TY - JOUR AU - Lampe, C. AU - Bellettato, C. M. AU - Karabul, N. AU - Scarpa, M. PY - 2013 DA - 2013// TI - Mucopolysaccharidoses and other lysosomal storage diseases JO - Rheum Dis Clin N Am VL - 39 UR - https://doi.org/10.1016/j.rdc.2013.03.004 DO - 10.1016/j.rdc.2013.03.004 ID - Lampe2013 ER - TY - JOUR AU - Rousselle, C. AU - Clair, P. AU - Lefauconnier, J. M. AU - Kaczorek, M. AU - Scherrmann, J. M. AU - Temsamani, J. PY - 2000 DA - 2000// TI - New advances in the transport of doxorubicin through the blood–brain barrier by a peptide vector-mediated strategy JO - Mol Pharmacol VL - 57 UR - https://doi.org/10.1124/mol.57.4.679 DO - 10.1124/mol.57.4.679 ID - Rousselle2000 ER - TY - JOUR AU - Tosi, G. AU - Bortot, B. AU - Ruozi, B. AU - Dolcetta, D. AU - Vandelli, M. A. AU - Forni, F. PY - 2013 DA - 2013// TI - Potential use of polymeric nanoparticles for drug delivery across the blood–brain barrier JO - Curr Med Chem VL - 20 UR - https://doi.org/10.2174/0929867311320170006 DO - 10.2174/0929867311320170006 ID - Tosi2013 ER - TY - JOUR AU - Sanchez-Covarrubias, L. AU - Slosky, L. M. AU - Thompson, B. J. AU - Davis, T. P. AU - Ronaldson, P. T. PY - 2014 DA - 2014// TI - Transporters at CNS barrier sites: obstacles or opportunities for drug delivery? JO - Curr Pharm Des VL - 20 UR - https://doi.org/10.2174/13816128113199990463 DO - 10.2174/13816128113199990463 ID - Sanchez-Covarrubias2014 ER - TY - JOUR AU - Begley, D. J. AU - Pontikis, C. C. AU - Scarpa, M. PY - 2008 DA - 2008// TI - Lysosomal storage diseases and the blood–brain barrier JO - Curr Pharm Des VL - 14 UR - https://doi.org/10.2174/138161208784705504 DO - 10.2174/138161208784705504 ID - Begley2008 ER - TY - JOUR AU - Abbott, N. J. AU - Patabendige, A. A. AU - Dolman, D. E. AU - Yusof, S. R. AU - Begley, D. J. PY - 2010 DA - 2010// TI - Structure and function of the blood–brain barrier JO - Neurobiol Dis VL - 37 UR - https://doi.org/10.1016/j.nbd.2009.07.030 DO - 10.1016/j.nbd.2009.07.030 ID - Abbott2010 ER - TY - JOUR AU - Begley, D. J. PY - 2004 DA - 2004// TI - ABC transporters and the blood–brain barrier JO - Curr Pharm Des VL - 10 UR - https://doi.org/10.2174/1381612043384844 DO - 10.2174/1381612043384844 ID - Begley2004 ER - TY - JOUR AU - Scarpa, M. AU - Bellettato, C. M. AU - Lampe, C. AU - Begley, D. J. PY - 2015 DA - 2015// TI - Neuronopathic lysosomal storage disorders: approaches to treat the central nervous system JO - Best Pract Res Clin Endocrinol Metab VL - 29 UR - https://doi.org/10.1016/j.beem.2014.12.001 DO - 10.1016/j.beem.2014.12.001 ID - Scarpa2015 ER - TY - JOUR AU - Preston, J. E. AU - Joan Abbott, N. AU - Begley, D. J. PY - 2014 DA - 2014// TI - Transcytosis of macromolecules at the blood–brain barrier JO - Adv Pharmacol VL - 71 UR - https://doi.org/10.1016/bs.apha.2014.06.001 DO - 10.1016/bs.apha.2014.06.001 ID - Preston2014 ER - TY - JOUR AU - Muldoon, L. L. AU - Alvarez, J. I. AU - Begley, D. J. AU - Boado, R. J. AU - Del Zoppo, G. J. AU - Doolittle, N. D. PY - 2013 DA - 2013// TI - Immunologic privilege in the central nervous system and the blood–brain barrier JO - J Cereb Blood Flow Metab VL - 33 UR - https://doi.org/10.1038/jcbfm.2012.153 DO - 10.1038/jcbfm.2012.153 ID - Muldoon2013 ER - TY - JOUR AU - Pardridge, W. M. PY - 2005 DA - 2005// TI - The blood–brain barrier: bottleneck in brain drug development JO - NeuroRx VL - 2 UR - https://doi.org/10.1602/neurorx.2.1.3 DO - 10.1602/neurorx.2.1.3 ID - Pardridge2005 ER - TY - JOUR AU - Dickson, P. I. AU - Chen, A. H. PY - 2011 DA - 2011// TI - Intrathecal enzyme replacement therapy for mucopolysaccharidosis I: translating success in animal models to patients JO - Curr Pharm Biotechnol VL - 12 UR - https://doi.org/10.2174/138920111795542642 DO - 10.2174/138920111795542642 ID - Dickson2011 ER - TY - JOUR AU - Kakkis, E. D. AU - Muenzer, J. AU - Tiller, G. E. AU - Waber, L. AU - Belmont, J. AU - Passage, M. PY - 2001 DA - 2001// TI - Enzyme-replacement therapy in mucopolysaccharidosis I JO - N Engl J Med VL - 344 UR - https://doi.org/10.1056/NEJM200101183440304 DO - 10.1056/NEJM200101183440304 ID - Kakkis2001 ER - TY - JOUR AU - King, B. AU - Setford, M. L. AU - Hassiotis, S. AU - Trim, P. J. AU - Duplock, S. AU - Tucker, J. N. PY - 2016 DA - 2016// TI - Low-dose, continual enzyme delivery ameliorates some aspects of established brain disease in a mouse model of a childhood-onset neurodegenerative disorder JO - Exp Neurol VL - 278 UR - https://doi.org/10.1016/j.expneurol.2015.11.013 DO - 10.1016/j.expneurol.2015.11.013 ID - King2016 ER - TY - JOUR AU - Jones, S. A. AU - Breen, C. AU - Heap, F. AU - Rust, S. AU - de Ruijter, J. AU - Tump, E. PY - 2016 DA - 2016// TI - A phase 1/2 study of intrathecal heparan-N-sulfatase in patients with mucopolysaccharidosis IIIA JO - Mol Genet Metab VL - 118 UR - https://doi.org/10.1016/j.ymgme.2016.05.006 DO - 10.1016/j.ymgme.2016.05.006 ID - Jones2016 ER - TY - JOUR AU - Sorrentino, N. C. AU - Fraldi, A. PY - 2016 DA - 2016// TI - Brain targeting in MPS-IIIA JO - Pediatr Endocrinol Rev VL - 13 ID - Sorrentino2016 ER - TY - JOUR AU - Muenzer, J. AU - Hendriksz, C. J. AU - Fan, Z. AU - Vijayaraghavan, S. AU - Perry, V. AU - Santra, S. PY - 2016 DA - 2016// TI - A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II JO - Genet Med VL - 18 UR - https://doi.org/10.1038/gim.2015.36 DO - 10.1038/gim.2015.36 ID - Muenzer2016 ER - TY - JOUR AU - Lu, C. -. T. AU - Zhao, Y. -. Z. AU - Wong, H. L. AU - Cai, J. AU - Peng, L. AU - Tian, X. -. Q. PY - 2014 DA - 2014// TI - Current approaches to enhance CNS delivery of drugs across the brain barriers JO - Intl J Nanomed VL - 9 UR - https://doi.org/10.2147/IJN.S61288 DO - 10.2147/IJN.S61288 ID - Lu2014 ER - TY - JOUR AU - Mikitsh, J. L. AU - Chacko, A. M. PY - 2014 DA - 2014// TI - Pathways for small molecule delivery to the central nervous system across the blood–brain barrier JO - Perspect Medicin Chem VL - 6 UR - https://doi.org/10.4137/PMC.S13384 DO - 10.4137/PMC.S13384 ID - Mikitsh2014 ER - TY - JOUR AU - Pardridge, W. M. PY - 2001 DA - 2001// TI - Brain drug targeting and gene technologies JO - Jpn J Pharmacol VL - 87 UR - https://doi.org/10.1254/jjp.87.97 DO - 10.1254/jjp.87.97 ID - Pardridge2001 ER - TY - JOUR AU - Pardridge, W. M. PY - 2015 DA - 2015// TI - Blood–brain barrier drug delivery of IgG fusion proteins with a transferrin receptor monoclonal antibody JO - Expert Opin Drug Deliv VL - 12 UR - https://doi.org/10.1517/17425247.2014.952627 DO - 10.1517/17425247.2014.952627 ID - Pardridge2015 ER - TY - JOUR AU - Pardridge, W. M. PY - 2002 DA - 2002// TI - Drug and gene targeting to the brain with molecular Trojan horses JO - Nat Rev Drug Discov VL - 1 UR - https://doi.org/10.1038/nrd725 DO - 10.1038/nrd725 ID - Pardridge2002 ER - TY - JOUR AU - Pardridge, W. M. AU - Boado, R. J. PY - 2012 DA - 2012// TI - Reengineering biopharmaceuticals for targeted delivery across the blood–brain barrier JO - Methods Enzymol VL - 503 UR - https://doi.org/10.1016/B978-0-12-396962-0.00011-2 DO - 10.1016/B978-0-12-396962-0.00011-2 ID - Pardridge2012 ER - TY - JOUR AU - Hersh, D. S. AU - Wadajkar, A. S. AU - Roberts, N. AU - Perez, J. G. AU - Connolly, N. P. AU - Frenkel, V. PY - 2016 DA - 2016// TI - Evolving drug delivery strategies to overcome the blood brain barrier JO - Curr Pharm Des VL - 22 UR - https://doi.org/10.2174/1381612822666151221150733 DO - 10.2174/1381612822666151221150733 ID - Hersh2016 ER - TY - CHAP AU - Grabrucker, A. AU - Chhabra, R. AU - Belletti, D. AU - Forni, F. AU - Vandelli, M. AU - Ruozi, B. ED - Fricker, G. ED - Ott, M. ED - Mahringer, A. PY - 2014 DA - 2014// TI - Nanoparticles as blood–brain barrier permeable cns targeted drug delivery systems BT - The blood brain barrier (BBB). Topics in medicinal chemistry PB - Springer Heidelberg CY - Berlin ID - Grabrucker2014 ER - TY - JOUR AU - Barua, S. AU - Mitragotri, S. PY - 2014 DA - 2014// TI - Challenges associated with penetration of nanoparticles across cell and tissue barriers: a review of current status and future prospects JO - Nano Today VL - 9 UR - https://doi.org/10.1016/j.nantod.2014.04.008 DO - 10.1016/j.nantod.2014.04.008 ID - Barua2014 ER - TY - JOUR AU - Mayer, F. Q. AU - Adorne, M. D. AU - Bender, E. A. AU - de Carvalho, T. G. AU - Dilda, A. C. AU - Beck, R. C. PY - 2015 DA - 2015// TI - Laronidase-functionalized multiple-wall lipid-core nanocapsules: promising formulation for a more effective treatment of mucopolysaccharidosis type I JO - Pharm Res VL - 32 UR - https://doi.org/10.1007/s11095-014-1508-y DO - 10.1007/s11095-014-1508-y ID - Mayer2015 ER - TY - JOUR AU - Mühlstein, A. AU - Gelperina, S. AU - Kreuter, J. PY - 2013 DA - 2013// TI - Development of nanoparticle-bound arylsulfatase B for enzyme replacement therapy of mucopolysaccharidosis VI JO - Pharmazie VL - 68 ID - Mühlstein2013 ER - TY - JOUR AU - Vilella, A. AU - Tosi, G. AU - Grabrucker, A. M. AU - Ruozi, B. AU - Belletti, D. AU - Vandelli, M. A. PY - 2014 DA - 2014// TI - Insight on the fate of CNS-targeted nanoparticles. Part I: Rab5-dependent cell-specific uptake and distribution JO - J Control Release VL - 174 UR - https://doi.org/10.1016/j.jconrel.2013.11.023 DO - 10.1016/j.jconrel.2013.11.023 ID - Vilella2014 ER - TY - JOUR AU - Vilella, A. AU - Ruozi, B. AU - Belletti, D. AU - Pederzoli, F. AU - Galliani, M. AU - Semeghini, V. PY - 2015 DA - 2015// TI - Endocytosis of nanomedicines: the case of glycopeptide engineered PLGA nanoparticles JO - Pharmaceutics VL - 7 UR - https://doi.org/10.3390/pharmaceutics7020074 DO - 10.3390/pharmaceutics7020074 ID - Vilella2015 ER - TY - JOUR AU - Salvalaio, M. AU - Rigon, L. AU - Belletti, D. AU - D'Avanzo, F. AU - Pederzoli, F. AU - Ruozi, B. PY - 2016 DA - 2016// TI - Targeted polymeric nanoparticles for brain delivery of high molecular weight molecules in lysosomal storage disorders JO - PLoS One VL - 11 UR - https://doi.org/10.1371/journal.pone.0156452 DO - 10.1371/journal.pone.0156452 ID - Salvalaio2016 ER - TY - JOUR AU - Tomanin, R. AU - Zanetti, A. AU - Zaccariotto, E. AU - D'Avanzo, F. AU - Bellettato, C. M. AU - Scarpa, M. PY - 2012 DA - 2012// TI - Gene therapy approaches for lysosomal storage disorders, a good model for the treatment of mendelian diseases JO - Acta Paediatr VL - 101 UR - https://doi.org/10.1111/j.1651-2227.2012.02674.x DO - 10.1111/j.1651-2227.2012.02674.x ID - Tomanin2012 ER - TY - JOUR AU - Wolf, D. A. AU - Banerjee, S. AU - Hackett, P. B. AU - Whitley, C. B. AU - McIvor, R. S. AU - Low, W. C. PY - 2015 DA - 2015// TI - Gene therapy for neurologic manifestations of mucopolysaccharidoses JO - Exp Opin Drug Deliv VL - 12 UR - https://doi.org/10.1517/17425247.2015.966682 DO - 10.1517/17425247.2015.966682 ID - Wolf2015 ER - TY - JOUR AU - Kamata, Y. AU - Tanabe, A. AU - Kanaji, A. AU - Kosuga, M. AU - Fukuhara, Y. AU - Li, X. K. PY - 2003 DA - 2003// TI - Long-term normalization in the central nervous system, ocular manifestations, and skeletal deformities by a single systemic adenovirus injection into neonatal mice with mucopolysaccharidosis VII JO - Gene Ther VL - 10 UR - https://doi.org/10.1038/sj.gt.3301869 DO - 10.1038/sj.gt.3301869 ID - Kamata2003 ER - TY - JOUR AU - Sands, M. S. AU - Haskins, M. E. PY - 2008 DA - 2008// TI - CNS-directed gene therapy for lysosomal storage diseases JO - Acta Paediatr Suppl VL - 97 UR - https://doi.org/10.1111/j.1651-2227.2008.00660.x DO - 10.1111/j.1651-2227.2008.00660.x ID - Sands2008 ER - TY - JOUR AU - Pan, D. PY - 2011 DA - 2011// TI - Cell- and gene-based therapeutic approaches for neurological deficits in mucopolysaccharidoses JO - Curr Pharm Biotechnol VL - 12 UR - https://doi.org/10.2174/138920111795542679 DO - 10.2174/138920111795542679 ID - Pan2011 ER - TY - JOUR AU - Hocquemiller, M. AU - Giersch, L. AU - Audrain, M. AU - Parker, S. AU - Cartier, N. PY - 2016 DA - 2016// TI - Adeno-associated virus-based gene therapy for CNS diseases JO - Hum Gene Ther VL - 27 UR - https://doi.org/10.1089/hum.2016.087 DO - 10.1089/hum.2016.087 ID - Hocquemiller2016 ER - TY - JOUR AU - Mathison, S. AU - Nagilla, R. AU - Kompella, U. B. PY - 1998 DA - 1998// TI - Nasal route for direct delivery of solutes to the central nervous system: fact or fiction? JO - J Drug Target VL - 5 UR - https://doi.org/10.3109/10611869808997870 DO - 10.3109/10611869808997870 ID - Mathison1998 ER - TY - JOUR AU - Belur, L. R. AU - Temme, A. AU - Podetz-Pedersen, K. M. AU - Riedl, M. AU - Vulchanova, L. AU - Robinson, N. PY - 2017 DA - 2017// TI - Intranasal adeno-associated virus mediated gene delivery and expression of human iduronidase in the central nervous system: a noninvasive and effective approach for prevention of neurologic disease in mucopolysaccharidosis type I JO - Hum Gene Ther VL - 28 UR - https://doi.org/10.1089/hum.2017.187 DO - 10.1089/hum.2017.187 ID - Belur2017 ER -