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Gene therapy presses on for Stargardt disease. — Weekly Glance

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Nanoscope Therapeutics announced that the first patient has been dosed in a phase 2 trial of its Multi-Characteristic Opsin (MCO-010) ambient-light activatable optogenetic monotherapy for Stargardt disease.


That was a mouthful ... let's discuss.

Optogenetics is a technology that uses light to control genetically modified neurons.

We heard about optogenetics a while back, after a man with retinitis pigmentosa received this treatment and noticed an improvement in his vision.


Got it. How does the Stargardt treatment work?

Nanoscope’s MCO-010 gene therapy reprograms healthy retina cells to make them photosensitive. It uses proprietary vector and promoter technology to deliver the MCO genes into retina bipolar cells, enabling vision in different color environments.

The therapy involves a single intravitreal injection in a medical office setting. (via)


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