A PHASE I SAFETY STUDY IN SUBJECTS WITH LEBER CONGENITAL AMAUROSIS (LCA) USING ADENO-ASSOCIATED VIRAL VECTOR TO DELIVER THE GENE FOR HUMAN RPE65 INTO THE RETINAL PIGMENT EPITHELIUM (RPE) [AAV.RPE65-101]: TREATMENT AND FOLLOW UP OF 3 ITALIAN PATIENTS

The aim of this project is to partcipate to a Phase I clinical trial for a rare genetic eye blinding disorder called Leber Congenital Amaurosis (LCA). Currently, there is no treatment or cure for LCA. This study is designed to establish the safety of this treatment for human patients and is based on solid scientific evidence of visual restoration in large animals. The Italian patients will be part of the total of 9 patients included in this study. The vector administration will take place in USA but the follow-up studies to determine treatment outcome will be performed at the Second University of Naples.