Laronidase-functionalized multiple-wall lipid-core nanocapsules: Promising formulation for a more effective treatment of mucopolysaccharidosis type i

Pharmaceutical Research

Volumn 32, Issue 3, 2015, Pages 941-954

  Mayer, Fabiana Quoos ^a,b,c   Adorne, Márcia Duarte ^b   Bender, Eduardo André ^b   De Carvalho, Talita Giacomet ^a,b   Dilda, Anna Cláudia ^a   Beck, Ruy Carlos Ruver ^b   Guterres, Sílvia Stanisçuaski ^b   Giugliani, Roberto ^a,b   Matte, Ursula ^a,b   Pohlmann, Adriana Raffin ^b  

^a HOSPITAL DE CLÍNICAS DE PORTO ALEGRE   (Brazil)

^b FEDERAL UNIVERSITY OF RIO GRANDE DO SUL   (Brazil)

^c Fundacao Estadual de Pesquisa Agropecuaria   (Brazil)

Author keywords

enzyme replacement therapy; laronidase; mucopolysaccharidosis I; multiple wall lipid core nanocapsules; surface functionalization

Indexed keywords

LARONIDASE; METAL COMPLEX; MULTIPLE WALL LIPID CORE NANOCAPSULE; NANOCAPSULE; SOMATOMEDIN B RECEPTOR; UNCLASSIFIED DRUG; LEVO IDURONIDASE; LIPID;

ACTIVE TRANSPORT; ANIMAL EXPERIMENT; ANIMAL TISSUE; AREA UNDER THE CURVE; ARTICLE; CONTROLLED STUDY; DRUG CYTOTOXICITY; DRUG FORMULATION; DRUG HALF LIFE; ENZYME ACTIVITY; ENZYME KINETICS; ENZYME METABOLISM; HUMAN; HUMAN CELL; HURLER SYNDROME; IN VITRO STUDY; IN VIVO STUDY; LASER DIFFRACTION; LIGHT SCATTERING; MOUSE; NONHUMAN; PRIORITY JOURNAL; ADVERSE EFFECTS; ANIMAL; CELL CULTURE; CELL SURVIVAL; CHEMISTRY; DRUG EFFECTS; ENZYME REPLACEMENT; ENZYMOLOGY; FIBROBLAST; GENETICS; INTRAVENOUS DRUG ADMINISTRATION; KNOCKOUT MOUSE; MEDICINAL CHEMISTRY; METABOLIC CLEARANCE RATE; METABOLISM; MUCOPOLYSACCHARIDOSIS I; NANOMEDICINE; PARTICLE SIZE; PATHOLOGY; PHARMACEUTICS; PROCEDURES; TISSUE DISTRIBUTION; TRANSPORT AT THE CELLULAR LEVEL;

ANIMALS; AREA UNDER CURVE; BIOLOGICAL TRANSPORT; CELL SURVIVAL; CELLS, CULTURED; CHEMISTRY, PHARMACEUTICAL; ENZYME REPLACEMENT THERAPY; FIBROBLASTS; IDURONIDASE; INJECTIONS, INTRAVENOUS; LIPIDS; METABOLIC CLEARANCE RATE; MICE, KNOCKOUT; MUCOPOLYSACCHARIDOSIS I; NANOCAPSULES; NANOMEDICINE; PARTICLE SIZE; TECHNOLOGY, PHARMACEUTICAL; TISSUE DISTRIBUTION;

EID: 84923059658     PISSN: 07248741     EISSN: 1573904X     Source Type: Journal    
DOI: 10.1007/s11095-014-1508-y     Document Type: Article

References (41)

1. Tan ML, Choong PF, Dass CR. Recent developments in liposomes, microparticles and nanoparticles for protein and peptide drug delivery. Peptides. 2010;31(1):184-93.
2. Carino GP, Jacob JS, Mathiowitz E. Nanosphere based oral insulin delivery. J Control Release. 2000;65(1-2):261-9.
3. Torchilin VP. Drug targeting. Eur J Pharm Sci. 2000;11 Suppl 2:S81-91.
4. Caruthers SD, Wickline SA, Lanza GM. Nanotechnological applications in medicine. Curr Opin Biotechnol. 2007;18(1):26-30.
5. Kreuter J. Nanoparticulate systems for brain delivery of drugs. Adv Drug Deliv Ver. 2001;47(1):65-81.
6. Di Marco M, Shamsuddin S, Razak KA, et al. Overview of the main methods used to combine proteins with nanosystems: absorption, bioconjugation, and encapsulation. Int J Nanomedicine. 2010;5:37-49.
7. Jonkheijn P, Weinrich D, Schröder H, Niemeyer CM, Waldmann H. Chemical strategies for generating protein biochips. Angew Chem Int Ed Engl. 2008;47(50):9618-47.
8. Rana S, Yeh YC, Rotello VM. Engineering the nanoparticle-protein interface: applications and possibilities. Curr Opin Chem Biol. 2010;14(6):828-34.
9. Jager E, Venturini CG, Poletto FS, et al. Sustained release from lipid-core nanocapsules by varying the core viscosity and the particle surface area. J Biomed Nanotechnol. 2009;5(1):130-40.
10. Venturini CG, Jäger E, Oliveira CP, et al. Formulation of lipid core nanocapsules. Colloids and Surf A: Physicochem Eng Aspects. 2011;375:200-8.
11. Jornada DS, Fiel LA, Bueno K, et al. Lipid-core nanocapsules: mechanism of self-assembly, control of size and loading capacity. Soft Matter. 2012;8:6646-55.
12. Fiel LA, Rebelo LM, Santiago TM, et al. Diverse deformation properties of polymeric nanocapsules and lipid-core nanocapsules. Soft Matter. 2011;7:7240-7.
13. Bernardi A, Frozza RL, Horn AP, et al. Protective effects of indomethacin-loaded nanocapsules against oxygen-glucose deprivation in organotypic hippocampal slice cultures: involvement of neuroinflammation. Neurochem Int. 2010;57(6):629-36.
14. Bernardi A, Zilberstein AC, Jager E, et al. Effects of indomethacin-loaded nanocapsules in experimental models of inflammation in rats. Br J Pharmacol. 2009;158(4):1104-11.
15. Bernardi A, Braganhol E, Jager E, et al. Indomethacin-loaded nanocapsules treatment reduces in vivo glioblastoma growth in a rat glioma model. Cancer Lett. 2009;281(1):53-63.
16. Ourique AF, Azoubel S, Ferreira CV, et al. Lipid-core nanocapsules as a nanomedicine for parenteral administration of tretinoin: development and in vitro antitumor activity on human myeloid leukaemia cells. J Biomed Nanotechnol. 2010;6(3):214-23.
17. Frozza RL, Bernardi A, Paese K, et al. Characterization of trans-resveratrol-loaded lipid-core nanocapsules and tissue distribution studies in rats. J Biomed Nanotechnol. 2010;6(6):3694-703.
18. Bender EA, Adorne MD, Colome LM, et al. Hemocompatibility of poly(epsilon-caprolactone) lipid-core nanocapsules stabilized with polysorbate 80-lecithin and uncoated or coated with chitosan. Int J Pharm. 2012;426(1-2):271-9.
19. Bender EA, Cavalcante MF, Adorne MD, et al. New strategy to surface functionalization of polymeric nanoparticles: one-pot synthesis of scFv anti-LDL(-)-functionalized nanocapsules. Res: Pharm; 2014. doi: 10.1007/s11095-014-1392-5.
20. Reynaud F, Tsapis N, Deyme M, et al. Spray-dried chitosan-metal microparticles for ciprofloxacin adsorption: Kinetic and equilibrium studies. Soft Matter. 2011;7:7304-12.
21. Neufeld EF, Muenzer J. The Mucopolysaccharidosis. In: Scriver CR, Beaudet AL, Sly WS, Valle D, editors. The Metabolic and Molecular Bases of Inherited Disease. New York: McGraw-Hill; 2001. p. 3421-52.
22. Giugliani R, Ferderhen A, Carvalho CG, Artigalas O. Enzyme replacement therapy for Mucopolysaccharidosis type I: Laronidase. Pediatr Health. 2010;4:133-45.
23. Souza MV, Krug BC, Picon P, Schwartz IVD. Medicamentos de alto custo para doenças raras no Brasil: o exemplo das doenças lisossômicas. Ciência e Saúde Coletiva. 2010;15(3):3443-554.
24. Giugliani R, Federhen A, Muñoz Rojas MV, et al. Enzyme replacement therapy for mucopolysaccharidoses I, II and VI: recommendations from a group of Brazilian F experts. Rev Assoc Med Bras. 2010;56(3):271-7.
25. Mengual O, Meunier G, Cayre I, Puech K, Snabre P. TURBISCAN MA 2000: multiple light scattering measurement for concentrated emulsion and suspension instability analysis. Talanta. 1999;50(2):445-56.
26. Mosmann T. Rapid colorimetric assay for cellular growth and survival: application to proliferation and cytotoxicity assays. J Immunol Methods. 1983;65:55-63.
27. Hopwood JJ, Muller V, Smithson A, Baggett N. A fluorometric assay using 4-methylumbelliferyl alpha-L-iduronide for the estimation of alpha-L-iduronidase activity and the detection of Hurler and Scheie syndromes. Clin Chim Acta. 1979;92(2):257-65.
28. Lowry OH, Rosebrough NJ, Farr AL, Randall RJ. Protein measurement with the Folin phenol reagent. J Biol Chem. 1951;193(1):265-75.
29. The European Commission, 2011/696/EU, Official Journal of the European Union, ISSN 1977-0677. 2011;54:L275.
30. Siqueira NM, Contri RV, Paese K, et al. Innovative sunscreen formulation based on benzophenone-3-loaded chitosan-coated polymeric nanocapsules. Skin Pharmacol Physiol. 2011;24(3):166-74.
31. Nelson DL, Cox MM. Enzimas. In: Nelson DL, Cox MM, editors. Princípios de bioquímica de Lehninger. São Paulo: Sarvier; 2006. p. 202-12.
32. Celia C, Trapasso E, Cosco D, Paolino D, Fresta M. Turbiscan lab expert analysis of the stability of ethosomes and ultradeformable liposomes containing a bilayer fluidizing agent. Colloids Surf B: Biointerfaces. 2009;72(1):155-60.
33. Ipe BI, Niemeyer CM. Nanohybrids composed of quantum dots and cytochrome P450 as photocatalysts. Angew Chem Int Ed Engl. 2006;45(3):504-7.
34. de Graaf AJ, Kooijman M, Hennink WE, Mastrobattista E. Nonnatural amino acids for site-specific protein conjugation. Bioconjug Chem. 2009;20(7):1281-95.
35. Hong R, Fischer NO, Verma A, et al. Control of protein structure and function through surface recognition by tailored nanoparticle scaffolds. J Am Chem Soc. 2004;126(3):739-43.
36. Abad JM, Mertens SF, Pita M, Fernandez VM, Schiffrin DJ. Functionalization of thioctic acid-capped gold nanoparticles for specific immobilization of histidine-tagged proteins. J Am Chem Soc. 2005;127(15):5689-94.
37. Rempel BP, Clarke LA, Withers SG. A homology model for human alpha-l-iduronidase: insights into human disease. Mol Genet Metab. 2005;85(1):28-37.
38. Goldberg M, Langer R, Jia X. Nanostructured materials for applications in drug delivery and tissue engineering. J Biomater Sci Polym Ed. 2007;18(3):241-68.
39. Giugliani R, Rojas VM, Martins AM, et al. A dose-optimization trial of laronidase (Aldurazyme) in patients with mucopolysaccharidosis I. Mol Genet Metab. 2009;96(1):13-9.
40. Garnacho C, Dhami R, Simone E, et al. Delivery of acid sphingomyelinase in normal and niemann-pick disease mice using intercellular adhesion molecule-1-targeted polymer nanocarriers. J Pharmacol Exp Ther. 2008;325(2):400-8.
41. Hsu J, Serrano D, Bhowmick T, et al. Enhanced endothelial delivery and biochemical effects of α-galactosidase by ICAM-1-targeted nanocarriers for Fabry disease. J Control Release. 2011;149:323-31.