CRISPR Gene Editing Used to Restore Eyesight

Scientists Use CRISPR Tech to Edit DNA Directly Inside Blind Patient for the First Time

Scientists Use CRISPR Tech to Edit DNA Directly Inside Blind Patient for the First Time

Scientists say they have used the gene editing tool CRISPR inside someone's body for the first time. But the new study could open the door to using gene editing to treat incurable conditions that involve cells that can't be removed from the body, like Huntington's disease and dementia.

It's not clear when the procedure at Oregon's Casey Eye Institute took place, but scientists hope they'll know within months if it worked and how much of the patient's vision was restored, per NPR.

Though particular details, such as the patient's name and the exact date of the initial procedure, have not been released to the public, it is known that the individual in question has Leber congenital amaurosis - a rare eye disorder that in most cases causes impaired vision or blindness and, in some cases, can be associated with intellectual disability and delayed development.

Clinicians with OHSU's Casey Eye Institute performed the procedure for the BRILLIANCE clinical trial, which seeks to fix mutations in the CEP290 gene that cause a rare form of inherited blindness called Leber congenital amaurosis type 10; also known as LCA10 and CEP290-related retinal dystrophy.

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Even if the therapy doesn't work as intended, the trial is a milestone in gene editing, signaling that medicine is ready to make the leap from treating cells in a dish.

"For something like sickle cell disease, it's possible to do the editing on cells in the lab and then the corrected bone marrow can be reinfused", Pierce said.

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.

The feat of in vivo gene editing was first achieved in humans in 2017, with the use of zinc finger nucleases to insert the gene for an enzyme that was lacking in a man with Hunter syndrome.

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Scientists have used CRISPR, controversially, to create gene-edited babies, and the tool has been used outside the body to potentially fight cancer. "Right now they have nothing".

"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.

The study has the approval of government regulators, unlike a Chinese scientist's work that brought worldwide scorn in 2018. 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. The Guardian's independence means we can set our own agenda and voice our own opinions. This makes us different. It has protected our independence, which has never been so critical.

Some independent experts were hopeful about the new study. Every reader contribution, however big or small, is so valuable.

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