Biomarkers for gene therapy clinical trials of lysosomal storage disorders

Autor: Rossi, Alessandro, Malvagia, Sabrina, la Marca, Giancarlo, Parenti, Giancarlo, Brunetti-Pierri, Nicola
Zdroj: In Molecular Therapy 4 September 2024 32(9):2930-2938
Databáze: ScienceDirect