Scientists created light-sensitive cells using algae and microbes.
These were the results of a clinical trial focused on treating a patient suffering from retinitis pigmentosae — a collection of rare, inherited disorders of vision that can result in the breakdown and death of cells in the retina, according to. After administering the new gene therapy, participants in the clinical trial experienced substantial restoration of vision.
Typically, when light enters a human eye, it's captured via the photoreceptor cells, which then transmit an electrical signal to their neighbors to report significant features, like motion, to the brain via the optic nerve. Earlier studies have also found success restoring vision using experimental genetic therapy, but typically were for other disorders.
Our results set a new standard of what biological improvements are possible with antisense oligonucleotide therapy in LCA caused by CEP290 mutations. Importantly, we establish a comparator for currently-ongoing gene-editing therapies for the same disease, which will allow comparison of the relative merits of two different interventions," said co-lead author and Research Professor of Ophthalmology Artur V.