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Engineering a lysosomal enzyme with a derivative of receptor-binding domain of apoE enables delivery across the blood–brain barrier
Open accessAbstract
To realize the potential of large molecular weight substances to treat neurological disorders, novel approaches are required to surmount the blood–brain barrier (BBB). We investigated whether fusion of a receptor-binding peptide from apolipoprotein E (apoE) with a potentially therapeutic protein can bind to LDL receptors on the BBB and be transcytosed into the CNS. A lysosomal enzyme, α- L -iduronidase (IDUA), was used for biological and therapeutic evaluation in a mouse model of mucopolysaccharidosis (MPS) type I, one of the most common lysosomal storage disorders with CNS deficits. We identified two fusion candidates, IDUAe1 and IDUAe2, by in vitro screening, that exhibited desirable receptor-mediated binding, endocytosis, and transendothelial transport as well as appropriate lysosomal enzyme trafficking and biological function. Robust peripheral IDUAe1 or IDUAe2 generated by transient hepatic expression led to elevated enzyme levels in capillary-depleted, enzyme-deficient brain tissues and protein delivery into nonendothelium perivascular cells, neurons, and astrocytes within 2 d of treatment. Moreover, 5 mo after long-term delivery of moderate levels of IDUAe1 derived from maturing red blood cells, 2% to 3% of normal brain IDUA activities were obtained in MPS I mice, and IDUAe1 protein was detected in neurons and astrocytes throughout the brain. The therapeutic potential was demonstrated by normalization of brain glycosaminoglycan and β-hexosaminidase in MPS I mice 5 mo after moderate yet sustained delivery of IDUAe1. These findings provide a noninvasive and BBB-targeted procedure for the delivery of large-molecule therapeutic agents to treat neurological lysosomal storage disorders and potentially other diseases that involve the brain.
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“Some of those exchangeable apolipoproteins (ApoA1 and ApoE) can cross the blood-brain barrier (BBB) via receptor-mediated transcytosis18–21”
From Brain-targeted drug delivery by manipulating protein corona functions (2019) · cites this paper as [19]
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Cites
Neurovascular pathways to neurodegeneration in Alzheimer’s disease and other disorders
Zlokovic, B. V. (2011) · Nature Reviews Neuroscience · doi:10.1038/nrn3114 · Cited by 2,969
Pardridge, W. M. (2007) · Drug Discovery Today · doi:10.1016/j.drudis.2006.10.013 · Cited by 1,191
Apolipoprotein E and its receptors in Alzheimer's disease: pathways, pathogenesis and therapy
Bu, G. (2009) · Nature Reviews Neuroscience · doi:10.1038/nrn2620 · Cited by 1,168
Astroglial networks: a step further in neuroglial and gliovascular interactions
Giaume, C., Koulakoff, A., Roux, L., Holcman, D., & Rouach, N. (2010) · Nature Reviews Neuroscience · doi:10.1038/nrn2757 · Cited by 789
Cord-Blood Transplants from Unrelated Donors in Patients with Hurler's Syndrome
Staba, S. L., Escolar, M. L., Poe, M. D., Kim, Y., Martin, P., Szabolcs, P., Allison-Thacker, J., Wood, S. J., Wenger, D. A., Rubinstein, P., et al. (2004) · New England Journal of Medicine · doi:10.1056/nejmoa032613 · Cited by 475
Drug and gene targeting to the brain with molecular trojan horses
Pardridge, W. M. (2002) · Nature Reviews Drug Discovery · doi:10.1038/nrd725 · Cited by 439
Lipoprotein binding to canine hepatic membranes. Metabolically distinct apo-E and apo-B,E receptors.
Hui, D. Y., Innerarity, T. L., & Mahley, R. W. (1981) · Journal of Biological Chemistry · doi:10.1016/s0021-9258(19)69254-9 · Cited by 383
Self-citationOutcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome
Peters, C., Balthazor, M., Shapiro, E., King, R., Kollman, C., Hegland, J., Henslee-Downey, J. P., Trigg, M. E., Cowan, M. J., Sanders, J. E., et al. (1996) · Blood · doi:10.1182/blood.v87.11.4894.bloodjournal87114894 · Cited by 315
Kreuter, J., Hekmatara, T., Dreis, S., Vogel, T., Gelperina, S. E., & Langer, K. (2006) · Journal of Controlled Release · doi:10.1016/j.jconrel.2006.12.012 · Cited by 263
Targeted delivery of proteins across the blood–brain barrier
Spencer, B. J., & Verma, I. M. (2007) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0702170104 · Cited by 249
Research works printed but not linked: 2
- ES Neufeld, J Muenzer, The mucopolysaccharidoses. The Online Metabolic and Molecular Bases of Inherited Disease, eds D Valle, et al. (McGraw-Hill, New York, Part 16, Chap 136. (2007).
- 10.1111/j.1651-2227.2002.tb03115.x · DOI
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Kato, N., Yamada, S., Suzuki, R., Iida, Y., Matsumoto, M., Fumoto, S., Arima, H., Mukai, H., & Kawakami, S. (2023) · Drug Delivery · doi:10.1080/10717544.2023.2173333 · Cited by 33
Díaz‐Perlas, C., Sánchez‐Navarro, M., Oller‐Salvia, B., Moreno, M. R., Teixidò, M., & Giralt, E. (2016) · Biopolymers · doi:10.1002/bip.22928 · Cited by 33
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Emerging therapies for neuropathic lysosomal storage disorders
Kelly, J. M., Bradbury, A. M., Martin, D. R., & Byrne, M. E. (2016) · Progress in Neurobiology · doi:10.1016/j.pneurobio.2016.10.002 · Cited by 32
Pinkiewicz, M., Zaczyński, A., Walecki, J. M., & Zawadzki, M. (2025) · International Journal of Molecular Sciences · doi:10.3390/ijms26157050 · Cited by 36
Pan, X., Sands, S. A., Yue, Y., Zhang, K., LeVine, S. M., & Duan, D. (2019) · Human Gene Therapy · doi:10.1089/hum.2019.008 · Cited by 29
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Ou, L., Przybilla, M. J., Koniar, B. L., & Whitley, C. B. (2017) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2017.11.013 · Cited by 23
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Song, P., Zhao, X., & Xiao, S. (2020) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2020.119865 · Cited by 22
El‐Amouri, S. S., Dai, M., Han, J., Brady, R. O., & Pan, D. (2014) · Molecular Therapy · doi:10.1038/mt.2014.152 · Cited by 22
Self-citationDai, M., Liou, B., Swope, B., Wang, X., Zhang, W., Inskeep, V., Grabowski, G. A., Sun, Y., & Pan, D. (2016) · PLoS ONE · doi:10.1371/journal.pone.0162367 · Cited by 21
Self-citationDavid, M., Lécorché, P., Masse, M., Faucon, A., Abouzid, K., Gaudin, N., Varini, K., Gassiot, F., Ferracci, G., Jacquot, G., et al. (2018) · PLoS ONE · doi:10.1371/journal.pone.0191052 · Cited by 23
Li, B., Lin, Y., Chen, G., Cai, M., Zhong, H., Xiao, Z., Lin, M., Li, T., Cai, Y., Shuai, X., et al. (2023) · Advanced Science · doi:10.1002/advs.202302134 · Cited by 21
Al-azzawi, S., Masheta, D., Guildford, A. L., Phillips, G. J., & Santin, M. (2019) · Frontiers in Bioengineering and Biotechnology · doi:10.3389/fbioe.2019.00049 · Cited by 20
Miwa, S., Watabe, A. M., Shimada, Y., Higuchi, T., Kobayashi, H., Fukuda, T., Kato, F., Ida, H., & Ohashi, T. (2020) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2020.06.007 · Cited by 20
Hu, P., Li, Y., Nikolaishvili‐Feinberg, N., Scesa, G., Bi, Y., Pan, D., Moore, D. T., Bongarzone, E. R., Sands, M. S., Miller, C. R., et al. (2016) · Journal of Neuroscience Research · doi:10.1002/jnr.23847 · Cited by 19
Self-citationHorgan, C., Jones, S. A., Bigger, B., & Wynn, R. F. (2022) · International Journal of Molecular Sciences · doi:10.3390/ijms23094854 · Cited by 18
Models to study basic and applied aspects of lysosomal storage disorders
Gaudioso, Á., Silva, T. P., & Ledesma, M. D. (2022) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2022.114532 · Cited by 18
Single enzyme nanoparticle, an effective tool for enzyme replacement therapy
Kim, D. H., Lee, H. S., Kwon, T.-W., Han, Y.-M., Kang, N.-W., Lee, M. Y., Kim, D., Kim, M. G., & Lee, J. (2020) · Archives of Pharmacal Research · doi:10.1007/s12272-020-01216-3 · Cited by 17
Hurdles in treating Hurler disease: potential routes to achieve a “real” cure
van den Broek, B. T. A., van Doorn, J., Hegeman, C. V., Nierkens, S., Lindemans, C. A., Verhoeven‐Duif, N. M., Boelens, J. J., & van Hasselt, P. M. (2020) · Blood Advances · doi:10.1182/bloodadvances.2020001708 · Cited by 17
Dogan, Y., Barese, C. N., Schindler, J. W., Yoon, J. K., Unnisa, Z., Guda, S., Jacobs, M. E., Oborski, C., Maiwald, T., Clarke, D. L., et al. (2022) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2022.10.017 · Cited by 17
Yu, Y., He, S.-Y., Kong, L., Shi, N.-N., Liu, Y., Zang, J., Guo, R., Zhang, L., Li, X., & Li, X. (2023) · Journal of Drug Delivery Science and Technology · doi:10.1016/j.jddst.2023.104794 · Cited by 16
Understanding the In Vivo Fate of Advanced Materials by Imaging
Li, R., Ng, T. S. C., Garlin, M. A., Weissleder, R., & Miller, M. A. (2020) · Advanced Functional Materials · doi:10.1002/adfm.201910369 · Cited by 18
Dai, M., Han, J., El‐Amouri, S. S., Brady, R. O., & Pan, D. (2014) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1323155111 · Cited by 16
Self-citationLin, Y., Wang, X., Rose, K. P., Dai, M., Han, J., Xin, M., & Pan, D. (2020) · Molecular Therapy · doi:10.1016/j.ymthe.2020.06.011 · Cited by 15
Self-citationMucopolysaccharidoses and the blood–brain barrier
Sahin, O., Thompson, H. P., Goodman, G. W., Li, J., & Urayama, A. (2022) · Fluids and Barriers of the CNS · doi:10.1186/s12987-022-00373-5 · Cited by 16
Arcangeli, C., Lico, C., Baschieri, S., & Mancuso, M. (2019) · International Journal of Nanomedicine · doi:10.2147/ijn.s225793 · Cited by 14
Metadata
- Authors
- Wang, D., El-Amouri, S. S., Dai, M., Kuan, C.-Y., Hui, D. Y., Brady, R. O., & Pan, D.
- Affiliations
- Cincinnati Children's Hospital Medical Center (US)
- Divisions of aExperimental Hematology and Cancer Biology and
- Departments of cPathology and
- National Institutes of Health (US)
- University of Cincinnati (US)
- Venue
- Proceedings of the National Academy of Sciences
- Publisher
- National Academy of Sciences
- Published
- 2013-02-04
- Volume
- 110
- Issue
- 8
- Pages
- 2999-3004
- ISSN
- 0027-8424 (ISSN-L)
- 1091-6490
- PMC
- PMC3581871
- OpenAlex
- W2058352256
- Cited by
- 159
- References
- 36
- Citations by year
- 2026: 12 · 2025: 5 · 2024: 8 · 2023: 12 · 2022: 12 · 2021: 12 · 2020: 19 · 2019: 17 · 2018: 13 · 2017: 8 · 2016: 10 · 2015: 8 · 2014: 17 · 2013: 5
- FWCI
- 9.28
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- Proceedings of the National Academy of Sciences (publishedVersion)
- Europe PMC (PubMed Central) (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- Funders
- Massachusetts Institute of Technology
- Seattle Children's Research Institute
- Children’s Hospital of Wisconsin Research Institute
- National Institutes of Health (NS064330)
- National Heart, Lung, and Blood Institute (Z01HL006008, U54 HL-06008)
- National Institute of Diabetes and Digestive and Kidney Diseases (DK074932, R01DK074932)
- National Institute of Neurological Disorders and Stroke (NS 064330, R01NS064330)
- Topics
- Health Sciences › Medicine › Genetics (clinical) › Lysosomal Storage Disorders Research
- Life Sciences › Neuroscience › Neurology › Barrier Structure and Function Studies
- Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
- Keywords
- blood-brain barrier, LDL receptor, receptor-mediated transcytosis, alpha-L-iduronidase, mucopolysaccharidosis type I, lysosomal storage disease, brain drug delivery, fusion protein, glycosaminoglycans, beta-hexosaminidase, lysosomal enzyme trafficking
- MeSH terms
- Animals, Apolipoproteins E, Binding Sites, Blood-Brain Barrier, Disease Models, Animal, Mucopolysaccharidosis I, Lysosomes, Receptors, LDL, Protein Engineering, Mice, Transcytosis
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