I’m actually at a loss to understand why that 3rd patient (with Usher’s syndrome) was enrolled. The whole focus of PYC’s treatment at this stage is on monogenic disease.
The 3rd patient has mutations in 2 genes, not one. So the (monogenic) treatment was bound to have little to no effect.
Surely they could have found a 3rd patient with just the single mutation?!?
Ann: Visual Improvement in Multiple RP11 Patients, page-13
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