Gene Therapy Explained

This video explains how gene therapy, delivered by AAV (adeno-associated virus) vectors, works.
Many diseases have a genetic basis, which means that the disease is caused by mutated genes which provide incorrect instructions that cause the cell to produce abnormal protein, or disable production of the protein completely. In gene therapy, a functional copy of the gene is delivered into a patient’s own cells. Normal protein that is produced from a functional gene corrects the underlying cause of the disease, and may provide restorative disease-modifying effects.
Gene therapy uses engineered viruses to deliver genes into cells, such as AAV vectors, which are especially well suited for treating retinal diseases. Genetic defects in the eye can cause vision loss or blindness, and gene therapy can provide a long-term therapeutic benefit.