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Optogenetic therapy restored partial vision in a blind 58-year-old man diagnosed with retinitis pigmentosa almost 40 years ago, according to a case report from the ongoing PIONEER study.
In this open-label phase I/IIa study, intraocular injection of an optogenetic sensor-expressing gene therapy vector combined with use of light-stimulating goggles was found to partially restore visual function in a patient with visual acuity of only light perception, reported José-Alain Sahel, MD, of the University of Pittsburgh, and colleagues in
Retinitis pigmentosa is an inherited neurodegenerative disease that breaks down light-receptive cells in the retina and can lead to complete blindness. More than 2 million people worldwide are affected by this progressive disease. There is no approved treatment, except for a gene replacement therapy that works only in an early-onset form of the disease caused by a mutation in the