A person with a genetic condition that causes blindness has become the first to receive a CRISPR–Cas9 gene therapy administered directly into their body.
The treatment is part of a landmark clinical trial to test the ability of CRISPR–Cas9 gene-editing techniques to remove mutations that cause a rare condition called Leber’s congenital amaurosis 10 (LCA10). No treatment is currently available for the disease, which is a leading cause of blindness in childhood.
Super-precise CRISPR tool enhanced by enzyme engineering
For the latest trial, the components of the gene-editing system – encoded in the genome of a virus — are injected directly into the eye, near photoreceptor cells. By contrast, previous CRISPR–Cas9 clinical trials have used the technique to edit the genomes of cells that have been removed from the body. The material is then infused back into the patient.
Read the full story here: https://www.nature.com/articles/d41586-020-00655-8