Preclinical Enzyme Replacement Therapy with a Recombinant β-Galactosidase-Lectin Fusion for CNS Delivery and Treatment of GM1-Gangliosidosis.
Weesner JA, Annunziata I, Yang T, Acosta W, Gomero E, Hu H, van de Vlekkert D, Ayala J, Qiu X, Fremuth LE, et al. Cells. 2022 Aug 19; 11(16). Epub 2022 Aug 19.