Age-related Macular Degeneration or AMD is an eye disease that primarily affects older adults and leads to blindness in severe cases. In the United States, AMD is the leading cause of blindness.

There are two types of AMD, wet and dry, and wet AMD is the more severe form. Currently, there is no cure for AMD, but different types of treatment are available. Recently, gene therapy has gained traction as a possible treatment option.

In a recent experiment, a macular degeneration patient underwent gene therapy as a part of a clinical trial.

What is Macular Degeneration?

AMD affects the macula, which is the part of the retina responsible for central vision. As a result of this disease, a person’s central vision becomes blurry, distorted, or completely lost.

It affects more than 10 million people in the United States alone, and the number is expected to double by 2050. AMD can either be wet or dry. Wet AMD is characterized by blood vessels leaking under the retina, leading to the death of retinal cells.

In contrast, dry AMD occurs due to the accumulation of waste products called drusen under the retina, leading to the death of retinal cells.

The Current Treatment Options for AMD

The treatment of AMD typically depends on the type of disease present. The current standard of care for wet AMD is anti-VEGF therapy. Anti-VEGF drugs help slow the growth of blood vessels, preventing leakage from the retina.

Some examples of these drugs include Lucentis and Avastin. On the other hand, no standard treatment exists for dry AMD. However, oral supplements of vitamins and minerals like zinc and copper have been suggested to slow down the progression of dry AMD.

What is Gene Therapy?

Gene therapy is a technique that involves altering or modifying a person’s genetic code to treat or cure diseases. The primary aim of gene therapy is to correct the faulty genes or replace them with healthy genes.

In the context of AMD, gene therapy is used to deliver healthy genes to the retina and replace the mutated genes that caused the disease. Gene therapy has been used to treat various genetic diseases, including Leber Congenital Amaurosis, a rare form of blindness.

How Does Gene Therapy Work?

Gene therapy is a multi-step process that involves the following:.

Isolating the healthy genes for the affected cell or tissue.
Integrating these genes into a virus or vector that targets the affected cells or tissues.
Administering these vectors through an injection under the retina or through the blood vessels.
Allowing the healthy genes to express themselves in the affected cells, thereby correcting the genetic defect causing the disease.

: The Gene Therapy Experiment

In a recent experiment published in the New England Journal of Medicine, a patient with Macular Degeneration received gene therapy. The clinical trial was conducted jointly by the University of Oxford and Cambridge in the UK.

The 86-year-old woman had advanced wet AMD that would not respond to traditional treatments. Doctors injected a harmless virus containing a newly designed DNA into her retina through a small surgical incision.

The viral vector carried the code to produce the protein sFLT01, which is designed to stop blood vessel growth in the retina and rescue the remaining cells.

: The Results of the Gene Therapy Experiment

The results of the experiment suggest that gene therapy is safe for treating patients with advanced wet AMD.

The genetic therapy not only rescued the remaining cells in the retina, but it also allowed the patient to read 24-28 letters more on an eye chart, as well as improve eyesight. However, the improvements were temporary, and the patient required additional injections to maintain the benefits.

The Future of Gene Therapy for AMD

The clinical trial conducted to treat macular degeneration was a significant step in the direction of developing a cure for AMD. The use of gene therapy for AMD can become the definitive treatment in the future, possibly providing long-term benefits.

The experimental results suggest that gene therapy can halt the progression of the disease, rescue the remaining cells, and improve eyesight in the affected individuals. However, more clinical trials are required to determine its safety and efficacy in a wide range of AMD patients.

: The Conclusion

The macular degeneration patient’s experience with gene therapy can be seen as a ray of hope, especially for those suffering from AMD. Gene therapy could revolutionize the way macular degeneration is treated in the future.

The experiment demonstrates the therapeutic potential of gene therapy as a viable treatment option and provides patients with hope for a cure to their incurable condition. Leveraging the advancements in gene therapy and developing tailored solutions for each AMD patient can become the future of AMD treatment.

However, it is vital to continue research, development, and trials towards making gene therapy more accessible and affordable so it can reach thousands of suffering patients worldwide.