We recently posted an article about what the CRISPR gene-editing technology is and why it’s been in the news lately, but there’s more big news to follow up with.
Highlights from MIT Technology Review article:
While there has been much ado about how easy and effective CRISPR is in animals like mice, Katrine Bosley, CEO of the biotech startup, Editas Medicine, has announced plans to use the gene-editing technology on people by 2017. Their goal is to use CRISPR to help bring sight back to people who suffer from a very rare disease, known as Leber congenital amaurosis. Only about 600 people in US have the condition. Researchers know exactly which gene causes the disease, and because of it’s location in the eye, “doctors can inject treatment directly under the retina.”
Antonio Regalado, author of the article, writes: “Editas picked the disease in part because it is relatively easy to address with CRISPR, Bosley said. The exact gene error is known, and the eye is easy to reach with genetic treatments. ‘It feels fast, but we are going at the pace science allows,’ she said. There are still questions about how well gene-editing will work in the retina and whether side effects could be caused by unintentional changes to DNA.”
Editas will continue research in the lab and on animals before they attempt research on humans.