Magnetic resonance imaging-guided phase 1 trial of putaminal AADC gene therapy for Parkinson's disease.

ABSTRACT:

Objective

To understand the safety, putaminal coverage, and enzyme expression of adeno-associated viral vector serotype-2 encoding the complementary DNA for the enzyme, aromatic L-amino acid decarboxylase (VY-AADC01), delivered using novel intraoperative monitoring to optimize delivery.

Methods

Fifteen subjects (three cohorts of 5) with moderately advanced Parkinson's disease and medically refractory motor fluctuations received VY-AADC01 bilaterally coadministered with gadoteridol to the putamen using intraoperative magnetic resonance imaging (MRI) guidance to visualize the anatomic spread of the infusate and calculate coverage. Cohort 1 received 8.3 × 10¹¹ vg/ml and ≤450 μl per putamen (total dose, ≤7.5 × 10¹¹ vg); cohort 2 received the same concentrat

SUBMITTER: Christine CW 

PROVIDER: S-EPMC6593762 | biostudies-literature | 2019 May

REPOSITORIES: biostudies-literature