Sheila Nirenberg | “A new and unexpectedly effective treatment for restoring sight”

Theoretical and computational neuroscience \ Manfred Eigen lecture theatre

An unexpected finding during a clinical trial for a disease that causes blindness opened the door to a new type of treatment. Briefly, the trial was focused on an optogenetic gene therapy for a disease called Retinitis Pigmentosa, but in the process of assessing patients, a new mechanism-of-action emerged. This allowed the treatment to be much more effective than predicted and provided a new strategy for approaching retinal degenerative diseases and possibly other neurodegenerative diseases as well.

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