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Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy
Open accessAbstract
The brain vasculature forms an immense network such that most neural cells are in contact with a microvessel. Here we tested the hypothesis that endothelia lining these vessels can be harnessed to create a cellular reservoir of enzyme replacement therapy to diseased brain. As a model system, we used mice with central nervous system (CNS) deficits due to lysosomal storage disease (LSD mice). The basic premise of this work is that recombinant enzyme expressed in, and secreted from, the vascular endothelia will be endocytosed by underlying neurons and glia, decreasing neuropathology. We screened a phage library in vivo by panning to identify peptides that bound the vascular endothelia in diseased and wild-type mice. Epitopes binding diseased brain were distinct from those panned from normal brain. Moreover, different epitopes were identified in two distinct LSD disease models, implying a unique vascular signature imparted by the disease state. Presentation of these epitopes on the capsid of adeno-associated virus (AAV) expanded the biodistribution of intravenously injected AAV from predominantly liver to include the CNS. Peripheral injection of the epitope-modified AAVs expressing the enzymes lacking in LSD mice reconstituted enzyme activity throughout the brain and improved disease phenotypes in two distinct disease models (pages 1123–1124).
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Grimm, D., Lee, J. S., Wang, L., Desai, T., Akache, B., Storm, T. A., & Kay, M. A. (2008) · Journal of Virology · doi:10.1128/jvi.00254-08 · Cited by 696
Directed evolution of adeno-associated virus yields enhanced gene delivery vectors
Maheshri, N., Koerber, J. T., Kaspar, B. K., & Schaffer, D. V. (2006) · Nature Biotechnology · doi:10.1038/nbt1182 · Cited by 538 · cited 1×
“Finally, the use of AAV libraries generated by inserting amino acid sequences into the viral capsid or by capsid shufflin”
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Mingozzi, F., & High, K. A. (2011) · Nature Reviews Genetics · doi:10.1038/nrg2988 · Cited by 893
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Asokan, A., Schaffer, D. V., & Jude Samulski, R. (2012) · Molecular Therapy · doi:10.1038/mt.2011.287 · Cited by 465
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Challis, R. C., Ravindra Kumar, S., Chan, K. Y., Challis, C., Beadle, K., Jang, M. J., Kim, H. M., Rajendran, P. S., Tompkins, J. D., Shivkumar, K., et al. (2019) · Nature Protocols · doi:10.1038/s41596-018-0097-3 · Cited by 409
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Castle, M. J., Turunen, H. T., Vandenberghe, L. H., & Wolfe, J. H. (2016) · Methods in Molecular Biology · doi:10.1007/978-1-4939-3271-9_10 · Cited by 151
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Chen, Y. H., Keiser, M. S., & Davidson, B. L. (2018) · Current Protocols in Mouse Biology · doi:10.1002/cpmo.58 · Cited by 146
Self-citationDavidsson, M., Wang, G., Aldrin-Kirk, P., Cardoso, T., Nolbrant, S., Hartnor, M., Mudannayake, J., Parmar, M., & Björklund, T. (2019) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1910061116 · Cited by 142
AAV vectors applied to the treatment of CNS disorders: Clinical status and challenges
Kang, L., Jin, S., Wang, J., Lv, Z., Xin, C., Tan, C., Zhao, M., Wang, L., & Liu, J. (2023) · Journal of Controlled Release · doi:10.1016/j.jconrel.2023.01.067 · Cited by 131
Merkel, S. F., Andrews, A. M., Lutton, E. M., Mu, D., Hudry, E., Hyman, B. T., Maguire, C. A., & Ramirez, S. H. (2017) · Journal of Neurochemistry · doi:10.1111/jnc.13861 · Cited by 132
Novel adeno-associated viral vectors for retinal gene therapy.
Vandenberghe, L. H., & Auricchio, A. (2012) · Gene Therapy · doi:10.1038/gt.2011.151 · Cited by 131
Gonzalez-Carter, D., Liu, X., Tockary, T. A., Dirisala, A., Toh, K., Anraku, Y., & Kataoka, K. (2020) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.2002016117 · Cited by 127
Tordo, J., O’Leary, C., Antunes, A. S. L. M., Palomar, N., Aldrin-Kirk, P., Basche, M., Bennett, A., D’Souza, Z., Gleitz, H., Godwin, A., et al. (2018) · Brain · doi:10.1093/brain/awy126 · Cited by 113
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Kantor, B., Bailey, R. M., Wimberly, K., Kalburgi, S. N., & Gray, S. J. (2014) · Advances in Genetics · doi:10.1016/b978-0-12-800149-3.00003-2 · Cited by 109
Modulation of CD8+ T cell responses to AAV vectors with IgG-derived MHC class II epitopes
Hui, D. J., Basner-Tschakarjan, E., Chen, Y., Davidson, R. J., Buchlis, G., Yazicioglu, M., Pien, G. C., Finn, J. D., Haurigot, V., Tai, A., et al. (2013) · Molecular Therapy · doi:10.1038/mt.2013.166 · Cited by 47
Self-citation
Metadata
- Authors
- Chen, Y. H., Chang, M., & Davidson, B. L.
- Venue
- Nature Medicine
- Published
- 2009-09-13
- Volume
- 15
- Issue
- 10
- Pages
- 1215-1218
- ISSN
- 1078-8956 (ISSN-L)
- 1546-170X
- OpenAlex
- W2074766632
- Cited by
- 168
- References
- 2
- FWCI
- 9.84
- Type
- journal article
- Language
- en
- Open access
- green
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