Three out of fourteen
The genetic method of treating blindness works only in some patients
Editas Medicine announced that a clinical trial of CRISPR genome editing for the treatment of Leber congenital amaurosis 10 (LCA10) resulted in clinically significant vision improvements in only three out of fourteen patients.
This disease is one of eleven varieties of Leber's amaurosis, which differ from each other in which gene the mutation occurred, leading to vision loss. In this case, there is a genetic defect in the CEP290 gene, which is responsible for the synthesis of a protein necessary for visual receptors.
The new method of treatment consisted in the fact that patients received an injection of a modified virus containing components of the CRISPR-Cas9 system under the retina of the eye, which identified a defective section of DNA and cut it out. This was the world's first trial in which a gene editing system was injected directly into the body, rather than being applied to patients' cells in a laboratory Petri dish, which were then returned to their body.
Two patients with improved vision — a woman and a 14-year-old boy — had two defective copies of the CEP290 gene. Both of them received an average dose of the modified virus. The third patient with some improvement had only one defective copy, and he was among the five adult participants who received the highest of the three doses of CRISPR-loaded viruses.
Now the company has suspended registration for participation in the trial and is looking for a partner who will continue and help finance the program. According to representatives of Editas Medicine, one of the candidates is another company dealing with retinal diseases.
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