Preclinical enzyme replacement therapy with a recombinant β-galactosidase-lectin fusion for CNS delivery and treatment of GM1-gangliosidosis

Autor: Weesner, Jason A., van de Vlekkert, Diantha, Acosta, Walter L., Yang, Tianhong, Gomero, Elida M., Hu, Huimin, Radin, David, Cramer, Carole L., d'Azzo, Alessandra
Zdroj: In Molecular Genetics and Metabolism February 2023 138(2)
Databáze: ScienceDirect
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