Early-stage clinical research reports that a small sample of individuals with genetic blindness had measurable improvements in eyesight due to injectable gene therapy.
Fourteen individuals with Leber Congenital Amaurosis (LCA), an uncommon genetic disorder that results in partial or total blindness in newborns, were selected by the researchers.
A single injection of a gene-editing drug improved the eyesight in one eye in eleven out of the fourteen cases, according to a study published in the New England Journal of Medicine.
Using gene editing, our patients’ daytime vision has been greatly improved—they are the first congenitally blind children to receive this kind of treatment, according to researcher Dr. Tomas Aleman, a pediatric ophthalmologist at the Children’s Hospital of Philadelphia.
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Gene Therapy
Director of the Mass Eye and Ear Ocular Genomics Institute and Berman-Gund Laboratory for the Study of Retinal Degenerations in Boston and principal researcher Dr. Eric Pierce said, “We consider the early results promising, while more research is needed to determine who may benefit most.”
It’s significant, Pierce added, to hear from multiple people how happy they were to finally be able to see the food on their plates. “Unfortunately, the majority of people with inherited retinal disorders are not able to read any lines on an eye chart and are left with no treatment options.”
According to studies, 2 to 3 of every 100,000 newborns born each year are affected by LCA.
Most infants with LCA are born blind, and those who aren’t usually start to lose their vision by the time they are six months old.
Gene Therapy
According to experts, mutations in the centrosomal protein 290 (CEP290) gene cause LCA.
According to experts, these mutations lead to abnormalities in the rods and cones of the retina, which are responsible for detecting incoming visual signals and converting them into electrical impulses that are received by the brain.
According to studies, the most common cause of inherited blindness that occurs in the first ten years of life is mutations in the CEP290 gene.
Gene Therapy
Researchers used CRISPR-Cas9, a gene editing toolset that functions effectively as a pair of gene-level scissors, to come up with a genetic answer. The instrument has the ability to remove a section of a defective gene, possibly rendering it functional following the edit.
According to researchers, the ensuing clinical trial demonstrated that this was the first time a patient had ever gotten a CRISPR-based gene therapy straight into their bodies.
Findings indicated that:
- Of the four evaluated vision outcomes, at least one showed improvement in 11 subjects, or approximately 79%.
- Six individuals, or almost 43%, demonstrated improvement in two or more of the outcomes.
- Six individuals, or almost 43%, reported having a better quality of life due to their vision.
- Clinically significant improvements in visual acuity, or the ability to distinguish letters and objects on a chart, were seen in 4 subjects, or roughly 29% of the total.
Gene Therapy
“We’ve shown that we can safely administer a CRISPR-based gene editing therapeutic to the retina and have outcomes that are clinically meaningful,” a news statement from Editas Medicine, the biotech business based in Cambridge, Massachusetts, stated through the chief medical officer, Dr. Baisong Mei.
Early in 2020, the Casey Eye Institute at Oregon Health & Science University in Portland performed the first CRISPR injection treatment for a patient with lipomatosis.
“Hearing a patient describe how their vision has improved after a treatment is the most rewarding thing a doctor can do,” stated OHSU professor of ophthalmology Dr. Mark Pennesi in a news release.
One of our study participants gave multiple examples, such as finding their phone after misplacing it and recognizing the presence of small lights on their coffee maker to indicate that it is operating, Pennesi added. “Those who are normally sighted may find these kinds of tasks insignificant, but for those who have low vision, these kinds of improvements can have a significant impact on their quality of life.”
Gene Therapy
The gene treatment was given to other LCA patients at the following locations: Mass Eye and Ear; Miami’s Bascom Palmer Eye Institute; Ann Arbor, Michigan’s W.K. Kellogg Eye Center; and the Children’s Hospital of Philadelphia’s Scheie Eye Institute.
Twelve people and two children received the treatments, according to the researchers. For a year, participants were observed every three months; after that, there were fewer, often for two more years.
The clinical trial was sponsored by Editas and was put on hold in November 2022 in order to look for alternative business partners to work with on the development of the experimental treatment.
In order to collaborate with Editas to carry out more extensive studies, researchers are currently investigating the possibility of partnering with other business partners.
Gene Therapy
Gene Therapy
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