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35 items
Eye is shown connected to visual processing regions of the brain via the optic nerve. Enlargement of retina shows photorecetors, biopolar cells and retinal ganglion cells.

Scientists discover gene therapy provides neuroprotection to prevent glaucoma vision loss

July 22, 2021

A form of gene therapy protects optic nerve cells and preserves vision in mouse models of glaucoma, according to research supported by NIH’s National Eye Institute. The findings suggest a way forward for developing neuroprotective therapies for glaucoma.

Gene therapy shows promise in treating rare eye disease in mice

April 13, 2021

A gene therapy protects eye cells in mice with a rare disorder that causes vision loss, especially when used in combination with other gene therapies, shows a study published in eLife.
Image shows cone cells, labeled in red.

Turning back the clock on a severe vision disorder

March 31, 2021

Scientists at the University of Pennsylvania School of Veterinary Medicine report findings of a gene therapy to treat a severe form of Leber congenital amaurosis, caused by mutations in the NPHP5 gene.

A new vision for AAV-delivered gene therapies

February 10, 2021

A broadly applicable AAV genome-coupled immunomodulation strategy helps cloak the AAV virus from unwanted immune responses, and offers important insights into ocular inflammation.

Researchers use patients’ cells to test gene therapy for rare eye disease

January 28, 2021

Scientists at the National Eye Institute (NEI) have developed a promising gene therapy strategy for a rare disease that causes severe vision loss in childhood.
Samarendra Mohanty and Subrata Batabyal

Scientists use gene therapy and a novel light-sensing protein to restore vision in mice

October 22, 2020

A newly developed light-sensing protein called the MCO1 opsin restores vision in blind mice when attached to retina bipolar cells using gene therapy.

UCI-led study reveals significant restoration of retinal and visual function following gene therapy

October 19, 2020

New generation CRISPR technology lays foundation for therapeutics to treat a wide range of inherited ocular diseases
David Gamm in the laboratory

UW researchers devise approach to treat rare, incurable form of blindness

July 30, 2020

Scientists at the University of Wisconsin‒Madison have published a proof-of-concept method to correct an inherited form of macular degeneration that causes blindness, and that is currently untreatable.
Multicolor image of retina in cross-section

Scientists Use Nanoparticle-Delivered Gene Therapy to Inhibit Blinding Eye Disease in Rodents

July 7, 2020

In experiments in rats and mice, two Johns Hopkins scientists report the successful use of nanoparticles to deliver gene therapy for blinding eye disease.
Field of green cells with red circle and red oval overlaid.

New gene therapy delivery system aims for precision

May 4, 2020

In a novel approach to gene therapy, scientists funded by the National Eye Institute (NEI) report using gold nanoparticles and light to target specific cells in mouse retina.