Discuss7 marksChallenge
A family has a history of an inherited eye disease called retinal dystrophy type Z, caused by a recessive mutation in the RQ gene. Affected individuals gradually lose vision from childhood and become blind by adulthood. A new gene therapy trial aims to deliver a functional copy of the RQ gene directly into the retinal cells of affected patients using an adeno-associated virus (AAV) vector. The treatment is somatic and requires a single injection into the eye.
Discuss the potential benefits and the social and ethical considerations of using gene therapy to treat this inherited eye disease. In your answer, refer to the specific context provided and to wider issues in genetic medicine.