Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice

McCarty, D M; DiRosario, J; Gulaid, K; Muenzer, J; Fu, H

doi:10.1038/gt.2009.85

Cited by 108Virus-based gene therapy researchLysosomal Storage Disorders ResearchRNA Interference and Gene Delivery

Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice

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McCarty, D M, DiRosario, J, Gulaid, K, Muenzer, J, Fu, H·2009·Gene Therapy

DOI 10.1038/gt.2009.85PMC PMC4289609

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Abstract

The presence of the blood-brain barrier (BBB) presents the most critical challenge in therapeutic development for mucopolysaccharidosis (MPS) IIIB, a lysosomal storage disease with severe neurological manifestation, because of alpha-N-acetylglucosaminidase (NaGlu) deficiency. Earlier, we showed a global central nervous system (CNS) transduction in mice by mannitol-facilitated entry of intravenous (IV)-delivered recombinant adeno-associated viral serotype 2 (rAAV2) vector. In this study, we optimized the approach and showed that the maximal transduction in the CNS occurred when the rAAV2 vector was IV injected at 8 min after mannitol administration, and was approximately 10-fold more efficient than IV delivery of the vector at 5 or 10 min after mannitol infusion. Using this optimal (8 min) regimen, a single IV infusion of rAAV2-CMV-hNaGlu vector is therapeutically beneficial for treating the CNS disease of MPS IIIB in adult mice, with significantly extended survival, improved behavioral performance, and reduction of brain lysosomal storage pathology. The therapeutic benefit correlated with maximal delivery to the CNS, but not peripheral tissues. This milestone data shows the first effective gene delivery across the BBB to treat CNS disease. The critical timing of vector delivery and mannitol infusion highlights the important contribution of this pretreatment to successful intervention, and the long history of safe use of mannitol in patients bodes well for its application in CNS gene therapy.

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Optimal transduction of AAV2 vectors in the brain was achieved by IV injection of AAV 8 min after mannitol administration; delivery less than 5 min or 10 min after mannitol infusion was ten times less efficient.
“a study showed that the optimal transduction of AAV2 vectors in the brain was achieved by IV injection of AAV 8 min after mannitol administration; delivery of the vector less than 5 min or 10 min after mannitol infusion was ten times less efficient.”
From Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside (2024) · cites this paper as [133]
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Mannitol-mediated BBB disruption allows broad CNS distribution of AAV vectors depending on mannitol dose, infusion route, and virus injection timing.
“Considering above, mannitol-mediated BBB disruption allows broad CNS distribution of AAV vectors depending on the mannitol dose, infusion route as well as virus injection timing [133].”
From Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside (2024) · cites this paper as [133]
The Results section reports diffuse CNS transgene expression after IV mannitol and rAAV2 delivery in mice, and shows that vector-injection timing strongly affected CNS entry. The Introduction and Discussion distinguish IV from intra-arterial delivery, but the paper does not establish how changing the mannitol dose or infusion route affects broad AAV distribution. Page numbers are unavailable in our copy. (LLM, conf=0.86)
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Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield
Zolotukhin, S., Byrne, B. J., Mason, E. G., Zolotukhin, I., Potter, M., Chesnut, K. A., Summerford, C., Samulski, R. J., & Muzyczka, N. (1999) · Gene Therapy · doi:10.1038/sj.gt.3300938 · Cited by 1,317 · see citing works
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
Kaplitt, M. G., Feigin, A., Tang, C., Fitzsimons, H. L., Mattis, P., Lawlor, P. A., Bland, R. J., Young, D., Strybing, K., Eidelberg, D., et al. (2007) · The Lancet · doi:10.1016/s0140-6736(07)60982-9 · Cited by 1,067 · see citing works
Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain
Kaplitt, M. G., Leone, P., Samulski, R. J., Xiao, X., Pfaff, D. W., O’Malley, K. L., & During, M. J. (1994) · Nature Genetics · doi:10.1038/ng1094-148 · Cited by 1,031 · see citing works
Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis
McCarty, D., Monahan, P., & Samulski, R. (2001) · Gene Therapy · doi:10.1038/sj.gt.3301514 · Cited by 735 · see citing works
Self-citation
Social interaction and sensorimotor gating abnormalities in mice lacking Dvl1
Lijam, N., Paylor, R., McDonald, M. P., Crawley, J. N., Deng, C.-X., Herrup, K., Stevens, K. E., Maccaferri, G., McBain, C. J., Sussman, D. J., et al. (1997) · Cell · doi:10.1016/s0092-8674(00)80354-2 · Cited by 470 · see citing works
Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector
McCown, T. J., Xiao, X., Li, J., Breese, G. R., & Samulski, R. J. (1996) · Brain Research · doi:10.1016/0006-8993(95)01488-8 · Cited by 450 · see citing works
Hurler and Hunter Syndromes: Mutual Correction of the Defect in Cultured Fibroblasts
Fratantoni, J. C., Hall, C. W., & Neufeld, E. F. (1968) · Science · doi:10.1126/science.162.3853.570 · Cited by 446 · see citing works
Drug delivery to the central nervous system: a review.
Misra, A. R., Ganesh, S. D., Shahiwala, A., & Shah, S. P. (2003) · PubMed · Cited by 434 · see citing works
Osmotic Opening of the Blood–Brain Barrier: Principles, Mechanism, and Therapeutic Applications
Rapoport, S. I. (2000) · Cellular and Molecular Neurobiology · doi:10.1023/a:1007049806660 · Cited by 428 · see citing works
Activated microglia in cortex of mouse models of mucopolysaccharidoses I and IIIB
Ohmi, K., Greenberg, D., Rajavel, K. S., Ryazantsev, S., Li, H. H., & Neufeld, E. F. (2003) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.252784899 · Cited by 413 · see citing works
Techniques in diagnostic human biochemical genetics: A laboratory manual
Strange, R. C. (1992) · FEBS Letters · doi:10.1016/0014-5793(92)81025-h · Cited by 413 · see citing works
Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA
Worgall, S., Sondhi, D., Hackett, N. R., Kosofsky, B., Kekatpure, M. V., Neyzi, N., Dyke, J. P., Ballon, D., Heier, L., Greenwald, B. M., et al. (2008) · Human Gene Therapy · doi:10.1089/hum.2008.022 · Cited by 402 · see citing works
Drug delivery to the central nervous system: A review
Tamargo, R. J., & Brem, H. (1992) · Neurosurgery Quarterly · Cited by 370 · see citing works
Quantitative correlation between the residual activity of β-hexosaminidase A and arylsulfatase A and the severity of the resulting lysosomal storage disease
Leinekugel, P., Michel, S., Conzelmann, E., & Sandhoff, K. (1992) · Human Genetics · doi:10.1007/bf00219337 · Cited by 327 · see citing works
Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)
Harmatz, P., Whitley, C. B., Waber, L. J., Pais, R. C., Steiner, R. D., Plecko, B., Kaplan, P. B., Simon, J., Butensky, E., & Hopwood, J. J. (2004) · The Journal of Pediatrics · doi:10.1016/j.jpeds.2004.03.018 · Cited by 270 · see citing works
The cryptic life style of adenoassociated virus
Berns, K. I., & Linden, R. M. (1995) · BioEssays · doi:10.1002/bies.950170310 · Cited by 268 · see citing works
Techniques in diagnostic human biochemical genetics : a laboratory manual
Hommes, F. A. (1991) · Wiley-Liss eBooks · Cited by 261 · see citing works
Molecular Trojan horses for blood–brain barrier drug delivery
Pardridge, W. M. (2006) · Current Opinion in Pharmacology · doi:10.1016/j.coph.2006.06.001 · Cited by 259 · see citing works
A randomized comparison of intra-arterial versus intravenous with or without intravenous 5-fluorouracil, for newly diagnosed patients with malignant glioma
Shapiro, W. R., Green, S. B., Burger, P. C., Selker, R. G., VanGilder, J. C., Robertson, J. T., Mealey, J., Ransohoff, J., & Mahaley, M. S. (1992) · Journal of neurosurgery · doi:10.3171/jns.1992.76.5.0772 · Cited by 243 · see citing works
Molecular Biology of the Blood–Brain Barrier
Pardridge, W. M. (2005) · Molecular Biotechnology · doi:10.1385/mb:30:1:057 · Cited by 220 · see citing works
Dimethylmethylene blue-based spectrophotometry of glycosaminoglycans in untreated urine: a rapid screening procedure for mucopolysaccharidoses.
de Jong, J. G. N., Wevers, R. A., Laarakkers, C. M., & Poorthuis, B. J. H. M. (1989) · Clinical Chemistry · doi:10.1093/clinchem/35.7.1472 · Cited by 219 · see citing works
Mouse model of Sanfilippo syndrome type B produced by targeted disruption of the gene encoding α- N -acetylglucosaminidase
Li, H. H., Yu, W.-H., Rozengurt, N., Zhao, H., Lyons, K. M., Anagnostaras, S., Fanselow, M. S., Suzuki, K., Vanier, M. T., & Neufeld, E. F. (1999) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.96.25.14505 · Cited by 210 · see citing works
Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease
Daly, T. M., Vogler, C. A., Levy, B. K., Haskins, M. E., & Sands, M. S. (1999) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.96.5.2296 · Cited by 204 · see citing works
Differential subcellular localization of cholesterol, gangliosides, and glycosaminoglycans in murine models of mucopolysaccharide storage disorders
McGlynn, R. H., Dobrenis, K., & Walkley, S. U. (2004) · The Journal of Comparative Neurology · doi:10.1002/cne.20355 · Cited by 200 · see citing works
Self-complementary adeno-associated virus serotype 2 vector: global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain
Fu, H., Muenzer, J., Samulski, R. J., Breese, G. R., Sifford, J., Zeng, X., & McCarty, D. M. (2003) · Molecular Therapy · doi:10.1016/j.ymthe.2003.08.021 · Cited by 181 · see citing works
Self-citation
Osmotic Therapy: Fact and Fiction
Diringer, M. N., & Zazulia, A. R. (2004) · Neurocritical Care · doi:10.1385/ncc:1:2:219 · Cited by 177 · see citing works
The Conjoint Importance of the Hippocampus and Anterior Thalamic Nuclei for Allocentric Spatial Learning: Evidence from a Disconnection Study in the Rat
Warburton, E. C., Baird, A. L., Morgan, A., Muir, J. L., & Aggleton, J. P. (2001) · Journal of Neuroscience · doi:10.1523/jneurosci.21-18-07323.2001 · Cited by 169 · see citing works
Drug Delivery Across the Blood-Brain Barrier
Chen, Y., Dalwadi, G., & Benson, H. A. E. (2004) · Current Drug Delivery · doi:10.2174/1567201043334542 · Cited by 163 · see citing works
Treatment of Lysosomal Storage Disorders
Rohrbach, M., & Clarke, J. T. R. (2007) · Drugs · doi:10.2165/00003495-200767180-00005 · Cited by 152 · see citing works
Enzyme replacement therapy in mucopolysaccharidosis type II (Hunter syndrome): a preliminary report
Muenzer, J., Lamsa, J. C., Garcia, A. R., DaCosta, J. M., García, J. L., & Treco, D. (2002) · Acta Paediatrica · doi:10.1111/j.1651-2227.2002.tb03118.x · Cited by 141 · see citing works
Self-citation
Improved Behavior and Neuropathology in the Mouse Model of Sanfilippo Type IIIB Disease after Adeno-Associated Virus-Mediated Gene Transfer in the Striatum
Cressant, A., Desmaris, N., Vérot, L., Bréjot, T., Froissart, R., Vanier, M.-T., Maire, I., & Heard, J. M. (2004) · Journal of Neuroscience · doi:10.1523/jneurosci.3558-04.2004 · Cited by 137 · see citing works
Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer
Daly, T., Ohlemiller, K. K., Roberts, M., Vogler, C., & Sands, M. S. (2001) · Gene Therapy · doi:10.1038/sj.gt.3301420 · Cited by 131 · see citing works
Targeted delivery across the blood–brain barrier
Gaillard, P. J., Visser, C. C., & de Boer, A. G. (2005) · Expert Opinion on Drug Delivery · doi:10.1517/17425247.2.2.299 · Cited by 130 · see citing works
Cytokines, neurotrophins, and oxidative stress in brain disease from mucopolysaccharidosis IIIB
Villani, G. R. D., Gargiulo, N., Faraonio, R., Castaldo, S., Reyero, E. G. Y., & Natale, P. D. (2006) · Journal of Neuroscience Research · doi:10.1002/jnr.21134 · Cited by 126 · see citing works
Neonatal Gene Therapy of MPS I Mice by Intravenous Injection of a Lentiviral Vector
Kobayashi, H., Carbonaro, D. A., Pepper, K. A., Petersen, D., Ge, S., Jackson, H., Shimada, H., Moats, R. A., & Kohn, D. B. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2004.10.006 · Cited by 125 · see citing works
Functional Correction of CNS Phenotypes in a Lysosomal Storage Disease Model Using Adeno-Associated Virus Type 4 Vectors
Liu, G., Martins, I., Wemmie, J. A., Chiorini, J. A., & Davidson, B. L. (2005) · Journal of Neuroscience · doi:10.1523/jneurosci.2936-05.2005 · Cited by 120 · see citing works
Cellular and Molecular Neurosurgery: Pathways from Concept to Reality-Part II: Vector Systems and Delivery Methodologies for Gene Therapy of the Central Nervous System
Zloković, B. V., & Apuzzo, M. L. J. (1997) · Neurosurgery · doi:10.1097/00006123-199704000-00028 · Cited by 116 · see citing works
Neurological Correction of Lysosomal Storage in a Mucopolysaccharidosis IIIB Mouse Model by Adeno-associated Virus-Mediated Gene Delivery
Fu, H., Samulski, R. J., McCown, T. J., Picornell, Y., Fletcher, D., & Muenzer, J. (2002) · Molecular Therapy · doi:10.1006/mthe.2001.0514 · Cited by 108 · see citing works
Self-citation
Gene Expression from Recombinant Viral Vectors in the Central Nervous System after Blood-Brain Barrier Disruption
Doran, S. E., Ren, X. D., Betz, A. L., Pagel, M. A., Neuwelt, E. A., Roessler, B. J., & Davidson, B. L. (1995) · Neurosurgery · doi:10.1227/00006123-199505000-00012 · Cited by 108 · see citing works
Blood-brain barrier disruption using mannitol: time course and electron microscopy studies
Cosolo, W., Martinello, P., Louis, W. J., & Christophidis, N. (1989) · American Journal of Physiology-Regulatory, Integrative and Comparative Physiology · doi:10.1152/ajpregu.1989.256.2.r443 · Cited by 102 · see citing works
Significantly increased lifespan and improved behavioral performances by rAAV gene delivery in adult mucopolysaccharidosis IIIB mice
Fu, H., Lu, K., Jennings, J. S., Moy, S. S., Perez, A., DiRosario, J., McCarty, D. M., & Muenzer, J. (2007) · Gene Therapy · doi:10.1038/sj.gt.3302961 · Cited by 100 · see citing works
Self-citation
Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human α-l-iduronidase gene
Hartung, S. D., Frandsen, J. L., Pan, D., Koniar, B. L., Graupman, P. C., Günther, R., Low, W. C., Whitley, C. B., & McIvor, R. S. (2004) · Molecular Therapy · doi:10.1016/j.ymthe.2004.03.011 · Cited by 99 · see citing works
Innate and adaptive immune activation in the brain of MPS IIIB mouse model
DiRosario, J., DiCaprio, E. L., Wang, C., Etter, J. P., Charrier, A., Jukkola, P. I., Auer, H., Best, V., Newsom, D. L., McCarty, D. M., et al. (2008) · Journal of Neuroscience Research · doi:10.1002/jnr.21912 · Cited by 98 · see citing works
Self-citation
Prevention of neuropathology in the mouse model of hurler syndrome
Desmaris, N., Vérot, L., Puech, J. P., Caillaud, C., Vanier, M. T., & Heard, J. M. (2004) · Annals of Neurology · doi:10.1002/ana.20150 · Cited by 97 · see citing works
Lysosomal accumulation of SCMAS (subunit c of mitochondrial ATP synthase) in neurons of the mouse model of mucopolysaccharidosis III B
Ryazantsev, S., Yu, W.-H., Zhao, H., Neufeld, E. F., & Ohmi, K. (2006) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2006.11.006 · Cited by 96 · see citing works
Enzyme Replacement with Recombinant β-Glucuronidase in Murine Mucopolysaccharidosis Type VII: Impact of Therapy during the First Six Weeks of Life on Subsequent Lysosomal Storage, Growth, and Survival
Vogler, C. A., Sands, M. S., Levy, B. K., Galvin, N. J., Birkenmeier, E. H., & Sly, W. S. (1996) · Pediatric Research · doi:10.1203/00006450-199606000-00019 · Cited by 93 · see citing works
Molecular Trojan horses for blood-brain barrier drug delivery.
Pardridge, W. M. (2006) · PubMed · Cited by 92 · see citing works
Percutaneous intravenous injection in neonatal mice.
Sands, M. S., & Barker, J. E. (1999) · PubMed · Cited by 91 · see citing works
Attenuated plasticity in neurons and astrocytes in the mouse model of Sanfilippo syndrome type B
Li, H. H., Zhao, H., Neufeld, E. F., Cai, Y., & Gómez‐Pinilla, F. (2002) · Journal of Neuroscience Research · doi:10.1002/jnr.10278 · Cited by 88 · see citing works
Advances in the Treatment of Mucopolysaccharidosis Type I
Muenzer, J., & Fisher, A. (2004) · New England Journal of Medicine · doi:10.1056/nejmp048084 · Cited by 87 · see citing works
Self-citation
Enzyme-Replacement Therapy from Birth Delays the Development of Behavior and Learning Problems in Mucopolysaccharidosis Type IIIA Mice
Gliddon, B. L., & Hopwood, J. J. (2004) · Pediatric Research · doi:10.1203/01.pdr.0000129661.40499.12 · Cited by 84 · see citing works
Neonatal retroviral vector-mediated hepatic gene therapy reduces bone, joint, and cartilage disease in mucopolysaccharidosis VII mice and dogs
Mango, R. L. (2004) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2004.01.015 · Cited by 77 · see citing works
Systemic Mannitol-Induced Hyperosmolality Amplifies rAAV2-Mediated Striatal Transduction to a Greater Extent Than Local Co-infusion
Bürger, C., Nguyen, F. N., Deng, J. V., & Mandel, R. J. (2004) · Molecular Therapy · doi:10.1016/j.ymthe.2004.08.031 · Cited by 70 · see citing works
Quantification and Characterization of Glycosaminoglycans at the Nanogram Level by a Combined Azure A-Silver Staining in Agarose Gels
van de Lest, C. H. A., Versteeg, E. M. M., Veerkamp, J. H., & VANKUPPEVELT, T. (1994) · Analytical Biochemistry · doi:10.1006/abio.1994.1425 · Cited by 67 · see citing works
Modification of the blood-brain barrier: increased concentration and fate of enzymes entering the brain.
Barranger, J. A., Рапопорт, С. И., Fredericks, W. R., Pentchev, P. G., MacDermot, K. D., Steusing, J. K., & Brady, R. O. (1979) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.76.1.481 · Cited by 66 · see citing works
Purification and Characterization of Recombinant Human α-N-Acetylglucosaminidase Secreted by Chinese Hamster Ovary Cells
Zhao, K., & Neufeld, E. F. (2000) · Protein Expression and Purification · doi:10.1006/prep.2000.1230 · Cited by 62 · see citing works
Synergistic Effect of Cold Mannitol and Na+/Ca2+ Exchange Blocker on Blood–Brain Barrier Opening
Ikeda, M., Bhattacharjee, A. K., Kondoh, T., Nagashima, T., & Tamaki, N. (2002) · Biochemical and Biophysical Research Communications · doi:10.1006/bbrc.2002.6495 · Cited by 62 · see citing works
Enzyme replacement therapy for the mucopolysaccharide storage disorders
Kakkis, E. (2002) · Expert Opinion on Investigational Drugs · doi:10.1517/13543784.11.5.675 · Cited by 60 · see citing works
Pathogenic mechanisms in lysosomal disease: a reappraisal of the role of the lysosome
Walkley, S. U. (2007) · Acta Paediatrica · doi:10.1111/j.1651-2227.2007.00202.x · Cited by 48 · see citing works
Brain-directed gene therapy for lysosomal storage disease: Going well beyond the blood– brain barrier
Sly, W. S., & Vogler, C. A. (2002) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.102175599 · Cited by 48 · see citing works
Selective neurodegeneration in murine mucopolysaccharidosis VII is progressive and reversible
Heuer, G. G., Passini, M. A., Jiang, K., Parente, M. K., Lee, V. M. ‐., Trojanowski, J. Q., & Wolfe, J. H. N. (2002) · Annals of Neurology · doi:10.1002/ana.10373 · Cited by 43 · see citing works
Effect of blood-brain barrier disruption on intact and fragmented monoclonal antibody localization in intracerebral lung carcinoma xenografts.
Neuwelt, E. A., Barnett, P. A., Hellström, K. E., Hellström, I., McCormick, C. I., & Ramsey, F. L. (1994) · PubMed · Cited by 43 · see citing works
Expression and Characterization of Human Recombinant and α-N-Actylglucosaminidase
Weber, B., Hopwood, J. J., & Yogalingam, G. (2001) · Protein Expression and Purification · doi:10.1006/prep.2000.1361 · Cited by 37 · see citing works
Simulation of Hematopoiesis: Implications for the Gene Therapy of Lysosomal Enzyme Disorders
Abkowitz, J. L., Persik, M. T., Catlin, S. N., & Guttorp, P. M. (2009) · Acta Haematologica · doi:10.1159/000203880 · Cited by 9 · see citing works
Current health behaviors and readiness to pursue life-style changes among men and women diagnosed with early stage prostate and breast carcinomas
Demark-Wahnefried, W., Peterson, B., McBride, C., Lipkus, I., & Clipp, E. (2000) · Cancer · doi:10.1002/(sici)1097-0142(20000201)88:3

Flagged first, then by citations

Research works printed but not linked: 2

[1]Neufeld EF Muenzer J The mucopolysaccharidoses Scriver CR Beaudet AL Sly WS Valle D The metabolic & molecular basis of inherited disease 8 McGraw-Hill New York, St Louis; San Francisco 2001 3421 3452
[15]Doolittle ND Miner ME Hall WA Siegal T Jerome E Osztie E Safety and efficacy of a multicenter study using intraarterial chemotherapy in conjunction with osmotic opening of the blood-brain barrier for the treatment of patients with malignant brain tumors Cancer 2000 88 637 647 10649259

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Pandit, R., Chen, L., & Götz, J. (2020) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2019.11.009 · Cited by 651 · see citing works
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Gray, S. J., Matagne, V., Bachaboina, L., Yadav, S., Ojeda, S. R., & Samulski, R. J. (2011) · Molecular Therapy · doi:10.1038/mt.2011.72 · Cited by 501 · see citing works
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Au, H. K. E., Isalan, M., & Mielcarek, M. (2022) · Frontiers in Medicine · doi:10.3389/fmed.2021.809118 · Cited by 284 · see citing works
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Current Strategies to Enhance Delivery of Drugs across the Blood–Brain Barrier
Teleanu, R. I., Preda, M. D., Niculescu, A.-G., Vladâcenco, O., Radu, C. I., Grumezescu, A. M., & Teleanu, D. M. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14050987 · Cited by 200 · see citing works
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Saraiva, J., Nobre, R. J., & Pereira de Almeida, L. (2016) · Journal of Controlled Release · doi:10.1016/j.jconrel.2016.09.011 · Cited by 201 · see citing works
Viral vectors for therapy of neurologic diseases
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Directed Evolution of a Novel Adeno-associated Virus (AAV) Vector That Crosses the Seizure-compromised Blood–Brain Barrier (BBB)
Gray, S. J., Blake, B. L., Criswell, H. E., Nicolson, S. C., Samulski, R. J., & McCown, T. J. (2010) · Molecular Therapy · doi:10.1038/mt.2009.292 · Cited by 185 · see citing works
Viral Strategies for Targeting the Central and Peripheral Nervous Systems
Bedbrook, C. N., Deverman, B. E., & Gradinaru, V. (2018) · Annual Review of Neuroscience · doi:10.1146/annurev-neuro-080317-062048 · Cited by 168 · see citing works
Biology of adeno-associated viral vectors in the central nervous system
Murlidharan, G., Samulski, R. J., & Asokan, A. (2014) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2014.00076 · Cited by 157 · see citing works
The advent of AAV9 expands applications for brain and spinal cord gene delivery
Dayton, R. D., Wang, D. B., & Klein, R. L. (2012) · Expert Opinion on Biological Therapy · doi:10.1517/14712598.2012.681463 · Cited by 144 · see citing works
Detection of amyloid plaques targeted by USPIO-Aβ1–42 in Alzheimer's disease transgenic mice using magnetic resonance microimaging
Yang, J., Zaim Wadghiri, Y., Minh Hoang, D., Tsui, W., Sun, Y., Chung, E., Li, Y., Wang, A., de Leon, M., & Wisniewski, T. (2011) · NeuroImage · doi:10.1016/j.neuroimage.2011.01.023 · Cited by 135 · see citing works
Detection of Amyloid Plaques Targeted by Bifunctional USPIO in Alzheimer’s Disease Transgenic Mice Using Magnetic Resonance Microimaging
Wadghiri, Y. Z., Li, J., Wang, J., Hoang, D. M., Sun, Y., Xu, H., Tsui, W., Li, Y., Boutajangout, A., Wang, A., et al. (2013) · PLoS ONE · doi:10.1371/journal.pone.0057097 · Cited by 114 · see citing works
Intra-arterial delivery of AAV vectors to the mouse brain after mannitol mediated blood brain barrier disruption
Foley, C. P., Rubin, D. G., Santillán, A., Sondhi, D., Dyke, J. P., Gobin, Y. P., Crystal, R. G., & Ballon, D. (2014) · Journal of Controlled Release · doi:10.1016/j.jconrel.2014.09.018 · Cited by 96 · see citing works
Current and potential therapeutic strategies for mucopolysaccharidoses
Noh, H., & Lee, J. I. (2014) · Journal of Clinical Pharmacy and Therapeutics · doi:10.1111/jcpt.12136 · Cited by 94 · see citing works
Crossing the blood-brain barrier with AAV vectors
Liu, D., Zhu, M., Zhang, Y., & Diao, Y. (2020) · Metabolic Brain Disease · doi:10.1007/s11011-020-00630-2 · Cited by 93 · see citing works
Feasibility and Safety of Systemic rAAV9-h NAGLU Delivery for Treating Mucopolysaccharidosis IIIB: Toxicology, Biodistribution, and Immunological Assessments in Primates
Murrey, D. A., Naughton, B. J., Duncan, F. J., Meadows, A. S., Bobo, T. A., Campbell, K., Bremer, W. G., Walker, C. M., Goodchild, L., Bolon, B. N., et al. (2014) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2013.208 · Cited by 88 · see citing works
Self-citation
Systemic gene delivery to the central nervous system using Adeno-associated virus
Bourdenx, M., Dutheil, N., Bézard, E., & Dehay, B. (2014) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2014.00050 · Cited by 79 · see citing works
Nanoparticles functionalized with Pep-1 as potential glioma targeting delivery system via interleukin 13 receptor α2-mediated endocytosis
Wang, B., Lv, L., Wang, Z., Zhao, Y., Wu, L. H., Fang, X., Xu, Q., & Xin, H. (2014) · Biomaterials · doi:10.1016/j.biomaterials.2014.03.068 · Cited by 78 · see citing works
Safe and stable noninvasive focal gene delivery to the mammalian brain following focused ultrasound
Stavarache, M. A., Petersen, N., Jurgens, E. M., Milstein, E. R., Rosenfeld, Z. B., Ballon, D. J., & Kaplitt, M. G. (2019) · Journal of Neurosurgery · doi:10.3171/2017.8.jns17790 · Cited by 76 · see citing works
Intrajugular Vein Delivery of AAV9-RNAi Prevents Neuropathological Changes and Weight Loss in Huntington's Disease Mice
Dufour, B. D., Smith, C. A., Clark, R. L., Walker, T. R., & McBride, J. L. (2014) · Molecular Therapy · doi:10.1038/mt.2013.289 · Cited by 71 · see citing works
Transvascular Delivery of Hydrophobically Modified siRNAs: Gene Silencing in the Rat Brain upon Disruption of the Blood-Brain Barrier
Godinho, B. M. D. C., Henninger, N., Bouley, J., Alterman, J. F., Haraszti, R. Á., Gilbert, J. W., Sapp, E., Coles, A. H., Biscans, A., Nikan, M., et al. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.08.005 · Cited by 62 · see citing works
Modulating the Blood–Brain Barrier: A Comprehensive Review
Whelan, R., Hargaden, G. C., & Knox, A. J. S. (2021) · Pharmaceutics · doi:10.3390/pharmaceutics13111980 · Cited by 63 · see citing works
Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development
Massaro, G., Geard, A. F., Liu, W., Coombe-Tennant, O., Waddington, S. N., Baruteau, J., Gissen, P., & Rahim, A. A. (2021) · Biomolecules · doi:10.3390/biom11040611 · Cited by 60 · see citing works
Expansive Gene Transfer in the Rat CNS Rapidly Produces Amyotrophic Lateral Sclerosis Relevant Sequelae When TDP-43 is Overexpressed
Wang, D. B., Dayton, R. D., Henning, P. P., Cain, C. D., Zhao, L. R., Schrott, L. M., Orchard, E. A., Knight, D. S., & Klein, R. L. (2010) · Molecular Therapy · doi:10.1038/mt.2010.191 · Cited by 59 · see citing works
Monocyte Trafficking, Engraftment, and Delivery of Nanoparticles and an Exogenous Gene into the Acutely Inflamed Brain Tissue – Evaluations on Monocyte-Based Delivery System for the Central Nervous System
Tong, H.-I., Kang, W., Davy, P. M. C., Shi, Y., Sun, S., Allsopp, R., & Lu, Y. (2016) · PLoS ONE · doi:10.1371/journal.pone.0154022 · Cited by 59 · see citing works
Crossing the blood–brain-barrier with viral vectors
Fu, H., & McCarty, D. M. (2016) · Current Opinion in Virology · doi:10.1016/j.coviro.2016.08.006 · Cited by 59 · see citing works
Self-citation
Biodegradable polymeric nanoparticles administered in the cerebrospinal fluid: Brain biodistribution, preferential internalization in microglia and implications for cell-selective drug release
Peviani, M., Palmiero, U. C., Cecere, F., Milazzo, R., Moscatelli, D., & Biffi, A. (2019) · Biomaterials · doi:10.1016/j.biomaterials.2019.04.012 · Cited by 58 · see citing works
Adeno-associated virus vector delivery to the brain: Technology advancements and clinical applications
Ye, D., Chukwu, C., Yang, Y., Hu, Z., & Chen, H. (2024) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2024.115363 · Cited by 58 · see citing works
Gene replacement ameliorates deficits in mouse and human models of cyclin-dependent kinase-like 5 disorder
Gao, Y., Irvine, E. E., Eleftheriadou, I., Naranjo, C. J., Hearn-Yeates, F., Bosch, L., Glegola, J. A., Murdoch, L., Czerniak, A., Meloni, I., et al. (2020) · Brain · doi:10.1093/brain/awaa028 · Cited by 54 · see citing works
Biochemical, histological and functional correction of mucopolysaccharidosis Type IIIB by intra-cerebrospinal fluid gene therapy
Ribera, A., Haurigot, V., Garcia, M., Marcó, S., Motas, S., Villacampa, P., Maggioni, L., León, X., Molas, M., Sánchez, V., et al. (2014) · Human Molecular Genetics · doi:10.1093/hmg/ddu727 · Cited by 53 · see citing works
Optimization strategies and advances in the research and development of AAV‐based gene therapy to deliver large transgenes
Kolesnik, V. V., Nurtdinov, R. F., Oloruntimehin, E. S., Karabelsky, A. V., & Malogolovkin, A. S. (2024) · Clinical and Translational Medicine · doi:10.1002/ctm2.1607 · Cited by 51 · see citing works
Defects in the Medial Entorhinal Cortex and Dentate Gyrus in the Mouse Model of Sanfilippo Syndrome Type B
Ohmi, K., Zhao, H., & Neufeld, E. F. (2011) · PLoS ONE · doi:10.1371/journal.pone.0027461 · Cited by 51 · see citing works
Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside
Xu, L., Yao, S., Ding, Y. E., Xie, M., Feng, D., Sha, P., Tan, L., Bei, F., & Yao, Y. (2024) · Journal of Translational Medicine · doi:10.1186/s12967-024-05661-2 · Cited by 48 · see citing works
β2 Agonists enhance the efficacy of simultaneous enzyme replacement therapy in murine Pompe disease
Koeberl, D. D., Li, S., Dai, J., Thurberg, B. L., Bali, D. S., & Kishnani, P. S. (2011) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2011.11.005 · Cited by 45 · see citing works
Selective stimulation of caveolae-mediated endocytosis by an osmotic polymannitol-based gene transporter
Park, T., Kang, B., Kim, Y.-K., Zhang, Q., Lee, W., Islam, M. A., Kang, S., Cho, M., Choi, Y., & Cho, C. (2012) · Biomaterials · doi:10.1016/j.biomaterials.2012.06.037 · Cited by 45 · see citing works
Intravenous rAAV2/9 injection for murine cochlear gene delivery
Shibata, S. B., Yoshimura, H., Ranum, P. T., Goodwin, A. T., & Smith, R. J. (2017) · Scientific Reports · doi:10.1038/s41598-017-09805-x · Cited by 44 · see citing works
AAV9-Retro mediates efficient transduction with axon terminal absorption and blood–brain barrier transportation
Lin, K., Zhong, X., Li, L., Ying, M., Yang, T., Zhang, Z., He, X., & Xu, F. (2020) · Molecular Brain · doi:10.1186/s13041-020-00679-1 · Cited by 45 · see citing works
Myeloperoxidase Nuclear Imaging for Epileptogenesis
Zhang, Y., Seeburg, D. P., Pulli, B., Wojtkiewicz, G. R., Buré, L., Atkinson, W. L., Schob, S., Iwamoto, Y., Ali, M. U., Zhang, W., et al. (2015) · Radiology · doi:10.1148/radiol.2015141922 · Cited by 42 · see citing works
Restoration of central nervous system α‐N‐acetylglucosaminidase activity and therapeutic benefits in mucopolysaccharidosis IIIB mice by a single intracisternal recombinant adeno‐associated viral type 2 vector delivery
Fu, H., DiRosario, J., Lu, K., Muenzer, J., & McCarty, D. M. (2010) · The Journal of Gene Medicine · doi:10.1002/jgm.1480 · Cited by 40 · see citing works
Self-citation
AAV-mediated human PEDF inhibits tumor growth and metastasis in murine colorectal peritoneal carcinomatosis model
Wu, Q. J., Gong, C. Y., Luo, S. T., Zhang, D. M., Zhang, S., Shi, H., Lu, L., Yan, H., He, S., Li, D. D., et al. (2012) · BMC Cancer · doi:10.1186/1471-2407-12-129 · Cited by 40 · see citing works
Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice
Heldermon, C. D., Qin, E. Y., Ohlemiller, K. K., Herzog, E. D., Brown, J. R. G., Vogler, C. A., Hou, W., Orrock, J. L., Crawford, B. E., & Sands, M. S. (2013) · Gene Therapy · doi:10.1038/gt.2013.14 · Cited by 39 · see citing works
Recent Advances in Gene Therapy for Hemophilia: Projecting the Perspectives
Chernyi, N., Gavrilova, D., Saruhanyan, M., Oloruntimehin, E. S., Karabelsky, A. V., Bezsonov, E. E., & Malogolovkin, A. S. (2024) · Biomolecules · doi:10.3390/biom14070854 · Cited by 42 · see citing works
Salinomycin-Loaded Iron Oxide Nanoparticles for Glioblastoma Therapy
Norouzi, M., Yathindranath, V., Thliveris, J. A., & Miller, D. W. (2020) · Nanomaterials · doi:10.3390/nano10030477 · Cited by 38 · see citing works
Insulin-like growth factor II peptide fusion enables uptake and lysosomal delivery of α-N-acetylglucosaminidase to mucopolysaccharidosis type IIIB fibroblasts
Kan, S., Troitskaya, L. A., Sinow, C., Haitz, K., Todd, A. K., Stefano, A. D., Le, S. Q., Dickson, P., & Tippin, B. L. (2013) · Biochemical Journal · doi:10.1042/bj20130845 · Cited by 36 · see citing works
Overexpression of α5β1 integrin and angiopoietin-1 co-operatively promote blood-brain barrier integrity and angiogenesis following ischemic stroke
Wang, L., Zhang, X., Liu, X., Feng, G., Fu, Y., Milner, R., & Li, L. (2019) · Experimental Neurology · doi:10.1016/j.expneurol.2019.113042 · Cited by 36 · see citing works
Mannitol-facilitated perfusion staining with 2,3,5-triphenyltetrazolium chloride (TTC) for detection of experimental cerebral infarction and biochemical analysis
Sun, Y., Yang, D., & Kuan, C.-Y. (2011) · Journal of Neuroscience Methods · doi:10.1016/j.jneumeth.2011.09.029 · Cited by 31 · see citing works
Identification of variable lymphocyte receptors that can target therapeutics to pathologically exposed brain extracellular matrix
Umlauf, B. J., Clark, P. A., Lajoie, J. M., Georgieva, J. V., Bremner, S. B., Herrin, B. R., Kuo, J. S., & Shusta, E. V. (2019) · Science Advances · doi:10.1126/sciadv.aau4245 · Cited by 30 · see citing works
A GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB
Meadows, A. S., Duncan, F. J., Camboni, M., Waligura, K., Montgomery, C. L., Zaraspe, K., Naughton, B. J., Bremer, W. G., Shilling, C. J., Walker, C. M., et al. (2015) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2015.132 · Cited by 28 · see citing works
Self-citation
Peripheral Nervous System Neuropathology and Progressive Sensory Impairments in a Mouse Model of Mucopolysaccharidosis IIIB
Fu, H., Bartz, J. D., Stephens, R. L., & McCarty, D. M. (2012) · PLoS ONE · doi:10.1371/journal.pone.0045992 · Cited by 27 · see citing works
Self-citation
Lessons learnt from animal models: pathophysiology of neuropathic lysosomal storage disorders
Hemsley, K. M., & Hopwood, J. J. (2010) · Journal of Inherited Metabolic Disease · doi:10.1007/s10545-010-9078-6 · Cited by 27 · see citing works
Broad Functional Correction of Molecular Impairments by Systemic Delivery of scAAVrh74-hSGSH Gene Delivery in MPS IIIA Mice
Duncan, F. J., Naughton, B. J., Zaraspe, K., Murrey, D. A., Meadows, A. S., Clark, K. R., Newsom, D. E., White, P., Fu, H., & McCarty, D. M. (2015) · Molecular Therapy · doi:10.1038/mt.2015.9 · Cited by 25 · see citing works
Self-citation
Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier
Critchley, B. J., Gaspar, H. B., & Benedetti, S. (2022) · Molecular Therapy · doi:10.1016/j.ymthe.2022.11.015 · Cited by 23 · see citing works
Intravenous mannitol does not increase blood–brain barrier permeability to inert dyes in the adult rat forebrain
Chen, K., Wei, V. C., Yen, L. F., Poon, K.-S., Liu, Y., Cheng, K., Chang, C.-S., & Lai, T. W. (2013) · Neuroreport · doi:10.1097/wnr.0b013e32835f8acb · Cited by 23 · see citing works
Effect of systemic high dose enzyme replacement therapy on the improvement of CNS defects in a mouse model of mucopolysaccharidosis type II
Cho, S. Y., Lee, J., Ko, A., Kwak, M. J., Kim, S., Sohn, Y. B., Park, S. W., & Jin, D. (2015) · Orphanet Journal of Rare Diseases · doi:10.1186/s13023-015-0356-0 · Cited by 21 · see citing works
Comparison of Endovascular and Intraventricular Gene Therapy With Adeno-Associated Virus–α-L-Iduronidase for Hurler Disease
Janson, C. G., Romanova, L. G., Leone, P., Nan, Z., Belur, L. R., McIvor, R. S., & Low, W. C. (2013) · Neurosurgery · doi:10.1227/neu.0000000000000157 · Cited by 21 · see citing works
Therapies for Neurological Disease in the Mucopolysaccharidoses
Anson, D. S., McIntyre, C., & Byers, S. (2011) · Current Gene Therapy · doi:10.2174/156652311794940791 · Cited by 21 · see citing works
Viral vector-mediated gene transfer for CNS disease
Snyder, B. R., Boulis, N. M., & Federici, T. (2010) · Expert Opinion on Biological Therapy · doi:10.1517/14712590903514074 · Cited by 20 · see citing works
Gene delivery with viral vectors for cerebrovascular diseases
Gan, Y. (2012) · Frontiers in Bioscience-Elite · doi:10.2741/e607 · Cited by 20 · see citing works
Differential distribution of heparan sulfate glycoforms and elevated expression of heparan sulfate biosynthetic enzyme genes in the brain of mucopolysaccharidosis IIIB mice
McCarty, D. M., DiRosario, J., Gulaid, K., Killedar, S., Oosterhof, A., Kuppevelt, T. H. V., Martin, P. T., & Fu, H. (2011) · Metabolic Brain Disease · doi:10.1007/s11011-010-9230-x · Cited by 20 · see citing works
Self-citation
Targeting Root Cause by Systemic scAAV9-hIDS Gene Delivery: Functional Correction and Reversal of Severe MPS II in Mice
Fu, H., Zaraspe, K., Murakami, N., Meadows, A. S., Pineda, R. J., McCarty, D. M., & Muenzer, J. (2018) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2018.07.005 · Cited by 20 · see citing works
Self-citation
Genetic strategies to study TDP-43 in rodents and to develop preclinical therapeutics for amyotrophic lateral sclerosis
Wang, D. B., Gitcho, M. A., Kraemer, B. C., & Klein, R. L. (2011) · European Journal of Neuroscience · doi:10.1111/j.1460-9568.2011.07803.x · Cited by 16 · see citing works
Selection of single domain anti-transferrin receptor antibodies for blood-brain barrier transcytosis using a neurotensin based assay and histological assessment of target engagement in a mouse model of Alzheimer’s related amyloid-beta pathology
Su, S., Esparza, T. J., & Brody, D. L. (2022) · PLoS ONE · doi:10.1371/journal.pone.0276107 · Cited by 15 · see citing works
Enzyme Replacement Therapy for Mucopolysaccharidosis IIID using Recombinant Human α-N-Acetylglucosamine-6-Sulfatase in Neonatal Mice
Wang, F., Moen, D. R., Sauni, C., Kan, S., Li, S., Le, S. Q., Lomenick, B., Zhang, X., Ekins, S., Singamsetty, S., et al. (2020) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.0c00831 · Cited by 16 · see citing works
High performance collection of cerebrospinal fluid in rats: Evaluation of erythropoietin penetration after osmotic opening of the blood–brain barrier
Ceaglio, N., Orozco, G., Etcheverrigaray, M., Mattio, M., Kratje, R. B., Perotti, N., & Oggero, M. (2013) · Journal of Neuroscience Methods · doi:10.1016/j.jneumeth.2013.07.009 · Cited by 15 · see citing works
Additive effect of low-frequency ultrasound and endothelial monocyte-activating polypeptide II on blood–tumor barrier in rats with brain glioma
Zhang, Z., Xue, Y., Liu, Y., & Shang, X. (2010) · Neuroscience Letters · doi:10.1016/j.neulet.2010.06.042 · Cited by 14 · see citing works
Behavioral deficits and cholinergic pathway abnormalities in male Sanfilippo B mice
Kan, S., Le, S. Q., Bui, Q. D., Benedict, B. C., Cushman, J. D., Sands, M. S., & Dickson, P. (2016) · Behavioural Brain Research · doi:10.1016/j.bbr.2016.06.023 · Cited by 14 · see citing works
Targeting Neurological Aspects of Mucopolysaccharidosis Type II: Enzyme Replacement Therapy and Beyond
Zanetti, A., & Tomanin, R. (2024) · BioDrugs · doi:10.1007/s40259-024-00675-0 · Cited by 12 · see citing works
Viral-Mediated Gene Replacement Therapy in the Developing Central Nervous System: Current Status and Future Directions
Uchitel, J., Kantor, B., Smith, E. C., & Mikati, M. A. (2020) · Pediatric Neurology · doi:10.1016/j.pediatrneurol.2020.04.010 · Cited by 11 · see citing works
Tissue blood volumes and weights in adult beagle dogs.
Cuddihy, R. G., Hall, R. P., HOBBS, C. H., Boecker, B. B., & Muggenburg, B. A. (1972) · OSTI OAI (U.S. Department of Energy Office of Scientific and Technical Information) · Cited by 10 · see citing works
Intracisternal delivery of NFκB-inducible scAAV2/9 reveals locoregional neuroinflammation induced by systemic kainic acid treatment
Bockstael, O., Tenenbaum, L., Dalkara, D., Melas, C., Witte, O. D., Levivier, M., & Chtarto, A. (2014) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2014.00092 · Cited by 8 · see citing works
Serum MIP-1 α level: a biomarker for the follow-up of lentiviral therapy in mucopolysaccharidosis IIIB mice
di Natale, P., di Domenico, C., & Napoli, D. D. (2010) · Journal of Inherited Metabolic Disease · doi:10.1007/s10545-010-9051-4 · Cited by 14 · see citing works
Perinatal Gene Delivery to the CNS
Rahim, A. A., Buckley, S. M. K., Chan, J. K. Y., Peebles, D., & Waddington, S. N. (2011) · Therapeutic Delivery · doi:10.4155/tde.11.16 · Cited by 7 · see citing works
Feasibility and Safety of Systemic rAAV9-hNAGLU Delivery for Treating MPS IIIB: Toxicology, Bio-distribution and Immunological Assessments in Primates
Murrey, D. A., Naughton, B. J., Duncan, F. J., Meadows, A. S., Ware, T., Campbell, K., Bremer, W. G., Walker, C. M., Goodchild, L., Bolon, B. N., et al. (2014) · Human Gene Therapy Clinical Development · doi:10.1089/hum.2013.208 · Cited by 6 · see citing works
Self-citation
Selective protection of murine cerebral Gi/o-proteins from inactivation by parenterally injected pertussis toxin
Vega, S. C., Leiss, V., Piekorz, R. P., Calaminus, C., Pexa, K., Vuozzo, M., Schmid, A. M., Devanathan, V., Kesenheimer, C., Pichler, B. J., et al. (2019) · Journal of Molecular Medicine · doi:10.1007/s00109-019-01854-1 · Cited by 6 · see citing works
Treatment of GM2 Gangliosidosis in Adult Sandhoff Mice Using an Intravenous Self-Complementary Hexosaminidase Vector
Walia, J. S., Osmon, K. J. L., Thompson, P., Woodley, E., Karumuthil‐Melethil, S., Heindel, C., Keimel, J. G., Kaemmerer, W. F., & Gray, S. J. (2021) · Current Gene Therapy · doi:10.2174/1566523221666210916153051 · Cited by 6 · see citing works
Carbogen inhalation opens the blood-brain barrier in rats without causing long-term metabolic or neurological deficit
Liao, K. H., Wei, V. C., Wang, H.-L., Chen, H.-Y., & Lai, T. W. (2019) · Brain Research · doi:10.1016/j.brainres.2019.146320 · Cited by 6 · see citing works
Present insights into the progress in gene therapy delivery systems for central nervous system diseases
Yang, Z., Jiang, X., & Gao, J. (2024) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2024.125069 · Cited by 5 · see citing works
Viral vectors for gene delivery to the central nervous system
Ingusci, S., Hall, B. L., Goins, W. F., Cohen, J. B., & Glorioso, J. C. (2024) · Handbook of clinical neurology · doi:10.1016/b978-0-323-90120-8.00001-0 · Cited by 5 · see citing works
Isoflurane attenuates carbogen-induced blood–brain barrier disruption independent of body temperature in mice and rats
Poon, K.-S., Pan, Y.-L., Liao, K. H., Wang, H., Chen, K., Chen, H.-Y., Liu, Y., & Lai, T. W. (2020) · Neuroreport · doi:10.1097/wnr.0000000000001390 · Cited by 4 · see citing works
Adeno-associated Virus-Mediated Gene Delivery Across the Blood-Brain Barrier
Yao, Y., & Bei, F. (2024) · Advances in neurobiology · doi:10.1007/978-3-031-69188-1_4 · Cited by 3 · see citing works
Viral Vectors for Treatment of Human Disease: Therapeutic and Manufacturing Challenges
Diallo, J. (2019) · Elsevier eBooks · doi:10.1016/b978-0-12-814470-1.00007-1 · Cited by 3 · see citing works
Functional and structural profiling of circulation via genetically encoded modular fluorescent probesPreprint
Vittani, M., Lee, A. B., Wang, X., Hiraoka, Y., Sasaki, D., Konno, A., Kusk, P., Kusk, P., Nagao, M., Courtin, J., et al. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2025.03.18.643859 · Cited by 3 · see citing works
Adeno-associated viruses in gene therapy for spinal muscular atrophies: trend or triumph?
Slobodkina, E. A., Akopyan, Z. A., & Макаревич, П. И. (2023) · Genes and Cells · doi:10.17816/gc516537 · Cited by 2 · see citing works
Applications and Developments of Gene Therapy Drug Delivery Systems for Neurological Disorders
Nguyen, N. H., Nguyen‐Thi, P., Nguyen, T. T., Bui, V. K. H., Nguyen, N. T. T., & Giau, V. V. (2024) · Advanced Therapeutics · doi:10.1002/adtp.202400269 · Cited by 2 · see citing works
A combination of systemic mannitol and mannitol modified polyester nanoparticles for caveolae-mediated gene delivery to the brain
Gilbert, B. R., Miglani, C., Karmakar, A., Pal, M., Chandran, V. C., Gupta, S., Pal, A., & Ganguli, M. (2025) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2025.102480 · Cited by 2 · see citing works
Gene Therapy for Parkinson’s Disease: Towards Non Invasive Approaches
Rodrı́guez, M., Jose, M., & Castro, R. (2011) · InTech eBooks · doi:10.5772/20631 · Cited by 1 · see citing works
CNS Gene Therapy Utilizing Intravenously Administered Viral Vectors
Bevan, A. K., & Kaspar, B. K. (2013) · AAPS advances in the pharmaceutical sciences series · doi:10.1007/978-1-4614-9105-7_17 · Cited by 1 · see citing works
Terapia génica no invasiva en enfermedades neurológicas
Armas, J. M. B., Castro‐Hernández, J., Díaz, M. R., & Fuentes, R. C. (2011) · Revista de Neurología · doi:10.33588/rn.5210.2010604 · Cited by 1 · see citing works
Gene Therapy of CNS Disorders Using Recombinant AAV Vectors
Murlidharan, G., Samulski, R. J., & Asokan, A. (2016) · Springer eBooks · doi:10.1007/978-1-4899-7654-3_2 · Cited by 1 · see citing works
Gene Therapy for Neurodevelopmental Disorders
McCarty, D. M., & Fu, H. (2014) · Encyclopedia of Life Sciences · doi:10.1002/9780470015902.a0025276 · Cited by 1 · see citing works
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A combination of systemic mannitol administration and mannitol-modified polyester nanoparticles facilitate gene delivery to the brain through caveolae-mediated endocytosisPreprint
Reshma, G., Miglani, C., Karmakar, A., Gupta, S., Pal, A., & Ganguli, M. (2024) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2024.08.29.610249 · Cited by 1 · see citing works
Therapeutics: Harnessing the Power of Molecular and Pharmacological Chaperones
Gross, D. S., Klein, R. L., & Witt, S. N. (2011) · Wiley eBooks · doi:10.1002/9781118063903.ch12
Molecular Therapy for Mucopolysaccharidosis Type I
Wolf, D. (2010) · University of Minnesota Digital Conservancy (University of Minnesota)
Biology of AAV Vectors in the Central Nervous System
Murlidharan, G. (2019) · Carolina Digital Repository (University of North Carolina at Chapel Hill) · doi:10.17615/cr64-be62
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Rising, A. C. (2010)

Significantly increased lifespan and improved behavioral performances by rAAV gene delivery in adult mucopolysaccharidosis IIIB mice
Fu, H., Lu, K., Jennings, J. S., Moy, S. S., Perez, A., DiRosario, J., McCarty, D. M., & Muenzer, J. (2007) · Gene Therapy · doi:10.1038/sj.gt.3302961 · Cited by 100 · see citing works
Self-citation
235. The Route of Vector Delivery Impacts the Therapeutic Effects of AAV-Mediated Gene Therapy in Adult Mucopolysaccharidosis IIIB Mice
(2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.06.238
Fu H: Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice. Gene Ther 2009
Mccarty, D. M., DiRosario, J., Gulaid, K., Muenzer, J., & Fu, H. (2016)
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Restoration of central nervous system α‐N‐acetylglucosaminidase activity and therapeutic benefits in mucopolysaccharidosis IIIB mice by a single intracisternal recombinant adeno‐associated viral type 2 vector delivery
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Self-citation
494. Optimizing the Mannitol-Facilitated CNS Entry of Peripherally-Delivered AAV2 Vector
Fu, H., DiRosario, J., & McCarty, D. M. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.08.564 · Cited by 1 · see citing works
Self-citation

Authors: McCarty, D. M., DiRosario, J., Gulaid, K., Muenzer, J., & Fu, H.
Affiliations: Nationwide Children's Hospital (US)
Abigail Wexner Research Institute (US)
University of North Carolina at Chapel Hill (US)
ORCID: Muenzer, J
Fu, H
Venue: Gene Therapy
Publisher: Springer Science and Business Media LLC
Published: 2009-07-09
Volume: 16
Issue: 11
Pages: 1340-1352
ISSN: 0969-7128 (ISSN-L)
1476-5462
DOI: 10.1038/gt.2009.85
PMC: PMC4289609
OpenAlex: W1978350886
Cited by: 105
References: 65
Citations by year: 2025: 4 · 2024: 9 · 2023: 2 · 2022: 4 · 2021: 4 · 2020: 7 · 2019: 10 · 2018: 6 · 2017: 1 · 2016: 7 · 2015: 4 · 2014: 10 · 2013: 7 · 2012: 6
FWCI: 5.23
Type: journal article
Language: en
Open access: green
europepmc.org
Licence: www.springer.com/tdm (tdm)
www.springer.com/tdm (vor)
Locations: Gene Therapy (publishedVersion)
PubMed (publishedVersion)
Europe PMC (PubMed Central) (acceptedVersion)
PubMed Central (acceptedVersion)
Carolina Digital Repository (University of North Carolina at Chapel Hill) (submittedVersion)
UNC Libraries
Funders: Sanfilippo Research Foundation
National Institute of Diabetes and Digestive and Kidney Diseases (R01DK063972)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Health Sciences › Medicine › Physiology › Lysosomal Storage Disorders Research
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Keywords: mannitol, blood-brain barrier, central nervous system gene delivery, mucopolysaccharidosis type IIIB, alpha-N-acetylglucosaminidase, lysosomal storage disease, behavioral performance, survival
MeSH terms: Acetylglucosaminidase, Dependovirus, Animals, Blood-Brain Barrier, Genetic Vectors, Mannitol, Mice, Inbred C57BL, Mucopolysaccharidosis III, Recombinant Proteins, Tissue Distribution, Transduction, Genetic, Survival Analysis, Mice, Knockout, Disease Progression, Maze Learning, Mice

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Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice

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