New gene therapy successfully restores part of the visual function of blind people for the first time

Today, the authoritative academic journal "Nature-Medical" published an exciting clinical trial result: a blind patient was diagnosed with retinitis pigmentosa 40 years ago.

Professor José-Alain Sahel, a well-known ophthalmologist from the University of Pittsburgh, is the first author and co-corresponding author of this study.

Retinitis pigmentosa is a neurodegenerative eye disease in which patients usually begin to have vision deterioration during childhood.

The research paper pointed out that "this report is the first case of neurodegenerative diseases that have been partially restored after optogenetic therapy.

In the past ten years, Professor Sahel and Professor Roska have led pioneering work using optogenetics to help visually impaired patients restore visual function.

The scientists used the harmless adeno-associated virus (AAV) as a vector to send the genetic instructions encoding the light-sensitive protein ChrimsonR into one eye of the patient, with the goal of transforming the ganglion cells in the fovea of ​​the retina.

Therefore, the patient needs to wear a special goggles.

▲Special goggles (picture source: reference [1])

The patient reported this time is the first participant in this clinical trial.

In the next few months, after a number of tests, the researchers confirmed that the visual function of this retinitis pigmentosa patient has been more and more obviously restored.

The Phase 1/2a clinical study of this optogenetic therapy is still in progress.

Note: The original text has been deleted

Reference

[1] José-Alain Sahel et al.