A clinical trial which used CRISPR-Cas9 gene editing, named BRILLIANCE, treated patients with Leber Congenital Amaurosis (LCA). LCA is a genetic blindness that is caused by CEP290 gene mutations. In this experiment, fourteen patients, which included two under 17 years old, received surgical injections of a gene editing agent. According to the study published in the New England Journal of Medicine almost half of the participants noted having a significant increase in their visual quality.
Visual improvement was tested in a variety of ways, such as testing how bright a light needed to shine to be detected. Another test was navigating a physical maze to test how easily patients could find their way around. Out of the fourteen patients, eleven of them displayed improvement in at least one of the many tests, and six improved in two or more. More importantly, six of the participants reported experiencing better vision related to seeing color, also called cone-mediated vision. Cones are the type of visual receptors responsible for processing color.
So far the injection of the gene editing agent has shown no significant side effects that would prove this treatment to be harmful. The study's success provides hope for those with genetic forms of blindness as more effective gene editing agent injections could lead to a potential restoration of vision. However, this method of gene editing injections could be used in many other genetic disorders, which makes understanding its methods so much more important for the development of genetic medicine. This study could be a foundational pillar for the medical treatment of genetic disorders as a whole, not just in the field of ophthalmology.
https://medlineplus.gov/genetics/condition/leber-congenital-amaurosis/
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