The Guard1 clinical trial - A first in-human, phase 1/2 study evaluating AVR-RD-02, a hematopoietic stem cell (HSC) gene therapy for Gaucher disease: Preliminary safety, pharmacodynamic and clinical efficacy results from the subjects observed for up to 24 months post-infusion - Adera, Mathews; Veselinovic, Milena; Treohan, Anita; Golipour, Azadeh; Ridha, Essra (Molecular genetics and metabolism, 2023)