How Gene Therapy Treats Inherited Retinal Diseases

The U.S Food and Drug Administration in October 2017 revealed their plans to recommend gene therapy as a way to treat inherited retinal diseases. These diseases usually cause a gradual loss of sight that eventually leads to total blindness.



What are Inherited Retinal Diseases (IRDs)?



Inherited retinal diseases (IRDs) are rare eye problems that are caused by mutations that occur in the RPE65 gene. These types of gene mutations are hereditary and responsible for specific conditions that include Leber’s congenital amaurosis and some forms of retinitis pigmentosa. Gene therapy can be used to treat these eye disorders.



How gene therapy can treat inherited retinal diseases



People suffering from various types of inherited retinal diseases either have genes that don’t work, or are missing. Gene therapy can be used to treat these diseases by adding new genes to their cells to replace the missing or dysfunctional ones.



Usually, they use a specific prepared virus that introduces its own different genetic materials into the victim’s cells. If it is successful, the patient might be able to regain their vision.



Inherited Retinal Diseases are many. Here are some of the most common ones.



Retinitis Pigmentosa



This type of eye disorder is caused by the deterioration of the light-sensing retina cells. These are the cone and rod photoreceptors. Usually, the first symptom is early night blindness that is later followed by loss of peripheral vision as well as central vision. It eventually leads to total blindness.



Juvenile Macular Degeneration (JMD)



This disorder occurs when the macula degenerates. It is a childhood problem that develops when the victim is in their childhood years. The most popular type of JMD is known as the Stargardt disease.



Other types of inherited retinal diseases are Choroideremia, Cone-Rod Dystrophy, and Leber Congenital Amaurosis.



With the application of gene therapy, patients with different types of inherited retinal diseases can be able to regain their vision. This is done by the introduction new genes into their cells. This procedure is a good treatment option for these incurable eye disorders.


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