just want feel they are experiencing the bleeding edge of technology first hand, says Wolfenden. And although DIY CRISPR may be some way off, it’s worth thinking about, she says. “Once they have access to the knowledge, we need policies in place to help them manage those projects.”

It is vital that gene editing community listen to the opinions of the people future treatments are likely to affect, says Leach Scully. “We don’t know a lot either about the science or the views of those people who we hope will benefit from these therapies,” she says. “We need to bear humility and modesty in mind.”