The treatment of biochemical genetic diseases: From substrate reduction to nucleic acid therapies

Autor: Vos, E. Naomi, Demirbas, Didem, Mangel, Matthew, Rubio-Gozalbo, M. Estela, Levy, Harvey L., Berry, Gerard T.
Zdroj: In Molecular Genetics and Metabolism November 2023 140(3)
Databáze: ScienceDirect