EXPRESSION, BIOACTIVITY, AND SAFETY 1 YEAR AFTER ADENO-ASSOCIATED VIRAL VECTOR TYPE 2–MEDIATED DELIVERY OF NEURTURIN TO THE MONKEY NIGROSTRIATAL SYSTEM SUPPORT CERE-120 FOR PARKINSON'S DISEASE

Abstract

The present study provides evidence of long-term expression and bioactivity of NTN on the dopaminergic nigrostriatal system after bilateral stereotactic delivery of CERE-120 to the striatum. Furthermore, no evidence of any adverse effects for up to 1 year postadministration was observed. These findings reveal a wide safety margin for CERE-120 and collectively support the ongoing clinical testing of the efficacy and safety of CERE-120 in patients with Parkinson's disease.