Nanotechnology Gene Therapy Shown to Prevent Retinal Degeneration

In a revolutionary study, researchers at Tufts University this week published study results showing that nanotechnology can be used to deliver vision-saving gene therapy to the eyes. The goal was to show that a nanoparticle could be used to deliver a protein (Glial Cell Line-Derived Neurotrophic Factor (GDNF)) that protects eyes from diseases of the retina, such as age-related macular degeneration and retinitis pigmentosa. Gene therapy is a favored potential treatment for retinal degeneration, but the previous delivery method, using a virus to deliver genetic material, had several drawbacks. According to the senior author in this study, Rajendra Kumar-Singh, PhD, “While viruses are very efficient carriers, they can prompt immune responses that may lead to inflammation, cancer, or even death. Non-viral methods offer a safer alternative, but until now, efficiency has been a significant barrier.”

In the study, some mice eyes were injected with the GDNF-carrying nanoparticle, while others were injected with just the nanoparticle, and still others with just a buffer solution. Then all mice were subjected to blue light that stimulates retinal degeneration. Overall, the mice injected with the GDNF-carrying nanoparticle saw a 3.9-7.7-fold reduction in retinal damage. When the eyesight of the mice was measured, it was shown that 7 days after treatment the GDNF-nanoparticle-injected mice had 27-39% better eyesight than mice in the control group. Even at 14 days after treatment, the GDNF-nanoparticle-injected mice had outer nuclear layers of the retina that were 23.6-39.3% thicker than controls. However, by 14 days the functional improvement in vision had disappeared.

Although the results were very temporary, this research shows that there is significant potential for nanotechnology to develop an effective carrier to deliver gene therapy, and that this therapy can lead to new treatments for retinal diseases. According to Dr. Kumar-Singh, “The next step in this research is to prolong this protection by adding elements to the DNA that permit its retention in the cell.”

Age-related macular degeneration is the leading cause of blindness among Americans over age 65, but there is currently no treatment capable of reversing this disease. Even a short-term rescue of vision is a positive step in protecting or restoring the vision of older Americans.

You can learn how to recognize the early signs of macular degeneration, but the most important thing you can do to protect your vision is to get regular eye exams that will help you detect this disease in its early stages. Click here to find an eye doctor near you and schedule an appointment.