Friday, March 14, 2025

Restoring vision with optogenetics

Good news!

"Optogenetic therapies, which aim to restore lost vision by introducing light-sensitive proteins into damaged retinal neurons, are moving quickly toward the clinic. Several companies are developing viruses that carry genes encoding the proteins into cells that have been damaged by diseases such as retinitis pigmentosa and macular degeneration. Other approaches pair gene therapies with goggles that deliver precise flashes of light to activate the protein more effectively."

"... results of the trial are not yet published, but in January the US Food and Drug Administration (FDA) designated the treatment as a ‘regenerative medicine advanced therapy’, which should accelerate its development. It marks a significant step in the 20-year pursuit of optogenetic therapies for RP. Clinical trials have previously had mixed success, with several companies exiting the space.

Now, many academic and industry researchers are investigating variations on the idea ... Differences include which light-sensitive protein is introduced; which cells this protein is placed in; and whether to use auxiliary technologies such as goggles that deliver precise flashes of light to better activate the protein. ...

RP [retinitis pigmentosa] is a collection of genetic conditions caused by hundreds of mutations in more than 80 genes, and preventing carriers from going blind could require tailored approaches for each form. Optogenetic therapy, however, is designed to restore lost vision, and therefore could help anyone with RP. ..."

Nature Briefing: Translational Research

Restoring vision with optogenetics [seems to be an overview article] "Trials of therapies that enhance the light-sensitivity of cells in the retina are returning surprisingly positive results in people with retinitis pigmentosa."

No comments: