About IRDs
What are Inherited Retinal Diseases (IRDs)?
Inherited retinal diseases (IRDs) are a group of eye conditions caused by genetic mutations that damage the retina’s light-sensing cells. The retina, a thin layer of cells at the back of the eye, captures light and sends signals to the brain to create images.
Because these diseases are inherited, they are passed from parents to children via faulty genes though some family members may carry these genes without ever showing symptoms.
AAV Gene Therapy in Inherited Retinal Diseases (IRDs)
Gene therapy has become one of the most promising areas of innovation in ophthalmology, particularly for inherited retinal diseases.
What is Usher Syndrome Type 1B (Usher 1B)?
There are three types of Usher syndrome and Usher Syndrome Type 1B is the most severe form affecting hearing, balance, and vision. It is caused by a fault in a gene called MYO7A, which provides instructions for a protein that plays an important role in both the inner ear and the retina.
What is Stargardt Disease?
Stargardt disease is the most common inherited form of macular degeneration seen in children and young adults. It affects approximately 1 in 8,000 to 10,000 people and is caused by changes in a gene called ABCA4, which is important for keeping the retina healthy.
Reference: Duncan JL, Pierce EA, Laster AM, et al. Inherited Retinal Degenerations: Current Landscape and Knowledge Gaps. Transl Vis Sci Technol. 2018;7(4):6. Published 2018 Jul 18. doi:10.1167/tvst.7.4.6