Effective encapsulation of therapeutic recombinant enzyme into polymeric nanoparticles as a potential vehicle for lysosomal disease treatment

CRIVARO, ANDREA N.; CECI, ROMINA; BOZTEPE, TUGCE; CISNEROS, JOSÉ SEBASTIÁN; CHAIN, CECILIA YAMIL; HUCK-IRIART, CRISTIÁN; LAMAS, DIEGO G.; ISLAN, GERMÁN A.; ROZENFELD, PAULA

INTERNATIONAL JOURNAL OF BIOLOGICAL MACROMOLECULES

ELSEVIER SCIENCE BV

Lugar: Amsterdam; Año: 2025 vol. 285

0141-8130

Gaucher Disease (GD) is a genetic disorder with defective activity of the lysosomal enzyme glucocerebrosidase. Velaglucerasa alfa is a recombinant glucocerebrosidase used for enzyme replacement therapy (ERT) of GD. Due to its limited stability and bioavailability, the use of nanosized systems carrying Velaglucerase alfa is proposed as a novel strategy to improve ERT for GD. Highly stable and low-dispersed Velaglucerase-loaded Eudragit nanoparticles (NPs) (NPs: Vela); 150–160 nm mean size, polydispersity index