I have watched scientists, ethicists, patient advocacy groups, and others grapple with these topics at The Third International Summit on Human Genome Editing in London earlier this week.
There’s a lot of fun when it comes to gene editing. In the decade since scientists discovered that they can use CRISPR to edit the genomes of cells, numerous clinical trials have emerged to test the technology’s use in diseases. serious. CRISPR has been used to save some lives and transform others.
But not all is smooth sailing. Not all trials went as planned, and some volunteers died. Successful treatments are likely to be expensive, and therefore limited to a few wealthy people. And while these trials tend to involve changes to genes in adult body cells, some hope to use CRISPR and other gene-editing tools in eggs, sperm and Embryo. The specter of the design kids continued to loom over the field.
At the most recent summit, held in Hong Kong in 2018, He Jiankui, then working at Southern University of Science and Technology in Shenzhen, China, announced that he had using CRISPR on human embryos. The news of the first “CRISPR babies,” when they became known, caused a great uproar, as you can imagine. “We will never forget the shock,” Victor Dzau, president of the US National Academy of Medicine, told us.
He Jiankui ended up in prison and was was only released last year. And although genome editing was banned in China at the time – it has been outlawed since 2003 – the country has since enacted a series of additional laws designed to designed to prevent anything similar from happening again. Yaojin Peng of the Beijing Institute of Stem Cells and Regenerative Medicine told the audience that today, genetic genome editing is prohibited under criminal law.
There was much less drama at this year’s summit. But there are a lot of emotions. In a session on how gene editing can be used to treat sickle cell disease, Victoria Grey, a 37-year-old survivor of the disease, took to the stage. She tells the audience how her severe symptoms have disrupted her childhood and teenage years, while also disappointing her about her dream of training to be a doctor. . She described the severe pain that kept her hospitalized for months. Her children are worried that she might die.
But then she underwent a treatment that involved editing genes in cells from her bone marrow. Her new “supercells,” as she called them, changed her life. Within minutes of being infused with the edited cells, she felt reborn and shed tears of joy, she told us. It took seven to eight months for her to feel better, but after that, “I really started to enjoy the life I used to feel like it was just passing me by,” she says. I could see the typical stoic scientists around me wiping away tears.
Victoria is one of more than 200 people who have been treated with CRISPR-based therapies in clinical trialsDavid Liu of the Broad Institute of MIT and Harvard, who led development of new and improved forms of CRISPR. Trials are also underway for a range of other diseases, including cancer, inherited vision loss and amyloidosis.
