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CRISPR is used to Cure Hereditary Blindness

Mar 4, 2020 by Ameer Helles

For the first time in an adult patient's body, the CRISPR gene-editing technique was used in an effort to cure a form of blindness.

The Oregon Health & Science Institute team injected three droplets of fluid that introduced CRISPR DNA fragments directly into the patient's eyeball, aiming to reverse a rare genetic disorder called Leber congenital amaurosis that causes early childhood blindness.

CRISPR restores sight
"We literally have the potential to take people who are essentially blind and make them see." Charles Albright, Chief Scientific Officer of Editas Medicine. said.

The CRISPR gene-editing technique has revolutionized scientific research by making it much easier to rewrite the genetic code. It also raises high hopes for the healing of many diseases. Previously, doctors had only used CRISPR to treat a minority of patients with cancer or rare blood disorders such as sickle cell anemia and beta-thalassemia. Although some of the initial findings were positive, it is still too early to know if the strategy is working.

In this new experiment, doctors at the Casey Eye Institute in Portland, OR, injected microscopic droplets carrying an innocuous virus that had been genetically modified to provide instructions for the CRISPR gene-editing machine.

CRISPR could have the power to create a permanent fix for certain diseases, but this also means that it could lead to permanent DNA damage – something that scientists want to avoid at all costs. Despite its success, scientists are vigilant to ensure that these methods are safe and do not cause any unintended consequences. They may be starting small by focusing on the eye, but what they learn from this research could have a significant impact in the years to come.

"We believe that the ability to edit inside the body is going to open entire new areas of medicine and lead to a whole new class of therapies for diseases that are not treatable any other way," says Charles Albright, the chief scientific officer at Editas.

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