The Disease Leber Congenital Amaurosis (LCA), Caused by Loss-Of-Function Mutation

Multiple Choice

The disease Leber congenital amaurosis (LCA) , caused by loss-of-function mutation of a gene called RPE65, has been treated with gene therapy by injecting recombinant AAV vectors containing a normal copy of the RPE65 gene into their retinal epithelial cells.Patients receiving this therapy

A) may acquire detrimental mutations due to integration of the virus.
B) may need to repeat the treatment as the viral DNA is degraded.
C) will pass the mutation to their offspring.
D) now have the normal RPE65 gene in all cells.