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Targeting the transferrin receptor to transport antisense oligonucleotides across the mammalian blood-brain barrier
Abstract
Antisense oligonucleotides (ASOs) are promising therapeutics for treating various neurological disorders. However, ASOs are unable to readily cross the mammalian blood-brain barrier (BBB) and therefore need to be delivered intrathecally to the central nervous system (CNS). Here, we engineered a human transferrin receptor 1 (TfR1) binding molecule, the oligonucleotide transport vehicle (OTV), to transport a tool ASO across the BBB in human TfR knockin (TfR mu/hu KI) mice and nonhuman primates. Intravenous injection and systemic delivery of OTV to TfR mu/hu KI mice resulted in sustained knockdown of the ASO target RNA, Malat1 , across multiple mouse CNS regions and cell types, including endothelial cells, neurons, astrocytes, microglia, and oligodendrocytes. In addition, systemic delivery of OTV enabled Malat1 RNA knockdown in mouse quadriceps and cardiac muscles, which are difficult to target with oligonucleotides alone. Systemically delivered OTV enabled a more uniform ASO biodistribution profile in the CNS of TfR mu/hu KI mice and greater knockdown of Malat1 RNA compared with a bivalent, high-affinity TfR antibody. In cynomolgus macaques, an OTV directed against MALAT1 displayed robust ASO delivery to the primate CNS and enabled more uniform biodistribution and RNA target knockdown compared with intrathecal dosing of the same unconjugated ASO. Our data support systemically delivered OTV as a potential platform for delivering therapeutic ASOs across the BBB.
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“In contrast, intrathecally administered compounds must travel further along the spinal cord before reaching rostral brain regions [47].”
From Elucidating brain transport pathways and cell type-dependent gene silencing of a durable lipid–siRNA conjugate administered into cerebrospinal fluid (2025) · cites this paper as [47]
In the citing paper
“numerous studies suggest that ASOs are not well suited for deep brain structure targets [16,47,52].”
From Elucidating brain transport pathways and cell type-dependent gene silencing of a durable lipid–siRNA conjugate administered into cerebrospinal fluid (2025) · cites this paper as [47]
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Metadata
- Authors
- Barker, S. J., Thayer, M. B., Kim, C., Tatarakis, D., Simon, M. J., Dial, R., Nilewski, L., Wells, R. C., Zhou, Y., Afetian, M., Akkapeddi, P., Chappell, A., Chew, K. S., Chow, J., Clemens, A., Discenza, C. B., Dugas, J. C., Dwyer, C., Earr, T., Ha, C., Ho, Y. S., Huynh, D., Lozano, E. I., Jayaraman, S., Kwan, W., Mahon, C., Pizzo, M., Robles-Colmenares, Y., Roche, E., Sanders, L., Stergioulis, A., Tong, R., Tran, H., Zuchero, Y., Estrada, A. A., Gadkar, K., Koth, C. M. M., Sanchez, P. E., Thorne, R. G., Watts, R. J., Sandmann, T., Kane, L. A., Rigo, F., Dennis, M. S., Lewcock, J. W., & DeVos, S. L.
- Affiliations
- Denali Therapeutics (United States) (US)
- Ionis Pharmaceuticals (United States) (US)
- Denali Therapeutics Inc., South San Francisco, CA, USA.
- Corresponding author
- Barker, Scarlett J.
- Thayer, Mai B.
- Kim, Chaeyoung
- Tatarakis, David
- ORCID
- Barker, Scarlett J.
- Thayer, Mai B.
- Kim, Chaeyoung
- Tatarakis, David
- Simon, Matthew J.
- Dial, Rebekah
- Nilewski, Lizanne
- Wells, Robert C.
- Afetian, Megan
- Akkapeddi, Padma
- Chappell, Alfred
- Chew, Kylie S.
- Chow, Johann
- Discenza, Claire B.
- Dugas, Jason C.
- Dwyer, Chrissa
- Earr, Timothy
- Ha, Connie
- Jayaraman, Srini
- Mahon, Cathal
- Pizzo, Michelle
- Robles-Colmenares, Yaneth
- Sanders, Laura
- Stergioulis, Alexander
- Tong, Raymond
- Tran, Hai
- Zuchero, Y.
- Estrada, Anthony A.
- Gadkar, Kapil
- Koth, Christopher M. M.
- Sanchez, Pascal E.
- Thorne, Robert G.
- Watts, Ryan J.
- Sandmann, Thomas
- Kane, Lesley A.
- Rigo, Frank
- Dennis, Mark S.
- Lewcock, Joseph W.
- DeVos, Sarah L.
- Venue
- Science Translational Medicine
- Published
- 2024-08-14
- Volume
- 16
- Issue
- 760
- Pages
- eadi2245-eadi2245
- ISSN
- 1946-6234 (ISSN-L)
- 1946-6242
- OpenAlex
- W4401596729
- Cited by
- 142
- References
- 70
- Citations by year
- 2026: 65 · 2025: 68 · 2024: 8
- FWCI
- 19.19
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Science Translational Medicine (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Neuroscience › Neurology › Barrier Structure and Function Studies
- Health Sciences › Medicine › Oncology › Drug Transport and Resistance Mechanisms
- Keywords
- antisense oligonucleotides, blood-brain barrier, transferrin receptor 1, MALAT1, brain drug delivery, systemic drug delivery, cynomolgus macaques, RNA knockdown, intrathecal drug delivery, tissue distribution
- MeSH terms
- Animals, Biological Transport, Blood-Brain Barrier, Humans, Macaca fascicularis, Receptors, Transferrin, Tissue Distribution, Oligonucleotides, Antisense, Mice, Gene Knockdown Techniques, RNA, Long Noncoding
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