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A potential therapy for blinding retinal diseases

Researchers engineer novel protein that restores vision in blind mice via gene therapy

03-Nov-2020

Key points from article :

There is no cure for diseases that damage photoreceptors(special cells in the retina) and cause vision loss.

Researchers have been developing gene therapy approaches for such diseases.

They deliver genes that allow remaining retinal cells to produce a protein called opsin (helps sense light).

But opsin replacement strategies in development require intense light.

A team synthesised a gene that would produce a more photosensitive form of opsin that can activate at lower levels of light.

The MCO1 opsin gene was tested in blind mice with retinal degeneration.

The gene was delivered to bipolar cells, which are still functional even when the photoreceptors no longer work.

The therapy restored retinal function and vision to the blind mice - offers promise for a wide range of degenerative retinal diseases.

“A clinical study in people will help us understand how signalling through bipolar cells affects vision quality,” -Dr. Subrata Batabyal.

Research by NEI published in Nature Gene Therapy.

Mentioned in this article:

Click on resource name for more details.

National Eye Institute (NEI)

Performs and supports vision research and education programs

Nature Gene Therapy

Journal with latest research into genetic and cell-based technologies to treat disease

Subrata Batabyal

Senior Technical Officer at Nanoscope Technologies

Topics mentioned on this page:
Gene Therapy, Vision (augmentation)