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First Human CRISPR Trial in the US Aims to Cure Inherited Blindness

First Human CRISPR Trial in the US Aims to Cure Inherited Blindness

Mathias Sundin
Mathias Sundin

Gene editing is advancing at a faster pace than most of us can keep up with. One significant recent announcement was gene editing tool CRISPR’s application to non-genetic diseases thanks to a new ability to edit single letters in RNA.

Even as CRISPR reaches milestones like this, scientists continue to find new uses for it to treat genetic conditions. The next one that will hit clinics is a CRISPR treatment for a form of blindness called Leber congenital amaurosis (LCA).

Having been approved by the FDA in December, the treatment will be the first of its kind to be trialed in the US.

What LCA Is

LCA is a group of inherited disorders that cause severe vision loss at birth. Both parents must have a defective gene for the condition in order for a child to inherit it; 2 to 3 out of every 100,000 babies are born with…
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