Researchers Use CRISPR To Repair Genetic Defect That Causes Blindness (dispatchtribunal.com)

hypnosec writes: In what has been claimed to be the first use of gene editing technique CRISPR for replacement of a defective gene associated with a sensory disease, researchers have repaired a genetic defect that causes blindness. The research that led to successful editing of defective genes responsible for retinitis pigmentosa (RP) – an inherited condition that causes the retina to degrade and leads to blindness in at least 1.5 million cases worldwide – was carried out using stem cells derived from a patient's tissue. Published in Scientific Reports, the study paves the way for using CRIPSR therapeutically to treat eye diseases.

Comments

Re:Side Effects

[Pharmboy]

I have a friend with Friedrich's Ataxia, and CRISPR is one of the silver bullets she's praying for. FA cripples then kills you: wheel chair by 25, dead by 40 is often the case (it hardens the heart so it can't pump). While CRISPR has some unknowns and risks, having FA is a certainty. FA affects a single gene pair, so if you can replace either side of that gene, you have solved the problem, the mitochondria will start producing frataxin again, and the nerves will stop being slowly destroyed.

There are no treatments and since it is so rare (1 in 50,000 have it in the US, 1 in 30k in Europe, almost no one in Africa or Asia), few are investing in finding a cure or treatment. FA isn't the only orphan disorder like this. So yes, I'm quite happy to see CRISPR move forward.