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Gene therapy hope for blindness


A gene therapy that could treat blindness in people with ‘inherited retinal diseases’ is on the brink of being approved by regulators in America.

Research presented this week to the American Academy of Ophthalmology revealed that, during trials, the potentially game-changing treatment had positively affected 27 out of 29 people with Leber congenital amaurosis (LCA), a condition which exists from infancy, and eventually leads to blindness.

The 27 saw their vision improve to a level where they could ‘navigate a maze in low to moderate light’. While this doesn’t mean a return to full vision, it does offer a future without a cane or guide dog.

Ophthalmologist, Dr Stephen Russell, from the University of Iowa, is one of the team behind the gene therapy, which the researchers say – if approved – could lead to treatments for ‘more than 225 genetic mutations known to cause blindness’.

The U.S. Food and Drug Administration (FDA) will make its final decision in January, 2018. And hopes are high for a positive decision, after an FDA advisory committee unanimously backed it in October.

It is part of a growing trend, where gene therapies are redefining what is possible. One biotech industry expert wrote this year that they could spell “the end for blood cancer” with more breakthroughs turning the tables on cancer. A gene therapy from Novartis, approved by the FDA in August, was also labelled as having blockbuster potential. While one developed in Europe has been used to give a young boy with a skin disease, which had destroyed 80% of his epidermis, his life back.



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