OHSU Doctors Try 1st CRISPR Editing In The Body For Blindness


For the first time, scientists say they used the gene-editing tool CRISPR inside the body of an adult patient to treat a rare form of blindness.

A patient recently had the procedure done for an inherited form of blindness, the companies that make the treatment announced Wednesday.

For now, there are no details concerning the procedure, such as when it took place or how the patient is fairing.

"We believe that the ability to edit inside the body is going to open entire new areas of medicine and lead to a whole new class of therapies for diseases that are not treatable any other way", Charles Albright, chief scientific officer of Editas told NPR.

The people in this study have Leber congenital amaurosis, caused by a gene mutation that keeps the body from making a protein needed to convert light into signals to the brain, which enables sight.

They are born with little vision and often lose what they have within a few years.

The virus had been engineered to instruct the cells to create CRISPR machinery.

So they aim to edit or remove the mutation by making two cuts around it.

According to doctors, the hourlong treatment involves that patient being placed under anesthesia while surgeons drip three drops of a fluid containing the microscopic gene-editing tool into the individual's eye.

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The lead author of the study, Eric Pierce, a professor of ophthalmology at Harvard Medical School and director of the Inherited Retinal Disorders Service at Massachusetts Eye and Ear said that the team of scientists are "Helping open, potentially, an era of gene-editing for therapeutic use that could have impact in many aspects of medicine".

The physicians think they need to fix one-tenth to one-third of the cells to restore vision. However, all previous animal tests showed that this treatment could correct the cells defective genetic code. Infections and bleeding are relatively rare complications. Many scientists believe that CRISPR is a simple tool for detecting and cutting DNA at specific sites, so interest in new research is very high.

However, this isn't the first time that gene-editing has been used in the human body. "We need technology that will be able to deal with problems like these large genes".

In one day, she had three calls from families seeking solutions to inherited blindness.

"It's a awful disease", he said. "Right now they have nothing". "It makes for a good first step for doing gene editing in the body".

The surgery itself carried minimal risks, and as the tool does not travel to any other part of the body, other than the eye, if something did go wrong the chance of harm would be very small.

All of these studies have been done openly, with the approval of the Chinese regulator, in contrast to the work of a Chinese scientist in 2018, which was condemned internationally in 20 sc. He Jiankui used Crispr to edit embryos at the time of conception to try to make them resistant to infection with the Aids virus. Changes in fetal "DNA" can go to future generations, as opposed to the work now being done in adults for the treatment of illness. This has been a turbulent decade across the world - protest, populism, mass migration and the escalating climate crisis. More information is in today's announcement from the trial sponsors.

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