A person with genetic blindness has become the first person to undergo direct human trials of CRISPR-Cas9 gene therapy. Scientists are conducting the first clinical trial to treat the hereditary eye disease, Leber’s congenital black mare (LCA10), in the first clinical trial, according to the Website of Nature. They said the trial was a landmark attempt to test the ability of the gene editing technique to remove mutations that cause LCA10.
LCA10 is the leading cause of blindness in children and there is no cure. IN THE LATEST TRIAL, COMPONENTS OF THE GENE-EDITING SYSTEM WILL BE ENCODED INTO THE VIRAL GENOME AND THEN INJECTED DIRECTLY INTO THE NEAR-LIGHT RECEPTOR CELLS OF THE PATIENT’S EYES, ACCORDING TO THE “GENE MAGIC SCISSORS.” Previously, when scientists treated eye diseases, they first removed the cell’s genome from the patient’s body, then used CRISPR-Cas9 to edit the genome, and then injected the edited cells back into the patient.
The latest experiment, called “BRILLIANCE,” is a genetic retinal disease specialist at Oregon Health and Science University, mark. Penles, who worked with editas Medicine in the U.S., hopes the technology will remove a mutation in the CEP290 gene that causes LCA10.
Artur A. Traditional gene therapy uses viruses to insert healthy copies of the mutated gene into the affected cells, but CEP290 is too big to fit the entire gene into the virus genome, Sidsian said. Moreover, although mutations in CEP290 invalidate photosensitive cells of the photoreceptors in the retina, these cells still exist and survive in the eyes of LCA10 patients.
“We hope CRISPR-Cas9 will reactivate these cells and improve the patient’s vision,” Penles said. “
Of course, this is not the first time gene editing technology has been used in human trials. Previously, zinc finger nucleases have been used directly in human clinical trials , an American company that uses a zinc finger-based therapy to treat Hunter syndrome. In the trial, the researchers inserted a healthy copy of an affected gene into a specific location in the liver cell genome, and while the treatment appeared safe, initial results suggested it might not help alleviate the symptoms of Hunter syndrome.
Fedor, a genome editing researcher at the University of California, Berkeley, said: “I’m very The direct use of CRISPR-Cas9 in the body is a significant leap forward compared to the treatment of cells in a petri dish, but there are also greater technical challenges and safety risks, Unov said.
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CRISPR-Cas9 gene editing technology, with its efficient and easy-to-use advantages, is rapidly gaining popularity in biology laboratories around the world, and countless scientists have used it as a powerful tool at hand. Whether it’s biopharmaceuticals, medical care, or farming, it’s useful. Arguably, having CRISPR-Cas9 serve human well-being is its best home. But as an emerging technology, CRISPR-Cas9 does not take long to apply. It may involve security risks and ethical issues in its application, which must be taken into account.