Stable and cost-effective production of GCASE-OPT using a low MOI lentiviral approach for enzyme replacement therapy in gaucher disease: development of L20_HEF1A_GBA-OPT-R534H, a stable human cell line - Suardi, Gabriela Aparecida Marcondes; Wiezel, Cláudia Emília Vieira; Tirapelli, Daniela Pretti da Cunha; Ramos, Ester Silveira; Almeida, Vania de; Siciliano, Velia; Gerson, Stanton; Weiss, Ron; Fontes, Aparecida Maria (International Congress of the Brazilian Genetics Society, 2024)