Gene Therapy 101

Close-up photograph of a scientist in a laboratory wearing laboratory gloves and taking notes

Gene therapy is the process of treating genetic disease by delivering therapeutic DNA to a patient’s cells. This therapeutic DNA contains a corrected copy of a disease-causing gene. At Odylia, we use adeno-associated viral (AAV) vectors to deliver the therapeutic DNA. AAV is a small, replication deficient virus that is not known to cause disease in humans. Using the AAV as a delivery vehicle, the new functional copy of the corrected gene is inserted into a patient’s cells, allowing their own body to produce proteins to treat or prevent genetic conditions. A single treatment can provide long lasting benefits, potentially leading to a better quality of life.

AAV gene therapy is well suited for retinal diseases. The eye is an ideal target for AAV gene therapy due to its small size, compartmentalization, accessibility, and immune privileged state. The small size and compartmentalization limits the amount of AAV needed to treat the diseased tissue, and the immune privileged state prevents the immune system from interacting with the virus. Over 300 genes that play a role in inherited retinal diseases have been identified, although fewer than 10 of these targets are currently in clinical development.

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