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

Recently, the authoritative academic journal "Nature-Medical" published an exciting clinical trial result: a blind patient was diagnosed with retinitis pigmentosa 40 years ago.
After years of darkness, he accepted a method called Gene therapy based on optogenetic therapy has recently successfully restored some vision.
After treatment, this patient has been able to identify, count, locate, and touch different objects.

Professor José-Alain Sahel, a well-known ophthalmologist from the University of Pittsburgh, is the first author and co-corresponding author of this study.
He said: "This is an important milestone!"

Retinitis pigmentosa is a neurodegenerative eye disease in which patients usually begin to have vision deterioration during childhood.
The loss of photoreceptor cells on the retina can eventually lead to complete blindness.
At present, scientists have found that more than 70 different mutant genes can cause this blindness.
However, apart from gene replacement therapy for one of the mutant genes, which is effective in the early stage of the disease, there is currently no approved therapy for the treatment of retinitis pigments.
Transgender patients.

The research paper pointed out that "this report is the first case of neurodegenerative diseases that have been partially restored after optogenetic therapy.
" This breakthrough presents a bright future to millions of patients around the world who have fallen into darkness due to retinal diseases.

In the past ten years, Professor Sahel and Professor Roska have led pioneering work using optogenetics to help visually impaired patients restore visual function.
This technology involves genetically modifying specific cells of the retina to make the cells express light-sensitive proteins to respond to light pulses, and then use the light pulses to control these cells.

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.
When the amber light shines on these cells, it activates the light-sensitive protein, allowing the ganglion cells to send signals along the optic nerve to the brain.

Therefore, the patient needs to wear a special goggles.
This kind of goggles comes with a dedicated camera that can take images and convert the visual images into light pulses, which are then projected onto the retina in real time, thereby activating the transformed nerve cells.

The patient reported this time is the first participant in this clinical trial.
He is 58 years old.
Before receiving optogenetic therapy, he couldn't see anything in one eye.
A few months after the injection of gene therapy, as the ganglion cells stably express light-sensitive proteins, the brain gradually adapts to the modified visual system.

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.
Not only can you see the zebra crossing, but can even count the number of white lines.
Dao! Seeing the notebooks, cups, bottled liquids and other objects in front of him, he can reach out and touch them with confidence.

The Phase 1/2a clinical study of this optogenetic therapy is still in progress.
( Academic latitude and longitude )

Note: The original text has been deleted

Reference

[1] José-Alain Sahel et al.
, (2021) Partial recovery of visual function in a blind patient after optogenetic therapy.
Nature DOI: 10.
1038/s41591-021-01351-4

[2] With engineered proteins, scientists use optogenetics for the first time to help a blind patient see again.
Retrieved May 24, 2021 from for-first-time-to-help-blind-patient-see/