Doctors recently used CRISPR to help treat a patient suffering from blindness (Image credit: Maurizio De Angelis/Science Photo Library)

 

Over the past few decades, researchers and doctors have been looking for ways to help restore sight for the blind. But one team of scientists made a significant breakthrough recently by using CRISPR inside the human body for the first time. Doctors at the Casey Eye Institute at the Oregon Health & Science University in Portland use the tool to alter the genes linked to a particular form of inherited blindness. Those behind the procedure say this has the potential to not only restore vision but lead to new medication used to target and alter DNA.

 

But let’s take a step back and look at what exactly CRISPR is. CRISPR is a recent technology used to edit genes. It’s shorthand for Clustered Regularly Interspaced Short Palindromic Repeat. They’re specific stretches of DNA and its associate protein, CAS9m is an enzyme capable of cutting strands of DNA. Based on the same method that bacteria use when fighting against viruses, it saves copies of a virus’ DNA, and then uses CAS9 to compare those copies to new virus’ DNA, and then cut out any 100% matches it finds. It’s been used for other scientific research, but this latest news is the first time it’s been used in the human body.

 

The patient who received the treatment suffers from Leber congenital amaurosis, an eye disorder caused by a gene mutation that prevents the body from making the needed protein to convert light into signals to the brain, which enables sight. It can’t be treated with the standard gene therapy because the required gene is too big to fit inside the disabled viruses that are used to carry it into cells. The CRISPR procedure took an hour and consisted of dripping drops of a fluid with the CRISPR DNA through a hair-wide tube into the linings in the back of the patient’s eyes.

 

While doctors at Casey Eye Institute at Oregon Health & Science wouldn’t provide details on the patient or when the procedure occurred, they did reveal that if the first few attempts went well, they would test it on 18 adults and children next. We’ll have to wait to find out since it may take up to a month to see if it worked to restore vision. Doctors say the surgery has little risks, but the biggest concern with the procedure is unintended changes in other genes caused by CRISPR. Doctors are doing all they can to minimize these risks.

 

Still, doctors and researchers are very excited about this breakthrough. “We literally have the potential to take people who are essentially blind and make them see,” said Charles Albright, chief scientific officer at Editas Medicine, the Cambridge, Massachusetts-based company developing the treatment with Dublin-based Allergan. “We think it could open up a whole new set of medicines to go in and change your DNA.”

 

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