Three patients received an innovative treatment to restore retinal cells. In a presentation at the annual meeting of the American Academy of Ophthalmology, researchers from Life Biosciences reported that this treatment appears to be safe, and two of the recipients have shown early signs of vision improvement.
Novel Cell Restoration Strategy
This study is one of the first studies approved by the U.S. Food and Drug Administration and utilizes a novel strategy for reprogramming cells to restore their normal function. Researchers used a modified version of the reprogramming strategy that won the Nobel Prize in 2012 for converting mature cells to an embryonic-like state. In this research, Japanese scientist Shinya Yamanaka used four genes to stimulate cellular age reversal.
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Therapeutic Trial in Glaucoma Patients
In the ongoing Phase 1 trial, scientists used three of the four factors to perform modified reprogramming of retinal ganglion cells in patients with a type of glaucoma. They returned these cells to a state before they lost their proper function. Retinal ganglion cells have long connections that link them from the retina to the optic nerve, which transmits visual signals to the brain. In some individuals, over time, fluids in the eye do not drain properly, and pressure on these cells causes the loss of their sensitive connections to the optic nerve.
To attempt to restore the function of these cells, in this trial, scientists injected specially designed proteins into the vitreous, the gel-like fluid at the back of the eye near the retina. This location is suitable for testing such treatment as it is protected from the body's immune defenses that might attack the newly produced proteins. These proteins then did their work to reprogram the damaged and aging cells to regain functionality.
This experimental treatment is based on work presented from the laboratory of David Sinclair, a genetics professor at Harvard Medical School and one of the founders of Life Biosciences. He believes that aging cells lose their ability to function properly because they accumulate confusing instructions that need to clarify which genes should be turned on or off. Sinclair believes that restoring the correct epigenetic information and removing epigenetic "noise" can help treat age-related diseases.
The main goal of this study is to demonstrate the safety of the treatment, but also two of the three participants have shown early signs of vision improvement.
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