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 as 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 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.
