The World Health Organization (WHO) has said no to the controversial subject of in utero gene editing, which gained international headlines and infamy last November after scientist He Jiankui announced the birth of the world's first CRISPR babies. Meanwhile, doctors in the US are getting ready to kickstart projects to treat conditions as diverse as sickle cell disease and inherited blindness using the same gene-editing technique – with the crucial difference being that their patients will be children and adults, not embryos.
The WHO's director-general issued a statement advising "regulatory or ethics authorities to refrain from issuing approvals concerning requests for clinical applications for work that involves human germline genome editing."
The notice does not outright ban the practice of using CRISPR in utero. Instead, it offers a firm recommendation condemning the practice and makes it extremely clear where the UN's public health agency stands on the matter. But while it is likely to quash enthusiasm to pursue this type of research at a national or institutional level, it might be less effective against more rogue actors – like, for example, a biologist at Russia's largest IVF clinic who recently announced plans to cure inherited deafness with CRISPR.
"Human germline genome editing poses unique and unprecedented ethical and technical challenges," said WHO Director-General Dr Tedros Adhanom Ghebreyesus.
"I have accepted the interim recommendations of WHO’s Expert Advisory Committee that regulatory authorities in all countries should not allow any further work in this area until its implications have been properly considered."
The debate surrounding CRISPR babies will continue with the WHO's Expert Advisory Committee expected to reconvene in Geneva in late August, where they plan to discuss ways to "deter and prevent irresponsible and unacceptable uses of genome-edited embryos to initiate human pregnancies."
In other CRISPR news: This week, CRISPR scientists in the US have taken the exciting step of announcing the first study to test CRISPR inside the human body in order to cure an inherited form of blindness. People who have the condition (a form of Leber congenital amaurosis) are born with normal eyes but without the gene to convert light into brain signals (and, thus, "produce" sight). Symptoms tend to start in childhood and get worse over time, with some patients losing vision completely.
The idea is to use the cut-and-paste design of CRISPR to add a healthy version of the missing gene in patients aged 3 years and up, involving a one-time procedure only. Unlike with CRISPR babies, the changed genes cannot be inherited so will not be passed down to any future generations. Trials are expected to begin in the fall.
Meanwhile, trials to cure up to 45 adults of sickle cell disease have already begun, with one patient discussing her experience on NPR. This, reports CNN, is the first time patients in the US will be treated for a genetic disease using CRISPR.
The disease can cause pain, anemia, organ damage, blindness, and a shortened lifespan and is thought to affect around 100,000 Americans, primarily those of African ancestry. The plan here is to remove premature cells from the bone marrow and then "edit" them so that they start to increase the production of fetal hemoglobin – a type of hemoglobin normally found in newborns and fetuses, which should make the blood cells less sticky and, therefore, less likely to stick together.
[H/T: Wired; Associated Press; NPR; CNN]
