Gene therapy restores vision in some patients with inherited blindness

“For her entire life, college student Olivia Cook had only a small degree of central vision. It was as if she was watching the world through a straw hole, and in dimly lit places, she could not make out people’s faces, only their silhouettes,” reports CNN:

But after receiving an experimental gene-editing treatment to one of her eyes, she now can see things she never saw before. Cook was born with an inherited retinal disorder that causes blindness, a rare type of eye disorder historically called Leber congenital amaurosis or LCA. A few years ago, she decided to participate in a clinical trial that involved using the gene-editing tool CRISPR to correct the form of inherited blindness that she has…

This study is the first time that CRISPR has been used in the eyes of living people. “The results of this study provide proof of concept that CRISPR-Cas9 gene editing can be used safely and effectively to treat inherited retinal disorders,” said the study’s first author Dr. Eric Pierce, director of the Ocular Genomics Institute at Mass Eye and Ear and Harvard Medical School.

The trial was funded by the biotechnology company Editas Medicine …. “We’re really hopeful that…gene editing technologies will now be applied to other genetic forms of inherited blindness, and indeed other genetic diseases in general,” Pierce said…the 14 participants underwent a surgical procedure in which a drug called EDIT-101 that encodes the CRISPR gene-editing components was injected under the retina of one of their eyes….“That drug encodes the CRISPR-Cas9 gene-editing machinery, and once that starts working inside the retinal cells of those patients, it cuts out the mutation in CEP290 from the genome of their retinal cells, allowing the function of the CEP290 gene to be restored.”

When the first patients in the study were treated in 2020, it was the first time in medical history that a CRISPR-based medicine, resulting in gene-editing, was inserted directly into the living human body.

In related news, an England toddler has had her hearing restored in a pioneering gene therapy trial: “Opal Sandy was born unable to hear anything due to auditory neuropathy, a condition that disrupts nerve impulses traveling from the inner ear to the brain and can be caused by a faulty gene. But after receiving an infusion containing a working copy of the gene during groundbreaking surgery that took just 16 minutes, the 18-month-old can hear almost perfectly and enjoys playing with toy drums.”

A child recently was cured of a type of brain cancer that previously always killed kids who had it. A recently developed cancer vaccine for dogs doubles their survival rate.

A new gene therapy blocks the painful hereditary condition angiodema.

A virus is being used to cure deafness in new gene therapy. Researchers also discovered that a plant virus could be used to save crops from root-eating pests.

A genetically-modified chicken lays eggs that are non-allergenic, which could make it possible for people with egg allergies to eat eggs and the multitude of foods containing them.

Scientists have developed tiny robots made of human cells to repair damaged cells. Nanorobots are also being used to fight cancer: “In a major advancement in nanomedicine, Arizona State University scientists…have successfully programmed nanorobots to shrink tumors by cutting off their blood supply.”

“Surgeons and scientists have developed a world-first blood test for brain cancer that experts say could revolutionize diagnosis, speed up treatment and boost survival rates.” Despite advances in fighting other kinds of cancer, “brain tumors have remained notoriously difficult to diagnose. They affect hundreds of thousands of people worldwide each year, and kill more children and adults under the age of 40 … than any other cancer.”

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By GIL