Researchers recently treated the first visually impaired patient. He received a new drug whose objective is to restore his sight. The fact is that this drug is based on CRISPR genetic modification.
The desire to give sight back to humans is at the heart of an experiment described in a press release published by Editas Medicine on March 4, 2020. This American genome-editing company has partnered with Oregon Health &Science University and Allergan plc, a pharmaceutical company also based in the United States. A first patient was treated in the phase 1/2 of a clinical trial baptized BRILLIANCE.
You should know that the patient's loss of sight is hereditary. Using CRISPR, the researchers are therefore seeking to eliminate the mutation of a gene in the CEP290 protein. However, the latter is at the origin of congenital amaurosis of Leber 10 (LCA10), visual blindness present in young children. In its press release, Editas Medicine mentions an experimental drug named AGN-151587 (EDIT-101), administered by subretinal injection.
You should know that this is the very first study to test on humans a genome-editing CRISPR drug in vivo , that is, inside the organism. In addition, this first phase intended to evaluate the treatment in question will concern no less than 18 patients. Researchers will consider several aspects:safety, tolerability and efficacy.
According to Prof. Mark Pennesi, the doctor assigned to the first patient treated, this trial is very important. This one brings science a little closer to the implementation of new and promising treatments for patients with pathogenic genetic mutations. However, we will have to wait before knowing the results of this trial which, of course, is not yet complete.
The last time we talked about CRISPR, it was also about research being conducted in the United States. A team from the University of Georgia has created albino lizards using CRISPR-Cas9 molecular scissors. The goal? Monitor oocyte fertilization in animals laying eggs.
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