Efficacy and safety of gene therapy with AAV4 in childhood blindness due to rpe65 mutations
Acta Ophthalmologica, 08/20/2012
Meur GL et al. – Subretinal injection of a serotype 4 AAV vector for gene therapy treatment of rpe65–/– retinal degeneration is safe. Visual field improvement found in the adult patients want us to treat 3 childrens scheduled in the third cohort.