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References

Aryal, M., N. Vykhodtseva, Y. Z. Zhang, J. Park, and N. McDannold. 2013. Multiple treatments with liposomal doxorubicin and ultrasound–induced disruption of blood–tumor and blood–brain barriers improve outcomes in a rat glioma model. Journal of Controlled Release 169(1–2):103–111.

Boado, R. J., and W. M. Pardridge. 2017. Brain and organ uptake in the rhesus monkey in vivo of recombinant iduronidase compared to an insulin receptor antibody-iduronidase fusion protein. Molecular Pharmaceutics 14(4):1271–1277.

Boado, R. J., E. K. Hui, J. Z. Lu, and W. M. Pardridge. 2009. Agt–181: Expression in cho cells and pharmacokinetics, safety, and plasma iduronidase enzyme activity in rhesus monkeys. Journal of Biotechnology 144(2):135–141.

Boado, R. J., E. K. Hui, J. Z. Lu, R. K. Sumbria, and W. M. Pardridge. 2013. Blood–brain barrier molecular Trojan horse enables imaging of brain uptake of radioiodinated recombinant protein in the rhesus monkey. Bioconjugate Chemistry 24(10):1741–1749.

Brynskikh, A. M., Y. Zhao, R. L. Mosley, S. Li, M. D. Boska, N. L. Klyachko, A. V. Kabanov, H. E. Gendelman, and E. V. Batrakova. 2010. Macrophage delivery of therapeutic nanozymes in a murine model of Parkinson’s disease. Nanomedicine 5(3):379–396.

Chan, G. N., R. A. Evans, D. B. Banks, E. V. Mesev, D. S. Miller, and R. E. Cannon. 2017. Selective induction of P-glycoprotein at the CNS barriers during symptomatic stage of an ALS animal model. Neuroscience Letters 639:103–113.

Cho, C. F., J. M. Wolfe, C. M. Fadzen, D. Calligaris, K. Hornburg, E. A. Chiocca, N. Y. R. Agar, B. L. Pentelute, and S. E. Lawler. 2017. Blood–brain barrier spheroids as an in vitro screening platform for brain-penetrating agents. Nature Communications 8:15623.

Deane, R., S. Du Yan, R. K. Submamaryan, B. LaRue, S. Jovanovic, E. Hogg, D. Welch, L. Manness, C. Lin, J. Yu, H. Zhu, J. Ghiso, B. Frangione, A. Stern, A. M. Schmidt, D. L. Armstrong, B. Arnold, B. Liliensiek, P. Nawroth, F.

Hofman, M. Kindy, D. Stern, and B. Zlokovic. 2003. RAGE mediates amyloid-beta peptide transport across the blood–brain barrier and accumulation in brain. Nature Medicine 9(7):907–913.

Deverman, B. E., P. L. Pravdo, B. P. Simpson, S. R. Kumar, K. Y. Chan, A. Banerjee, W. L. Wu, B. Yang, N. Huber, S. P. Pasca, and V. Gradinaru. 2016. CRE-dependent selection yields AAV variants for widespread gene transfer to the adult brain. Nature Biotechnology 34(2):204–209.

Dou, H., C. B. Grotepas, J. M. McMillan, C. J. Destache, M. Chaubal, J. Werling, J. Kipp, B. Rabinow, and H. E. Gendelman. 2009. Macrophage delivery of nanoformulated antiretroviral drug to the brain in a murine model of neuroaids. Journal of Immunology 183(1):661–669.

Foust, K. D., E. Nurre, C. L. Montgomery, A. Hernandez, C. M. Chan, and B. K. Kaspar. 2009. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nature Biotechnology 27(1):59–65.

Griffin, J. H., B. V. Zlokovic, and L. O. Mosnier. 2015. Activated protein C: Biased for translation. Blood 125(19):2898–2907.

Griffin, J. H., J. A. Fernandez, P. D. Lyden, and B. V. Zlokovic. 2016. Activated protein C promotes neuroprotection: Mechanisms and translation to the clinic. Thrombosis Research 141(Suppl 2):S62–S64.

Haney, M. J., P. Suresh, Y. Zhao, G. D. Kanmogne, I. Kadiu, M. Sokolsky–Papkov, N. L. Klyachko, R. L. Mosley, A. V. Kabanov, H. E. Gendelman, and E. V. Batrakova. 2012. Blood–borne macrophage–neural cell interactions hitchhike on endosome networks for cell–based nanozyme brain delivery. Nanomedicine 7(6):815–833.

Haney, M. J., Y. Zhao, E. B. Harrison, V. Mahajan, S. Ahmed, Z. He, P. Suresh, S. D. Hingtgen, N. L. Klyachko, R. L. Mosley, H. E. Gendelman, A. V. Kabanov, and E. V. Batrakova. 2013. Specific transfection of inflamed brain by macrophages: A new therapeutic strategy for neurodegenerative diseases. PLoS ONE 8(4):e61852.

Hatherell, K., P. O. Couraud, I. A. Romero, B. Weksler, and G. J. Pilkington. 2011. Development of a three-dimensional, all-human in vitro model of the blood–brain barrier using mono-, co-, and tri-cultivation transwell models. Journal of Neuroscience Methods 199(2):223–229.

Ivanova, E., C. W. Yee, and B. T. Sagdullaev. 2016. Leveraging optogeneticbased neurovascular circuit characterization for repair. Neurotherapeutics 13(2):341–347.

Jones, A. R., and E. V. Shusta. 2007. Blood–brain barrier transport of therapeutics via receptor-mediation. Pharmaceutical Research 24(9):1759–1771.

Kaplitt, M. G., A. Feigin, C. Tang, H. L. Fitzsimons, P. Mattis, P. A. Lawlor, R. J. Bland, D. Young, K. Strybing, D. Eidelberg, and M. J. During. 2007. Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson’s disease: An open label, phase I trial. Lancet 369(9579):2097–2105.

Khot, A., J. Tibbitts, D. Rock, and D. K. Shah. 2017. Development of a translational physiologically based pharmacokinetic model for antibody-drug conjugates: A case study with T–DM1. AAPS Journal 19(6):1715–1734.

Lochhead, J. J., D. J. Wolak, M. E. Pizzo, and R. G. Thorne. 2015. Rapid transport within cerebral perivascular spaces underlies widespread tracer distribution in the brain after intranasal administration. Journal of Cerebral Blood Flow & Metabolism 35(3):371–381.

Lockman, P. R., R. K. Mittapalli, K. S. Taskar, V. Rudraraju, B. Gril, K. A. Bohn, C. E. Adkins, A. Roberts, H. R. Thorsheim, J. A. Gaasch, S. Huang, D. Palmieri, P. S. Steeg, and Q. R. Smith. 2010. Heterogeneous blood-tumor barrier permeability determines drug efficacy in experimental brain metastases of breast cancer. Clincal Cancer Research 16(23):5664–5678.

Marty, B., B. Larrat, M. Van Landeghem, C. Robic, P. Robert, M. Port, D. Le Bihan, M. Pernot, M. Tanter, F. Lethimonnier, and S. Meriaux. 2012. Dynamic study of blood–brain barrier closure after its disruption using ultrasound: A quantitative analysis. Journal of Cerebral Blood Flow & Metabolism 32(10):1948–1958.

McDannold, N., C. D. Arvanitis, N. Vykhodtseva, and M. S. Livingstone. 2012. Temporary disruption of the blood–brain barrier by use of ultrasound and microbubbles: Safety and efficacy evaluation in rhesus macaques. Cancer Research 72(14):3652–3663.

Muller, P. Y., M. Milton, P. Lloyd, J. Sims, and F. R. Brennan. 2009. The minimum anticipated biological effect level (MABEL) for selection of first human dose in clinical trials with monoclonal antibodies. Current Opinion in Biotechnology 20(6):722–729.

Neufield, E., and J. Muenzer. 2001. The mucopolysaccharidoses. In The metabolic and molecular bases of inherited disease, edited by C. Scriver, A. Beaudet, D. Valle, and W. Sly. New York: McGraw–Hill Companies. Pp. 3421–3452.

Pardridge, W. M. 2006. Molecular Trojan horses for blood–brain barrier drug delivery. Current Opinion in Pharmacology 6(5):494–500.

Park, J., Y. Zhang, N. Vykhodtseva, F. A. Jolesz, and N. J. McDannold. 2012. The kinetics of blood–brain barrier permeability and targeted doxorubicin delivery into brain induced by focused ultrasound. Journal of Controlled Release 162(1):134–142.

Park, J., M. Aryal, N. Vykhodtseva, Y. Z. Zhang, and N. McDannold. 2017. Evaluation of permeability, doxorubicin delivery, and drug retention in a rat brain tumor model after ultrasound-induced blood–tumor barrier disruption. Journal of Controlled Release 250:77–85.

Shapiro, E. G., I. Nestrasil, K. Rudser, K. Delaney, V. Kovac, A. Ahmed, B. Yund, P. J. Orchard, J. Eisengart, G. R. Niklason, J. Raiman, E. Mamak, M. J. Cowan, M. Bailey–Olson, P. Harmatz, S. P. Shankar, S. Cagle, N. Ali, R. D. Steiner, J. Wozniak, K. O. Lim, and C. B. Whitley. 2015. Neurocognition

across the spectrum of mucopolysaccharidosis type I: Age, severity, and treatment. Molecular Genetics and Metabolism 116(1–2):61–68.

Thorne, R. G., L. R. Hanson, T. M. Ross, D. Tung, and W. H. Frey, 2nd. 2008. Delivery of interferon–beta to the monkey nervous system following intranasal administration. Neuroscience 152(3):785–797.

Whiteman, D. A., and A. Kimura. 2017. Development of idursulfase therapy for mucopolysaccharidosis type II (Hunter syndrome): The past, the present and the future. Drug Design, Development and Therapy 11:2467–2480.

Winkler, E. A., Y. Nishida, A. P. Sagare, S. V. Rege, R. D. Bell, D. Perlmutter, J. D. Sengillo, S. Hillman, P. Kong, A. R. Nelson, J. S. Sullivan, Z. Zhao, H. J. Meiselman, R. B. Wendy, J. Soto, E. D. Abel, J. Makshanoff, E. Zuniga, D. C. De Vivo, and B. V. Zlokovic. 2015. GLUT1 reductions exacerbate Alzheimer’s disease vasculo–neuronal dysfunction and degeneration. Nature Neuroscience 18(4):521–530.

Yuan, D., Y. Zhao, W. A. Banks, K. M. Bullock, M. Haney, E. Batrakova, and A. V. Kabanov. 2017. Macrophage exosomes as natural nanocarriers for protein delivery to inflamed brain. Biomaterials 142:1–12.

Zhao, Y., M. J. Haney, N. L. Klyachko, S. Li, S. L. Booth, S. M. Higginbotham, J. Jones, M. C. Zimmerman, R. L. Mosley, A. V. Kabanov, H. E. Gendelman, and E. V. Batrakova. 2011. Polyelectrolyte complex optimization for macrophage delivery of redox enzyme nanoparticles. Nanomedicine 6(1):25–42.

Zhao, Y., M. J. Haney, R. Gupta, J. P. Bohnsack, Z. He, A. V. Kabanov, and E. V. Batrakova. 2014. GDNF–transfected macrophages produce potent neuroprotective effects in Parkinson’s disease mouse model. PLoS ONE 9(9):e106867.

Zhao, Z., A. R. Nelson, C. Betsholtz, and B. V. Zlokovic. 2015. Establishment and dysfunction of the blood–brain barrier. Cell 163(5):1064–1078.

Zlokovic, B. V. 2011. Neurovascular pathways to neurodegeneration in Alzheimer’s disease and other disorders. Nature Review Neuroscience 12(12):723–738.