First Patient Receives In Vivo CRISPR Editing

Scientists say they have used the gene-editing tool CRISPR to fix a person's eyesight for the first time.

The treatment: According to the Associated Press, doctors dripped just a few drops of a gene-editing mixture beneath the retina of a patient in OR who suffers from leber congenital amaurosis, a rare inherited disease that leads to progressive vision loss.

Researchers at the Oregon Health & Science University in Portland, in collaboration with pharmaceutical companies Editas Medicine (USA) and Allergen (Ireland), encoded the components of the CRISPR gene therapy into a virus that was injected directly into the eye of the patient. They hope to know within weeks whether the approach is working and, if so, to know within two or three months how much vision will be restored.

In this world-first trial, the gene-editing tool was used to address mutations that cause a rare form of inherited blindness called Leber congenital amaurosis type 10.

This disease can not be treated with standard gene therapy, where a replacement gene is provided because it is too large to be incorporated into incapacitated viruses, the classical route for the transport of new genes into cells. "We need technology that will be able to deal with problems like these large genes".

Pierce, Albright and others stressed that only one patient has been treated so far and that the study, still at a very early stage, is designed primarily to determine whether injecting the gene-editing tool directly into the eye is safe.

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In a family seeking a solution to the blindness of inheritance, he received three calls a day.

The fact that gene-editing was performed within a live human is a "milestone moment for the gene-editing field" and especially for understanding the clinical potential of CRISPR enzymes, commented Satish K. Pillai, PhD, of the University of California San Francisco, who is not involved with the trial. "It's a bad disease", she said. "They have nothing right now".

"We're thrilled to be doing this because we could be helping open an era of therapeutic gene editing for many different disorders", Pierce said.

All of these studies have been done in the open, with government regulators' approval, unlike a Chinese scientist's work that brought global scorn in 2018. He Jiankui used Crispr to edit embryos at the time of conception to try to make them resistant to infection with the Aids virus. At a time when factual information is both scarcer and more essential than ever, we believe that each of us deserves access to accurate reporting with integrity at its heart.

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Some independent experts were hopeful about the new study. And that is here for the long term.

  • Douglas Reid