A Shot of CRISPR Could Restore Sight to the Blind
It’s the first time the gene-editing tool has been used inside the human body
Scientists have used the gene-editing technique CRISPR in an attempt to restore vision in a patient with a type of inherited blindness, researchers announced last week.
The experimental treatment is meant to snip out a genetic mutation that causes severe visual impairment, often starting at birth. It also marks the first time doctors have used CRISPR directly inside a person’s body. In a few weeks, they hope to know whether the treatment is working. If it works and is safe, it opens up the possibility of using CRISPR against many more conditions.
CRISPR is a powerful technique often likened to molecular scissors for its ability to cut DNA with relative ease. Scientists think it could be applied to a variety of diseases to delete or correct faulty segments of DNA. Repairing DNA at its source could stop or reverse diseases in their tracks rather than just treating symptoms like the vast majority of drugs are designed to do. The hope is that CRISPR could be a one-time, permanent treatment.
It has already been used to treat a handful of people with genetic blood disorders and advanced cancers. In those cases, doctors removed cells from patients’ bodies, used…
