A Phase I/IIa clinical trial is currently underway to ascertain the safety, tolerability and clinical efficacy of AAV-GBA1 vector PR001 (LY3884961) in GBA1-PD patients and children with type 2 GD (ClinicalTrials.gov Identifier: NCT04127578 and NCT04411654, respectively). This evidence concerns the gene GBA1 and Parkinson disease.