Blind patients watch their families and recolor themselves after pioneering treatment
Visually impaired patients were able to recognize their families for the first time in years after volunteering for a pioneering gene editing experiment.
Michael Kalberer, 43, and Carlene Knight, 54, both suffer from an incurable eye disease that left them blind as adults.
Earlier this year, they were one of seven patients who allowed scientists to modify their DNA by injecting the CRISPR gene-editing tool.
Their sight is not fully restored, but they can see colors, navigate the hallways and create silhouettes.
Calverer from Long Island has revealed that he was able to recognize his loved ones on the dance floor at his cousin’s wedding. This has been impossible for years.
He told National Public Radio: It was a very, very fun and enjoyable time. “
Born in Portland, Oregon, Knight said the colors were finally visible from childhood, and described them as “just wonderful.” To celebrate, she dyed her hair in her favorite color, green.
The couple signed up for an experimental treatment at Oregon Health & Science University in May.
It was the first time that CRISPR, a promising treatment for diseases like sickle cell anemia, had been used to alter genes in the human body.
Michael Calverer, 43, of Long Island, has revealed he was able to recognize his loved ones on the dance floor at his cousin’s wedding. This has been impossible for years.
Carlene Knight, 54, of Portland, Ore., Said she had only been able to see the colors for the first time since childhood and described them as “just wonderful.”
Kalberer and Knight were both born with a rare inherited eye disease called Leber’s congenital amaurosis (LCA).
Victims have rods and cones (cells that detect weak light and bright light respectively) in the retina, but these do not work properly.
Some are born blind, while others lose their sight over time. This is the case for Calverer and Knight.
According to the National Organization for Rare Diseases, stroke is estimated to affect one or two babies per 100,000 live births.
What is genome editing?
Genome editing allows scientists to make changes to their DNA, resulting in changes in physical characteristics.
To do this, scientists use various techniques.
These techniques act like scissors, cutting DNA at specific places.
Scientists can then remove, add, or replace the cleaved DNA.
The first genome editing technology was developed in the late 1900s.
Recently, a new genome editing tool called CRISPR, invented in 2009, has made DNA editing easier than ever.
Source: National Institute for Research on the Human Genome (NHGRI)
Both patients have a version of the disease caused by a defect in the CEP290 gene, which regulates proteins that keep the eyes healthy.
CRISPR typically requires cells to be removed from the body, edited in the lab, and returned to the patient, which is not possible with diseases like stroke.
This is because retinal cells are too fragile to be removed, modified or reinserted, NPR reported.
In this study, three small incisions were made in Knight’s left eye and Calverer’s right eye, and billions of benign viral particles with CRISPR technology were inserted.
Then CRISPR suppressed genetic mutations in cells that affect rods and cones, which prompted the body to produce genes that were working properly.
Scientists initially operated on one eye, but will operate on the other eye if the patient continues to improve.
Not all of the patients in the trial saw results for unknown reasons.
And for people like Knight and Calverer who have seen profit, their vision is still far from perfect.
However, the results are so promising that researchers have been given the green light to move to a larger patient group.
Mr Calverer said if he could create shapes and lights and see the surroundings well, he could eat outside and restore some normalcy to his life.
Born in Portland, Oregon, Carlene Knight, 54, is one of two patients with a rare inherited eye disease called Leber’s congenital amaurosis (LCA) while her DNA is in her body. We are undergoing a new CRISPR study to confirm the modified gene.
Another patient being studied in hopes of restoring vision is Michael Calverer, 43, of Long Island, New York.
It’s too early to tell if the technology is still working, but the team expects it to prevent further vision loss or even restore vision.Photo: Night
“Looking at the plate of food, if you have a spoon or a utensil in it, you can see the edge of the utensil on the outside of the bowl or the plate.
“So these changes are very, very important to me.”
Surprisingly, he also got to see the colors for the first time in a few years. About a month after the operation, he noticed it as the red car passed by.
Mr. Calverer was able to enjoy the simple enjoyment of life, like watching the sunset with his friends.
Knight also likes his restored ability to make colors and tells NPR:
“It’s one of the things I’ve enjoyed with a little sight since I was a kid.
“But now I understand how bright they were when I was a kid, because now I can see them better. It’s just amazing.
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