The gene-editing tool CRISPR has been used to address a blindness-causing gene mutation at Oregon Health & Science University for a clinical trial.
The procedure marks the first time CRISPR has been used to edit human genes within the body, which is also called in vivo gene editing. Previous gene-editing methods have involved editing genetic material after it was removed from the body. The trial’s gene editing approach is designed to be permanent, but not passed onto the offspring of those who receive it.
Clinicians performed the procedure for the clinical trial, which seeks to repair mutations in the CEP290 gene that cause a rare form of inherited blindness called LCA10 or CEP290-related retinal dystrophy. Most people with the mutation are either born blind or become blind within the first decade of their life. Currently, patients living with LCA10 have no approved treatment options.
This marks a major advance for all of the medicine as we enter an era of repairing inherited genetic diseases.
The doctors say that being able to edit genes inside the human body is incredibly profound. Beyond potentially offering treatment for a previously untreatable form of blindness, in vivo gene editing could also enable treatments for a much wider range of diseases.
News Source: OHSU
