Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients.

ABSTRACT:

Objective

In an effort to account for deficiencies in axonal transport that limit the effectiveness of neurotrophic factors, this study tested the safety and feasibility, in moderately advanced Parkinson disease (PD), of bilaterally administering the gene therapy vector AAV2-neurturin (CERE-120) to the putamen plus substantia nigra (SN, a relatively small structure deep within the midbrain, in proximity to critical neuronal and vascular structures).

Methods

After planning and minimizing risks of stereotactically targeting the SN, an open-label, dose-escalation safety trial was initiated in 6 subjects with PD who received bilateral stereotactic injections of CERE-120 into the SN and putamen.

Results

Two-year safety data for all subjects suggest the procedures were well

SUBMITTER: Bartus RT 

PROVIDER: S-EPMC3716474 | biostudies-literature | 2013 Apr

REPOSITORIES: biostudies-literature