Targeting the transferrin receptor to transport antisense oligonucleotides across the mammalian blood-brain barrier

Moderated estimation of fold change and dispersion for RNA-seq data with DESeq2

Love, M. I., Huber, W., & Anders, S. (2014) · Genome Biology · doi:10.1186/s13059-014-0550-8 · Cited by 95,106 · see citing works

Molecular Architecture of the Mouse Nervous System

Zeisel, A., Hochgerner, H., Lönnerberg, P., Johnsson, A., Memic, F., van der Zwan, J., Häring, M., Braun, E., Borm, L. E., La Manno, G., et al. (2018) · Cell · doi:10.1016/j.cell.2018.06.021 · Cited by 2,995 · see citing works

Advances in oligonucleotide drug delivery

Roberts, T. C., Langer, R., & Wood, M. J. A. (2020) · Nature Reviews Drug Discovery · doi:10.1038/s41573-020-0075-7 · Cited by 2,026 · see citing works

Diffusion in Brain Extracellular Space

Syková, E., & Nicholson, C. (2008) · Physiological Reviews · doi:10.1152/physrev.00027.2007 · Cited by 1,481 · see citing works

RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform

Bennett, C. F., & Swayze, E. E. (2010) · The Annual Review of Pharmacology and Toxicology · doi:10.1146/annurev.pharmtox.010909.105654 · Cited by 1,381 · see citing works

Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides

Geary, R. S., Norris, D. A., Yu, R. Z., & Bennett, C. F. (2015) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2015.01.008 · Cited by 846 · see citing works

Increased Brain Penetration and Potency of a Therapeutic Antibody Using a Monovalent Molecular Shuttle

Niewoehner, J., Bohrmann, B., Collin, L., Urich, E., Sade, H., Maier, P., Rueger, P., Stracke, J. O., Lau, W., Tissot, A. C., et al. (2014) · Neuron · doi:10.1016/j.neuron.2013.10.061 · Cited by 622 · see citing works

An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study

Miller, T. M., Pestronk, A., David, W., Rothstein, J., Simpson, E., Appel, S. H., Andres, P. L., Mahoney, K., Allred, P., Alexander, K., et al. (2013) · The Lancet Neurology · doi:10.1016/s1474-4422(13)70061-9 · Cited by 617 · see citing works

Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for <i>SOD1</i> ALS

Miller, T., Cudkowicz, M., Shaw, P. J., Andersen, P. M., Atassi, N., Bucelli, R. C., Genge, A., Glass, J., Ladha, S., Ludolph, A. L., et al. (2020) · New England Journal of Medicine · doi:10.1056/nejmoa2003715 · Cited by 565 · cited 1× · see citing works

Expression of Cux‐1 and Cux‐2 in the subventricular zone and upper layers II–IV of the cerebral cortex

Nieto, M., Monuki, E. S., Tang, H., Imitola, J., Haubst, N., Khoury, S. J., Cunningham, J., Götz, M., & Walsh, C. A. (2004) · The Journal of Comparative Neurology · doi:10.1002/cne.20322 · Cited by 548 · see citing works

Receptor‐mediated transcytosis of transferrin across the blood‐brain barrier

Fishman, J. B., Rubin, J. B., Handrahan, J. V., Connor, J. R., & Fine, R. E. (1987) · Journal of Neuroscience Research · doi:10.1002/jnr.490180206 · Cited by 541 · see citing works

Transcytosis and brain uptake of transferrin-containing nanoparticles by tuning avidity to transferrin receptor

Wiley, D. T., Webster, P. C., Gale, A., & Davis, M. E. (2013) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1307152110 · Cited by 521 · see citing works

Tau reduction prevents neuronal loss and reverses pathological tau deposition and seeding in mice with tauopathy

DeVos, S. L., Miller, R. L., Schoch, K. M., Holmes, B. B., Kebodeaux, C. S., Wegener, A. J., Chen, G., Shen, T., Tran, H., Nichols, B., et al. (2017) · Science Translational Medicine · doi:10.1126/scitranslmed.aag0481 · Cited by 516 · see citing works

An efficient route to human bispecific IgG

Merchant, A. M., Zhu, Z., Yuan, J. Q., Goddard, A. D., Adams, C. W., Presta, L. G., & Carter, P. M. (1998) · Nature Biotechnology · doi:10.1038/nbt0798-677 · Cited by 513 · see citing works

CSF, blood-brain barrier, and brain drug delivery

Pardridge, W. M. (2016) · Expert Opinion on Drug Delivery · doi:10.1517/17425247.2016.1171315 · Cited by 508 · see citing works

The long noncoding RNA Malat1: Its physiological and pathophysiological functions

Zhang, X., Hamblin, M. H., & Yin, K. (2017) · RNA Biology · doi:10.1080/15476286.2017.1358347 · Cited by 489 · see citing works

Delivery of Neurotrophic Factors to the Central Nervous System: Pharmacokinetic Considerations

Thorne, R. G., & Frey, W. H. (2001) · Clinical Pharmacokinetics · doi:10.2165/00003088-200140120-00003 · Cited by 487 · see citing works

Characterization of Foxp2 and Foxp1 mRNA and protein in the developing and mature brain

Ferland, R. J., Cherry, T. J., Preware, P. O., Morrisey, E. E., & Walsh, C. A. (2003) · The Journal of Comparative Neurology · doi:10.1002/cne.10654 · Cited by 477 · see citing works

Crossing the Blood–Brain Barrier: Recent Advances in Drug Delivery to the Brain

Patel, M. M., & Patel, B. M. (2017) · CNS Drugs · doi:10.1007/s40263-016-0405-9 · Cited by 430 · see citing works

Pharmacology of Antisense Drugs

Bennett, C. F., Baker, B. F., Pham, N. C., Swayze, E. E., & Geary, R. S. (2016) · The Annual Review of Pharmacology and Toxicology · doi:10.1146/annurev-pharmtox-010716-104846 · Cited by 429 · see citing works

Blood-Brain Barrier and Delivery of Protein and Gene Therapeutics to Brain

Pardridge, W. M. (2020) · Frontiers in Aging Neuroscience · doi:10.3389/fnagi.2019.00373 · Cited by 405 · see citing works

Transferrin receptor (TfR) trafficking determines brain uptake of TfR antibody affinity variants

Bien‐Ly, N., Yu, Y. J., Bumbaca, D., Elstrott, J., Boswell, C. A., Zhang, Y., Luk, W., Lu, Y., Dennis, M. S., Weimer, R. M., et al. (2014) · The Journal of Experimental Medicine · doi:10.1084/jem.20131660 · Cited by 357 · see citing works

Brain delivery of therapeutic proteins using an Fc fragment blood-brain barrier transport vehicle in mice and monkeys

Kariolis, M. S., Wells, R. C., Getz, J. A., Kwan, W., Mahon, C. S., Tong, R. K., Kim, D. J., Srivastava, A., Bédard, C., Henne, K. R., et al. (2020) · Science Translational Medicine · doi:10.1126/scitranslmed.aay1359 · Cited by 356 · see citing works

Antisense Oligonucleotide Therapies for Neurodegenerative Diseases

Bennett, C. F., Krainer, A. R., & Cleveland, D. W. (2019) · Annual Review of Neuroscience · doi:10.1146/annurev-neuro-070918-050501 · Cited by 353 · see citing works

Diffusion of Macromolecules in the Brain: Implications for Drug Delivery

Wolak, D. J., & Thorne, R. G. (2013) · Molecular Pharmaceutics · doi:10.1021/mp300495e · Cited by 299 · see citing works

MALAT1 Long Non-Coding RNA: Functional Implications

Arun, G., Aggarwal, D., & Spector, D. L. (2020) · Non-Coding RNA · doi:10.3390/ncrna6020022 · Cited by 276 · see citing works

Antisense oligonucleotides: translation from mouse models to human neurodegenerative diseases

Schoch, K. M., & Miller, T. M. (2017) · Neuron · doi:10.1016/j.neuron.2017.04.010 · Cited by 274 · see citing works

Intrathecal antibody distribution in the rat brain: surface diffusion, perivascular transport and osmotic enhancement of delivery

Pizzo, M. E., Wolak, D. J., Kumar, N. N., Brunette, E., Brunnquell, C. L., Hannocks, M., Abbott, N. J., Meyerand, M. E., Sorokin, L., Stanimirovic, D. B., et al. (2018) · The Journal of Physiology · doi:10.1113/jp275105 · Cited by 272 · see citing works

Expanding RNAi therapeutics to extrahepatic tissues with lipophilic conjugates

Brown, K. M., Nair, J. K., Janas, M. M., Anglero-Rodriguez, Y. I., Dang, L. T. H., Peng, H., Theile, C. S., Castellanos-Rizaldos, E., Brown, C., Foster, D., et al. (2022) · Nature Biotechnology · doi:10.1038/s41587-022-01334-x · Cited by 258 · see citing works

Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic

Logan, T., Simon, M. J., Rana, A., Cherf, G. M., Srivastava, A., Davis, S. S., Low, R. L. Y., Chiu, C.-L., Fang, M., Huang, F., et al. (2021) · Cell · doi:10.1016/j.cell.2021.08.002 · Cited by 233 · see citing works

A TREM2-activating antibody with a blood–brain barrier transport vehicle enhances microglial metabolism in Alzheimer’s disease models

van Lengerich, B., Zhan, L., Xia, D., Chan, D., Joy, D., Park, J. I., Tatarakis, D., Calvert, M., Hummel, S., Lianoglou, S., et al. (2023) · Nature Neuroscience · doi:10.1038/s41593-022-01240-0 · Cited by 233 · see citing works

Intracellular transport and regulation of transcytosis across the blood–brain barrier

Villaseñor, R., Lampe, J., Schwaninger, M., & Collin, L. (2018) · Cellular and Molecular Life Sciences · doi:10.1007/s00018-018-2982-x · Cited by 213 · see citing works

Brain delivery and activity of a lysosomal enzyme using a blood–brain barrier transport vehicle in mice

Ullman, J. C., Arguello, A., Getz, J. A., Bhalla, A., Mahon, C. S., Wang, J., Giese, T., Bedard, C., Kim, D. J., Blumenfeld, J. R., et al. (2020) · Science Translational Medicine · doi:10.1126/scitranslmed.aay1163 · Cited by 205 · see citing works

Differential expression of receptors mediating receptor-mediated transcytosis (RMT) in brain microvessels, brain parenchyma and peripheral tissues of the mouse and the human

Zhang, W., Liu, Q. Y., Haqqani, A. S., Leclerc, S., Liu, Z., Fauteux, F., Baumann, E., Delaney, C. E., Ly, D., Star, A. T., et al. (2020) · Fluids and Barriers of the CNS · doi:10.1186/s12987-020-00209-0 · Cited by 200 · see citing works

Sequence motifs associated with hepatotoxicity of locked nucleic acid—modified antisense oligonucleotides

Burdick, A. D., Sciabola, S., Mantena, S. R., Hollingshead, B. D., Stanton, R. V., Warneke, J. A., Zeng, M., Martsen, E., Medvedev, A. V., Makarov, S. S., et al. (2014) · Nucleic Acids Research · doi:10.1093/nar/gku142 · Cited by 179 · see citing works

Direct Intraventricular Delivery of Drugs to the Rodent Central Nervous System

DeVos, S. L., & Miller, T. M. (2013) · Journal of Visualized Experiments · doi:10.3791/50326 · Cited by 174 · see citing works

Nusinersen: A Novel Antisense Oligonucleotide for the Treatment of Spinal Muscular Atrophy

Knierbein, E. E. N., & Bisaccia, E. K. (2019) · The Journal of Pediatric Pharmacology and Therapeutics · doi:10.5863/1551-6776-24.3.194 · Cited by 164 · see citing works

α-Synuclein antisense oligonucleotides as a disease-modifying therapy for Parkinson’s disease

Cole, T. A., Zhao, H. T., Collier, T. J., Sandoval, I. M., Sortwell, C. E., Steece‐Collier, K., Daley, B. F., Booms, A., Lipton, J. W., Welch, M., et al. (2021) · JCI Insight · doi:10.1172/jci.insight.135633 · Cited by 157 · see citing works

Delivery is key: lessons learnt from developing splice‐switching antisense therapies

Godfrey, C., Desviat, L. R., Smedsrød, B. H., Piétri‐Rouxel, F., Denti, M. A., Disterer, P., Lorain, S., Nogales‐Gadea, G., Sardone, V., Anwar, R., et al. (2017) · EMBO Molecular Medicine · doi:10.15252/emmm.201607199 · Cited by 151 · see citing works

Iduronate-2-Sulfatase with Anti-human Transferrin Receptor Antibody for Neuropathic Mucopolysaccharidosis II: A Phase 1/2 Trial

Okuyama, T., Eto, Y., Sakai, N., Minami, K., Yamamoto, T., Sonoda, H., Yamaoka, M., Tachibana, K., Hirato, T., & Sato, Y. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.12.005 · Cited by 151 · see citing works

Cholesterol-functionalized DNA/RNA heteroduplexes cross the blood–brain barrier and knock down genes in the rodent CNS

Nagata, T., Dwyer, C. A., Yoshida‐Tanaka, K., Ihara, K., Ohyagi, M., Kaburagi, H., Miyata, H., Ebihara, S., Yoshioka, K., Ishii, T., et al. (2021) · Nature Biotechnology · doi:10.1038/s41587-021-00972-x · Cited by 140 · see citing works

The atlas of RNase H antisense oligonucleotide distribution and activity in the CNS of rodents and non-human primates following central administration

Jafar-nejad, P., Powers, B., Soriano, A., Zhao, H., Norris, D. A., Matson, J., DeBrosse-Serra, B., Watson, J., Narayanan, P., Chun, S. J., et al. (2021) · Nucleic Acids Research · doi:10.1093/nar/gkaa1235 · Cited by 128 · see citing works

Characterization of Target mRNA Reduction Through In Situ RNA Hybridization in Multiple Organ Systems Following Systemic Antisense Treatment in Animals

Hung, G., Xiao, X., Peralta, R. Q., Bhattacharjee, G., Murray, S., Norris, D., Guo, S., & Monia, B. P. (2013) · Nucleic Acid Therapeutics · doi:10.1089/nat.2013.0443 · Cited by 126 · see citing works

Receptor Crosslinking: A General Method to Trigger Internalization and Lysosomal Targeting of Therapeutic Receptor:Ligand Complexes

Moody, P. R., Sayers, E. J., Magnusson, J. P., Alexander, C., Borri, P., Watson, P., & Jones, A. T. (2015) · Molecular Therapy · doi:10.1038/mt.2015.178 · Cited by 118 · see citing works

Double setback for ASO trials in Huntington disease

Kingwell, K. (2021) · Nature Reviews Drug Discovery · doi:10.1038/d41573-021-00088-6 · Cited by 114 · see citing works

Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice

Klein, A. F., Varela, M. A., Arandel, L., Holland, A., Naouar, N., Arzumanov, A. A., Seoane, D., Revillod, L., Bassez, G., Ferry, A., et al. (2019) · Journal of Clinical Investigation · doi:10.1172/jci128205 · Cited by 113 · see citing works

Sorting Tubules Regulate Blood-Brain Barrier Transcytosis

Villaseñor, R., Schilling, M. R., Sundaresan, J., Lutz, Y., & Collin, L. (2017) · Cell Reports · doi:10.1016/j.celrep.2017.11.055 · Cited by 107 · see citing works

Design, Synthesis And Evaluation Of Constrained Methoxyethyl (cMOE) and Constrained Ethyl (cEt) Nucleoside Analogs

Seth, P. P., Siwkowski, A., Allerson, C. R., Vàsquez, G., Lee, S. S., Prakash, T. P., Kinberger, G. A., Migawa, M. T., Gaus, H. J., Bhat, B., et al. (2008) · Nucleic Acids Symposium Series · doi:10.1093/nass/nrn280 · Cited by 101 · see citing works

Clinical Applications of Single-Stranded Oligonucleotides: Current Landscape of Approved and In-Development Therapeutics

Scharner, J., & Aznarez, I. (2020) · Molecular Therapy · doi:10.1016/j.ymthe.2020.12.022 · Cited by 96 · see citing works

Antisense Oligonucleotide-Mediated Exon Skipping for Duchenne Muscular Dystrophy: Progress and Challenges

Arechavala‐Gomeza, V., Anthony, K., Morgan, J. E., & Francesco Muntoni, A. (2012) · Current Gene Therapy · doi:10.2174/156652312800840621 · Cited by 90 · see citing works

Molecular architecture determines brain delivery of a transferrin receptor–targeted lysosomal enzyme

Arguello, A., Mahon, C. S., Calvert, M., Chan, D., Dugas, J. C., Pizzo, M. E., Thomsen, E. R., Chau, R., Damo, L. A., Duque, J. N., et al. (2021) · The Journal of Experimental Medicine · doi:10.1084/jem.20211057 · Cited by 86 · see citing works

Concepts, technologies, and practices for drug delivery past the blood–brain barrier to the central nervous system

Crawford, L., Rosch, J. G., & Putnam, D. A. (2015) · Journal of Controlled Release · doi:10.1016/j.jconrel.2015.12.041 · Cited by 83 · see citing works

Antibody-oligonucleotide conjugate achieves CNS delivery in animal models for spinal muscular atrophy

Hammond, S. M., Abendroth, F., Goli, L., Burrell, M., Thom, G., Gurrell, I., Stoodley, J., Ahlskog, N., Gait, M. J., Wood, M. J. A., et al. (2022) · JCI Insight · doi:10.1172/jci.insight.154142 · Cited by 78 · see citing works

Mechanisms of palmitic acid-conjugated antisense oligonucleotide distribution in mice

Chappell, A. E., Gaus, H. J., Berdeja, A., Gupta, R., Jo, M., Prakash, T. P., Oestergaard, M. E., Swayze, E. E., & Seth, P. P. (2020) · Nucleic Acids Research · doi:10.1093/nar/gkaa164 · Cited by 73 · see citing works

A physiologically-based pharmacokinetic model to describe antisense oligonucleotide distribution after intrathecal administration

Monine, M. I., Norris, D. A., Wang, Y., & Nestorov, I. A. (2021) · Journal of Pharmacokinetics and Pharmacodynamics · doi:10.1007/s10928-021-09761-0 · Cited by 67 · see citing works

Brain delivery of biologics using a cross‐species reactive transferrin receptor 1 VNAR shuttle

Sehlin, D., Stocki, P., Gustavsson, T., Hultqvist, G., Walsh, F. S., Rutkowski, J. L., & Syvänen, S. (2020) · The FASEB Journal · doi:10.1096/fj.202000610rr · Cited by 66 · see citing works

Technical Guide for Nervous System Sampling of the Cynomolgus Monkey for General Toxicity Studies

Pardo, I. D. R., Garman, R. H., Weber, K., Bobrowski, W. F., Hardisty, J. F., & Morton, D. G. (2012) · Toxicologic Pathology · doi:10.1177/0192623311436180 · Cited by 60 · see citing works

The therapeutic potential of siRNA in gene therapy of neurodegenerative disorders.

Koutsilieri, E., Rethwilm, A., & Scheller, C. (2007) · Journal of neural transmission. Supplementum · doi:10.1007/978-3-211-73574-9_7 · Cited by 59 · see citing works

Passive Immunotherapies for Central Nervous System Disorders: Current Delivery Challenges and New Approaches

Kumar, N. N., Pizzo, M. E., Nehra, G., Wilken-Resman, B., Boroumand, S., & Thorne, R. G. (2018) · Bioconjugate Chemistry · doi:10.1021/acs.bioconjchem.8b00548 · Cited by 57 · see citing works

Direct Intraventricular Delivery of Drugs to the Rodent Central Nervous System

DeVos, S. L., & Miller, T. M. (2013) · Journal of Visualized Experiments · doi:10.3791/50326-v · Cited by 56 · see citing works

Convective forces increase rostral delivery of intrathecal radiotracers and antisense oligonucleotides in the cynomolgus monkey nervous system

Sullivan, J. M., Mazur, C., Wolf, D. A., Horky, L. L., Currier, N. V., Fitzsimmons, B., Hesterman, J. Y., Pauplis, R., Haller, S., Powers, B. E., et al. (2020) · Journal of Translational Medicine · doi:10.1186/s12967-020-02461-2 · Cited by 47 · see citing works

Molecular and Cellular Changes in Breast Cancer and New Roles of lncRNAs in Breast Cancer Initiation and Progression

Kumar, M., DeVaux, R. S., & Herschkowitz, J. I. (2016) · Progress in molecular biology and translational science · doi:10.1016/bs.pmbts.2016.09.011 · Cited by 34 · see citing works

Iduronate-2-sulfatase transport vehicle rescues behavioral and skeletal phenotypes in a mouse model of Hunter syndrome

Arguello, A., Meisner, R., Thomsen, E. R., Nguyen, H. N., Ravi, R., Simms, J. A., Lo, I., Speckart, J., Holtzman, J., Gill, T. M., et al. (2021) · JCI Insight · doi:10.1172/jci.insight.145445 · Cited by 34 · see citing works

Transferrin Receptor Controls AMPA Receptor Trafficking Efficiency and Synaptic Plasticity

Liu, K., Lei, R., Li, Q., Wang, X., Wu, Q., An, P., Zhang, J., Zhu, M., Xu, Z., Hong, Y., et al. (2016) · Scientific Reports · doi:10.1038/srep21019 · Cited by 32 · see citing works

POE Immunoassay: Plate-based oligonucleotide electro-chemiluminescent immunoassay for the quantification of nucleic acids in biological matrices

Thayer, M. B., Humphreys, S. C., Chung, K., Lade, J. M., Cook, K. D., & Rock, B. M. (2020) · Scientific Reports · doi:10.1038/s41598-020-66829-6 · Cited by 24 · see citing works

Quantification of siRNA-Antibody Conjugates in Biological Matrices by Triplex-Forming Oligonucleotide ELISA

Humphreys, S. C., Thayer, M. B., Campuzano, I. D. G., Netirojjanakul, C., & Rock, B. M. (2019) · Nucleic Acid Therapeutics · doi:10.1089/nat.2018.0770 · Cited by 22 · see citing works

Brain and Nasal Cavity Anatomy of the Cynomolgus Monkey: Species Differences from the Viewpoint of Direct Delivery from the Nose to the Brain

Sakane, T., Okabayashi, S., Kimura, S., Inoue, D., Tanaka, A., & Furubayashi, T. (2020) · Pharmaceutics · doi:10.3390/pharmaceutics12121227 · Cited by 16 · see citing works

Frankenstein’ protocol for nuclei isolation from FRESH and FROZEN tissue for snRNA-Seq (10x Genomics Platform) v1Preprint

Martelotto, L. G. (2020) · doi:10.17504/protocols.io.bq24mygw · Cited by 1 · see citing works

Topographic organization of connections between the hypothalamus and prefrontal cortex in the rhesus monkey

Rempel-Clower, N. L., & Barbas, H. (1998) · The Journal of Comparative Neurology · doi:10.1002/(sici)1096-9861(19980831)398:3

RNA chemistry and therapeutics

Wang, S., Weissman, D., & Dong, Y. (2025) · Nature Reviews Drug Discovery · doi:10.1038/s41573-025-01237-x · Cited by 98 · see citing works

Lipid nanoparticles for mRNA delivery in brain via systemic administration

Cao, D., Hou, X., Wang, C., Wang, S., Liu, Z., Tian, M., Guo, K., Li, H., Kang, D. D., Zhong, Y., et al. (2025) · Science Advances · doi:10.1126/sciadv.adw0730 · Cited by 73 · see citing works

Fc-engineered large molecules targeting blood-brain barrier transferrin receptor and CD98hc have distinct central nervous system and peripheral biodistribution

Khoury, N., Pizzo, M. E., Discenza, C. B., Joy, D. A., Tatarakis, D., Todorov, M. I., Negwer, M., Ha, C., de Melo, G. L., Sarrafha, L., et al. (2025) · Nature Communications · doi:10.1038/s41467-025-57108-x · Cited by 73 · see citing works

Drug Delivery Across the Blood–Brain Barrier: A New Strategy for the Treatment of Neurological Diseases

Jiao, Y., Yang, L., Wang, R., Song, G., Fu, J., Wang, J., Gao, N., & Wang, H. (2024) · Pharmaceutics · doi:10.3390/pharmaceutics16121611 · Cited by 63 · see citing works

Crossing the Blood–Brain Barrier: Innovations in Receptor- and Transporter-Mediated Transcytosis Strategies

Ding, L., Kshirsagar, P., Agrawal, P., & Murry, D. J. (2025) · Pharmaceutics · doi:10.3390/pharmaceutics17060706 · Cited by 62 · see citing works

Transferrin receptor-targeted anti-amyloid antibody enhances brain delivery and mitigates ARIA.

Pizzo, M. E., Plowey, E. D., Khoury, N., Kwan, W., Abettan, J., DeVos, S. L., Discenza, C. B., Earr, T., Joy, D., Lye-Barthel, M., et al. (2025) · Science · doi:10.1126/science.ads3204 · Cited by 63 · see citing works

Metabolic Stability and Targeted Delivery of Oligonucleotides: Advancing RNA Therapeutics Beyond The Liver

Anand, P., Zhang, Y., Patil, S., & Kaur, K. (2025) · Journal of Medicinal Chemistry · doi:10.1021/acs.jmedchem.4c02528 · Cited by 51 · see citing works

Intravenous administration of blood–brain barrier-crossing conjugates facilitate biomacromolecule transport into central nervous system

Wang, C., Wang, S., Xue, Y., Zhong, Y., Li, H., Hou, X., Kang, D. D., Liu, Z., Tian, M., Wang, L., et al. (2024) · Nature Biotechnology · doi:10.1038/s41587-024-02487-7 · Cited by 39 · see citing works

Clinical applications of oligonucleotides for cancer therapy

DeFranciscis, V., Amabile, G., & Kortylewski, M. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.02.045 · Cited by 32 · see citing works

Bispecific brain-penetrant antibodies for treatment of Alzheimer’s disease

Sehlin, D., Hultqvist, G., Michno, W., Aguilar, X., Dahlén, A. D., Cerilli, E., Bucher, N. M., van den Broek, S. L., & Syvänen, S. (2025) · The Journal of Prevention of Alzheimer s Disease · doi:10.1016/j.tjpad.2025.100214 · Cited by 29 · see citing works

Beyond the Walls of Troy: A Scoping Review on Pharmacological Strategies to Enhance Drug Delivery Across the Blood–Brain Barrier and Blood–Tumor Barrier

Pinkiewicz, M., Zaczyński, A., Walecki, J. M., & Zawadzki, M. (2025) · International Journal of Molecular Sciences · doi:10.3390/ijms26157050 · Cited by 33 · see citing works

Conjugation to a transferrin receptor 1-binding Bicycle peptide enhances ASO and siRNA potency in skeletal and cardiac muscles

Østergaard, M. E., Carrer, M., Anderson, B. A., Afetian, M. E., Bakooshli, M. A., Santos, J. A., Klein, S., Capitanio, J. S., Freestone, G. C., Tanowitz, M., et al. (2025) · Nucleic Acids Research · doi:10.1093/nar/gkaf270 · Cited by 26 · see citing works

Dual targeting of transferrin receptor and CD98hc enhances brain exposure of large molecules

Wells, R. C., Akkapeddi, P., Chan, D., Joy, D. A., Moshkforoush, A., Chau, R., Chow, J., Cherf, G. M., Chien, E. Y. T., Clemens, A., et al. (2025) · Cell Reports · doi:10.1016/j.celrep.2025.116038 · Cited by 26 · see citing works

A manifesto for Alzheimer’s disease drug discovery in the era of disease-modifying therapies

Hering, H., Bussière, T., Liu, C., Glajch, K. E., Weihofen, A., Grogan, J. L., & Walsh, D. M. (2025) · Molecular Neurodegeneration · doi:10.1186/s13024-025-00872-7 · Cited by 26 · see citing works

Emerging clinical applications of ADAR based RNA editing

Rainaldi, J., Mali, P., & Nourreddine, S. (2025) · Stem Cells Translational Medicine · doi:10.1093/stcltm/szaf016 · Cited by 21 · see citing works

Evolution of antisense oligonucleotides: navigating nucleic acid chemistry and delivery challenges

Ruchi, R., Raman, G. M., Kumar, V., & Bahal, R. (2024) · Expert Opinion on Drug Discovery · doi:10.1080/17460441.2024.2440095 · Cited by 20 · see citing works

Targeting Neuroinflammation in Central Nervous System Diseases by Oral Delivery of Lipid Nanoparticles

Zou, Y., Zhang, J., Chen, L., Xu, Q., Yao, S., & Chen, H. (2025) · Pharmaceutics · doi:10.3390/pharmaceutics17030388 · Cited by 19 · see citing works

RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice

Weiss, A., Gilbert, J. W., Flores, I. V. R., Belgrad, J., Ferguson, C. M., Dogan, E. O., Wightman, N., Mocarski, K., Echeverria, D., Harkins, A. L., et al. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.05.010 · Cited by 18 · see citing works

Nanomedicine for targeting brain Neurodegeneration: Critical barriers and circadian rhythm Considerations

Pineiro‐Alonso, L., Rubio-Prego, I., Lobyntseva, A., González‐Freire, E., Langer, R., & Alonso, M. J. (2025) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2025.115606 · Cited by 18 · see citing works

A brain-targeted and ROS-responsive natural polysaccharide nanogel for enhancing antidepressant therapy

Xu, D. W., Qiao, T., Zhou, Y.-M., Wu, X., & Cui, Y. (2025) · Chemical Engineering Journal · doi:10.1016/j.cej.2025.160719 · Cited by 17 · see citing works

Engineering considerations for next-generation oligonucleotide therapeutics

Ebrahimi, S. B., Bhattacharjee, H., Sonti, S., Fuerst, D. E., Doyle, P. S., Lu, Y., & Samanta, D. (2024) · Nature Chemical Engineering · doi:10.1038/s44286-024-00152-z · Cited by 16 · see citing works

PET imaging of antisense oligonucleotide distribution in rat and nonhuman primate brains using click chemistry

Cook, B. E., Pickel, T. C., Nag, S., Bolduc, P. N., Beshr, R., Forsberg, A., Muste, C. A., Boscutti, G., Jiang, D. M., Yuan, L., et al. (2025) · Science Translational Medicine · doi:10.1126/scitranslmed.adl1732 · Cited by 16 · see citing works

Assessing central nervous system drug delivery

Hammarlund‐Udenaes, M., & Loryan, I. (2025) · Expert Opinion on Drug Delivery · doi:10.1080/17425247.2025.2462767 · Cited by 17 · see citing works

Brain delivery of biotherapeutics via receptor-mediated transcytosis across the blood–brain barrier

Boado, R. J. (2025) · RSC Pharmaceutics · doi:10.1039/d5pm00204d · Cited by 18 · see citing works

Molecular and cellular characteristics of cerebrovascular cell types and their contribution to neurodegenerative diseases

García, F., & Heiman, M. (2025) · Molecular Neurodegeneration · doi:10.1186/s13024-025-00799-z · Cited by 14 · see citing works

Comparative Radiotracing Quantifies Brain Cellular Uptake and Catabolism of Bispecific Antibodies Targeting Transferrin Receptor and CD98hc

Veire, J. L., Lucas, M. J., Bond, L. G., Tripu, D. R., Tessier, P. M., & Greineder, C. F. (2025) · ACS Chemical Neuroscience · doi:10.1021/acschemneuro.4c00552 · Cited by 14 · see citing works

Padlock-designed MOFs triggers an “avalanche effect” to enhance apoptosis and suppress metastasis in central nervous system lymphoma

Gong, Y., Zhou, Z., Xu, T., Liu, F., Chen, A., Zou, L., Jiang, T., Shi, Y., & Miao, Y. (2025) · Science Advances · doi:10.1126/sciadv.adv2647 · Cited by 13 · see citing works

Bridging the blood-brain barrier: strategies to improve delivery of biologics to tumors in the brain

Gampa, G., Vadlakonda, R., Stefanich, E. G., Kamath, A. V., Sadekar, S., & Shivva, V. (2026) · Fluids and Barriers of the CNS · doi:10.1186/s12987-026-00760-2 · Cited by 13 · see citing works

Durable suppression of seizures in a preclinical model of KCNT1 genetic epilepsy with divalent small interfering RNA

Andreone, B. J., Lin, J. S., Tocci, J., Rook, M. L., Omer, A., Carito, L. M., Yang, C., Zhoba, H. B., Dejesus, C., Traore, M., et al. (2025) · Epilepsia · doi:10.1111/epi.18278 · Cited by 12 · see citing works

RNA Therapeutics: Focus on Antisense Oligonucleotides in the Nervous System

Ertural, B., Çiçek, B. N., & Kurnaz, I. A. (2025) · Biomolecules & Therapeutics · doi:10.4062/biomolther.2025.022 · Cited by 13 · see citing works

Angelman syndrome patient-derived neuron screen leads to clinical ASO rugonersen targeting UBE3A -ATS with long-lasting effect in monkeys

Jagasia, R., Bon, C., Rasmussen, S. V., Badillo, S., Tehler, D., Buchy, D., Berrera, M., Prasad, M. K., Chamberlain, S. J., Terrigno, M., et al. (2025) · Nucleic Acids Research · doi:10.1093/nar/gkaf851 · Cited by 18 · see citing works

Balancing brain exposure, pharmacokinetics and safety of transferrin receptor antibodies for delivery of neuro-therapeutics

Smith, B. A., Meeks, C., Murray-Brown, I., McCarthy, E. A., Julian, M. C., Arndt, J. W., Stewart, M., Spilker, K. A., D’Lima, N., Dolníková, J., et al. (2025) · mAbs · doi:10.1080/19420862.2025.2592422 · Cited by 12 · see citing works

The emerging roles of long non-coding RNAs in the nervous system

Altaf, S., Cummins, M. J., Ittner, L. M., & Mattick, J. S. (2025) · Nature reviews. Neuroscience · doi:10.1038/s41583-025-00960-z · Cited by 14 · see citing works

Long non-coding rnas as key modulators of the immune microenvironment in hepatocellular carcinoma: implications for Immunotherapy

Zhao, S., Chen, F., Hu, L., Li, X., Gao, Z., Chen, M., Wang, X., & Song, Z. (2025) · Frontiers in Immunology · doi:10.3389/fimmu.2025.1523190 · Cited by 11 · see citing works

Chemical strategies for brain delivery of genomic therapy

Li, H., Yu, C., Markovic, T., Nestler, E. J., & Dong, Y. (2025) · Nature Reviews Chemistry · doi:10.1038/s41570-025-00770-y · Cited by 14 · see citing works

Bioconjugates for improved delivery of oligonucleotide therapeutics to the central nervous system

Lee, H. J., Xie, Y., Greineder, C. F., & Tessier, P. M. (2026) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2026.115778 · Cited by 11 · see citing works

Alzheimer's Targeted Treatments: Focus on Amyloid and Inflammation

Iwatsubo, T., Irizarry, M. C., Lewcock, J. W., & Carrillo, M. C. (2023) · Journal of Neuroscience · doi:10.1523/jneurosci.1576-23.2023 · Cited by 10 · see citing works

Harnessing the circadian nature of the choroid plexus and cerebrospinal fluid

Fame, R. M. (2025) · npj Biological Timing and Sleep · doi:10.1038/s44323-025-00033-5 · Cited by 9 · see citing works

Insights into pathophysiology, biomarkers, and therapeutics in tauopathies: Proceedings of the Tau2024 Global Conference

Frost, B. E., Rowe, J. B., Akinyemi, R., Abisambra, J. F., Ashton, N. J., Brendel, M., Buée, L., Butler, D., Carrillo, M. C., Chung, P. S., et al. (2025) · Alzheimer s & Dementia · doi:10.1002/alz.70078 · Cited by 9 · see citing works

Targeting tau in Alzheimer’s Disease: rationale, approach and challenges

Aslanyan, A., Foiani, M. S., Giovannucci, T. A., McDade, E., Duff, K., Mummery, C. J., Schöll, M., Wildsmith, K. R., & Paterson, R. W. (2026) · Molecular Neurodegeneration · doi:10.1186/s13024-026-00925-5 · Cited by 10 · see citing works

Bioanalysis of free antisense oligonucleotide payload from antibody–oligonucleotide conjugate by hybridization LC-MS/MS

Jiang, D. M., Li, P., & Yuan, L. (2024) · Bioanalysis · doi:10.1080/17576180.2024.2368339 · Cited by 8 · see citing works

Translational minimal physiologically based pharmacokinetic model for transferrin receptor-mediated brain delivery of antibodies

Muliaditan, M., van Steeg, T. J., Avery, L. B., Sun, W., Hammond, T. R., Hijdra, D., Choi, S.-L., Pillai, N., Leksa, N. C., & Mavroudis, P. D. (2025) · mAbs · doi:10.1080/19420862.2025.2515414 · Cited by 8 · see citing works

Beyond the blood–brain barrier: the fate of transcytosed therapeutics

Baghirov, H. (2025) · Trends in Pharmacological Sciences · doi:10.1016/j.tips.2025.08.006 · Cited by 8 · see citing works

Chemical Modifications in Nucleic Acid Therapeutics

Lennox, K. A., Young, R. C., & Behlke, M. A. (2025) · Methods in molecular biology · doi:10.1007/978-1-0716-4742-4_3 · Cited by 7 · see citing works

Nanoparticle-mediated magnetic hyperthermia in the treatment of neurological disorders

Naveed, M., & Kim, M. (2025) · Nanomedicine · doi:10.1080/17435889.2025.2563499 · Cited by 7 · see citing works

Improved targeted delivery of antisense oligonucleotide with an antibody mask

Hsia, H., Zanini, C., Simonneau, C., Fraidling, J., Kraft, T. E., Mayer, K., Sommer, A., Indlekofer, A., Wirth, T., Benz, J., et al. (2025) · Nucleic Acids Research · doi:10.1093/nar/gkaf487 · Cited by 7 · see citing works

Advances in oligonucleotide therapeutics: recent bench-side progress in ASO, siRNA, and aptamer

Li, Y., Ding, F., Zhu, L., Feng, H., & Zhang, C. (2026) · Materials Today · doi:10.1016/j.mattod.2026.103204 · Cited by 7 · see citing works

Inflamed vessel–anchored release of H 2 across the blood-brain barrier for ischemic stroke neuroprotection

Yu, Y., Fan, M., Wu, G., Li, Y., Xia, C., Ding, W., Li, G., He, Q., Tang, W., & Liu, C. (2026) · Science Advances · doi:10.1126/sciadv.aea3355 · Cited by 7 · see citing works

Blocking somatic repeat expansion and lowering huntingtin via RNA interference synergize to prevent Huntington’s disease pathogenesis in micePreprint

Belgrad, J., Summers, A., Landles, C., Greene, J. R., Hildebrand, S. R., Knox, E. G., Sapp, E., Yamada, N., Furgal, R. C., Miller, R., et al. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2025.06.24.661398 · Cited by 7 · see citing works

Elucidating brain transport pathways and cell type-dependent gene silencing of a durable lipid–siRNA conjugate administered into cerebrospinal fluid

Sorets, A. G., Schwensen, K. R., Francini, N., Kjar, A., Abdulrahman, A. M., Shostak, A., Katdare, K. A., Schoch, K. M., Cowell, R. P., Park, J. C., et al. (2025) · Nucleic Acids Research · doi:10.1093/nar/gkaf600 · Cited by 6 · see citing works

Long non-coding RNA MALAT1: A crucial factor in fibrotic diseases

Ran, L., Pan, W., Feng, J., & Tang, L. (2025) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2025.102630 · Cited by 6 · see citing works

CTAD taskforce: genetic therapies in Alzheimer’s disease

Jakabek, D., Isaacs, A. M., Strooper, B. D., Tuszynski, M. H., Lane, R. M., Uspenskaya, O., McDade, E., Rafii, M. S., & Mummery, C. J. (2025) · The Journal of Prevention of Alzheimer s Disease · doi:10.1016/j.tjpad.2025.100269 · Cited by 6 · see citing works

Strengths, Weaknesses, Opportunities, and Potential Threat Analysis of Targeting Leucine-Rich Repeat Kinase 2 for Parkinson’s Disease Treatment: Status Quo and Quo Vadis

Dang, C., Feng, Z., Xu, T., Li, M., Wang, Z., Song, H. L., & Wang, R. (2025) · Journal of Medicinal Chemistry · doi:10.1021/acs.jmedchem.5c00013 · Cited by 5 · see citing works

Functionalized biomimetic nanoplatforms breaching the blood–brain barrier: A targeted therapeutic strategy for cerebral ischemia–reperfusion injury

Wang, Y., Wang, B., Su, F., Wu, E., Ni, Z., & Chen, Y. (2025) · Materials & Design · doi:10.1016/j.matdes.2025.115154 · Cited by 5 · see citing works

Unlocking the future: Precision oligonucleotide therapy for targeted treatment of neurodegenerative disorders

Jain, N., Roy, A. A., Madhusoodanan, G., Preman, N. K., Pokale, R., Pisay, M., Mukharya, A., Pandey, A. P., & Mutalik, S. (2025) · International Journal of Biological Macromolecules · doi:10.1016/j.ijbiomac.2025.143515 · Cited by 5 · see citing works

Development of an LC-MS/MS assay to analyze a lipid-conjugated siRNA by solid phase extraction (SPE) in mouse plasma and tissue using a stable isotope labeled internal standard (SILIS)

Sanford, E. J., Chen, J., Tran, J., Korboukh, I., & Zhang, G. (2025) · Bioanalysis · doi:10.1080/17576180.2025.2535953 · Cited by 5 · see citing works

Single-dose administration of therapeutic divalent siRNA targeting MECP2 prevents lethality for one year in an MECP2 duplication mouse modelPreprint

Hariharan, V. N., Summers, A., Clipperton‐Allen, A. E., Caiazzi, J., Hildebrand, S. R., Reilly, D. O., Tang, Q., Kennedy, Z., Echeverria, D., McHugh, N., et al. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2025.03.26.645328 · Cited by 5 · see citing works

Skeleton keys and Trojan horses: a review of therapeutic delivery to the brain

Stoub, R. E., & Bailus, B. J. (2025) · Frontiers in Cell and Developmental Biology · doi:10.3389/fcell.2025.1674333 · Cited by 4 · see citing works

Ineffective behavioral rescue despite partial brain Dp427 restoration by AAV9-U7-mediated exon 51 skipping in mdx52 mice

Vacca, O., Saoudi, A., Doisy, M., Phongsavanh, X., Coz, O. L., Nagy, C., Kuzniar, J., Vaillend, C., & Goyenvalle, A. (2025) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2025.102779 · Cited by 4 · see citing works

Therapeutic oligonucleotides revisited: Focus on siRNA and antisense technologies

Chen, L., Xu, J., Li, J., Liu, G., & Ma, H. (2026) · The Innovation Drug Discovery · doi:10.59717/j.xinn-drugdisc.2026.100007 · Cited by 4 · see citing works

Strategies for Transcytosis-Based Delivery across the Blood-Brain Barrier

Baghirov, H. (2025) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.5c01139 · Cited by 4 · see citing works

Recent advances and clinical prospects of non-viral brain-targeted gene delivery systems

Wang, S., Xu, L.-L., Ma, F., Xu, M., Chen, G., Wang, D., Wu, X., Wang, P., Yu, J., & Shi, L. (2025) · Applied Physics Reviews · doi:10.1063/5.0255745 · Cited by 4 · see citing works

The landscape of dementia research, diagnosis, treatment, and care in Latin America

Suemoto, C. K., Custodio, N., Aguilar, D., Ávila‐Funes, J. A., Báez, S., Bagnati, P., Barnes, L. L., Brucki, S. M. D., Calandri, I. L., Caramelli, P., et al. (2026) · Alzheimer s & Dementia · doi:10.1002/alz.71309 · Cited by 4 · see citing works

Engineering the blood–brain barrier on chip: advances in preclinical drug penetration and disease modeling

Sissoko, C. A., Spitz, S., Zhang, Z., Abedi, K., Kamm, R. D., & Barrile, R. (2026) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2026.115881 · Cited by 4 · see citing works

Transport pathways across the blood-brain barrier for waste clearance and drug delivery

Gröbner, L. S., & Pietrzik, C. U. (2026) · Fluids and Barriers of the CNS · doi:10.1186/s12987-026-00812-7 · Cited by 4 · see citing works

The Versatile Applications of Antisense Oligonucleotides in Modern Medicine

Liang, X., & Zhang, L. (2026) · International Journal of Molecular Sciences · doi:10.3390/ijms27125612 · Cited by 4 · see citing works

Advances in RNA delivery to extrahepatic tissues and cells

Li, Z., Guo, S., & Huang, Y. (2026) · Journal of Controlled Release · doi:10.1016/j.jconrel.2026.115170 · Cited by 4 · see citing works

Towards Aptamer-Targeted Drug Delivery to Brain Tumors: The Synthesis of Ramified Conjugates of an EGFR-Specific Aptamer with MMAE on a Cathepsin B-Cleavable Linker

Brylev, V. A., Ryabukhina, E. V., Nazarova, E. V., Samoylenkova, N. S., Gulyak, E. L., Sapozhnikova, K. A., Dzarieva, F., Ustinov, A. V., Pronin, I., Usachev, D. Y., et al. (2024) · Pharmaceutics · doi:10.3390/pharmaceutics16111434 · Cited by 5 · see citing works

Potent and durable gene modulation in heart and muscle with chemically defined siRNAsPreprint

Fakih, H. H., Lochmann, C., Gagnon, R., Summers, A., Caiazzi, J., Buchwald, J. E., Tang, Q., Maru, B., Hildebrand, S. R., Abideen, M. Z. U., et al. (2024) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2024.10.01.616183 · Cited by 4 · see citing works

Multiplex screening of transferrin receptor-targeted lipid nanoparticles for systemic brain delivery using DNA barcodes

Ghosh, S., Fan, Y., Laufer, B. I., Stephenson, W., Zang, N., Lawrence, J., Lin, H. H., Sen, S., Miller, S. E., Chu, S., et al. (2026) · Cell Biomaterials · doi:10.1016/j.celbio.2025.100303 · Cited by 3 · see citing works

Cell Type Distribution of Intrathecal Antisense Oligonucleotide Activity in Deep Brain Regions of Non-Human Primates

Frei, J. A., Gentile, J. E., Lian, Y., Mortberg, M. A., Capitanio, J. S., Jafar‐Nejad, P., Vallabh, S. M., Zhao, H. T., & Minikel, E. V. (2025) · Nucleic Acid Therapeutics · doi:10.1177/21593337251371594 · Cited by 4 · see citing works

Artificial miRNA-mediated reduction of SNCA for the treatment of α-synucleinopathies

Elmer, B. M., Wischhof, E., Chan, T., Mahendran, T., Ardinger, J. A., Richards, B., Liu, D., Bladon, J. H., He, Y., Jackson, R., et al. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.11.017 · Cited by 3 · see citing works

TfR-aptamer programmed DNA nanoflowers penetrate the blood-brain barrier for synergistic NLRP3 silencing and neuroinflammation therapy

Kong, X., Hu, B., Luo, X., Zhou, J., Zhang, C., Zi, R., Zhang, C., Zhang, Y., Jiang, H., Liu, T., et al. (2025) · Chemical Engineering Journal · doi:10.1016/j.cej.2025.170771 · Cited by 3 · see citing works

Transferrin-Functionalized Liposomes Enhance MAPT-ASO Transport Across a 3D Blood–Brain Barrier Microvascular Network Model

Konig, S., Shen, X., Mantovani, G., Winkler, G. S., Zhu, Z., & Moeendarbary, E. (2025) · International Journal of Molecular Sciences · doi:10.3390/ijms262311347 · Cited by 3 · see citing works

The application of CRISPR-Cas9 system in brain diseases

Miao, Y., Li, C., Su, Y., Peng, T., Wang, J., Liu, S., Ma, C., Li, L., & Wang, Y. (2026) · Nanomedicine · doi:10.1080/17435889.2026.2629031 · Cited by 3 · see citing works

Brain and Liver Dual‐Targeting Oridonin Nanoparticles to Enhance Aβ Clearance for Alzheimer's Disease Therapy

Gong, W., Hui, W., Qiao, S., Ji, Q., Liu, M., Zhang, B., Liu, D., Wu, Y., & Zhou, S. (2026) · Advanced Science · doi:10.1002/advs.202523458 · Cited by 3 · see citing works

Metabolic regulation of ferroptosis in cancer: mechanisms, pharmacological inducers, and translational challenges

Liu, Y. (2026) · APOPTOSIS · doi:10.1007/s10495-026-02377-0 · Cited by 3 · see citing works

Impact of ASO conjugation and receptor binding affinity on intracellular transport of mono- and bispecific TfR- and CD98-Brainshuttle TM variants

Sela, T., Wirth, T., Sommer, A., Larraillet, V., Butzer, J., Lohmann, S., Wittmann, P., Klein, C., Knoetgen, H., Villaseñor, R., et al. (2026) · mAbs · doi:10.1080/19420862.2026.2691351 · Cited by 3 · see citing works

Skeletal muscle effects of antisense oligonucleotides targeting glycogen synthase 1 in a mouse model of Pompe disease

Weiss, L. N., Carrer, M., Shmara, A., Martin, A., Yin, H. Z., Pal, P., Cheng, C., Ta, L., Boock, V., Fazeli, Y., et al. (2025) · Clinical and Translational Medicine · doi:10.1002/ctm2.70314 · Cited by 3 · see citing works

Brain drugs can now cross the once impenetrable blood–brain barrier

Abbott, A. (2025) · Nature · doi:10.1038/d41586-025-01569-z · Cited by 3 · see citing works

Intravenous lipid-siRNA conjugate mediates gene silencing at the blood-brain barrier and blood-CSF barrier

Sorets, A. G., Schwensen, K. R., Francini, N., Kjar, A., Lyons, S., Park, J. C., Palmer, D., Abdulrahman, A. M., Cowell, R. P., Katdare, K. A., et al. (2025) · Journal of Controlled Release · doi:10.1016/j.jconrel.2025.114226 · Cited by 2 · see citing works

Potent and durable gene modulation in heart and muscle with chemically defined lipophilic siRNAs

Fakih, H. H., Lochmann, C., Gagnon, R., Summers, A., Caiazzi, J., Buchwald, J. E., Tang, Q., Maru, B., Hildebrand, S. R., Abideen, M. Z. U., et al. (2025) · Nucleic Acids Research · doi:10.1093/nar/gkaf1374 · Cited by 2 · see citing works

Model selection in preclinical nucleic acid therapeutics research

Oliver, P. L., & Hill, A. C. (2026) · Communications Biology · doi:10.1038/s42003-026-09650-7 · Cited by 2 · see citing works

The Tale of the Guanosine Tract in Repeat Expansion Disorders

Karati, S., Rawat, S., Kumari, A., Das, S., Shukla, S., & Kumar, A. (2026) · Molecular Neurobiology · doi:10.1007/s12035-026-05735-w · Cited by 2 · see citing works

Molecular signatures of cell diversity modulated by long noncoding RNAs in the human fetal spinal cord

Miao, N., Lee, T., Chen, L., Zhang, H., Wang, J. H., Sun, J., Sun, J., Sha, Y., Huang, S., & Sun, T. (2026) · iScience · doi:10.1016/j.isci.2026.115399 · Cited by 2 · see citing works

Hypoxia-induced circSPECC1 drives temozolomide resistance in glioblastoma via IGF2BP2-mediated PGK1 mRNA stabilization

Zeng, Y., Zhao, L., Que, T., Zhou, L. T., Yang, X., Xu, X., Cao, K., Wang, X. A., 王旭辉, Zhang, W., et al. (2026) · Cell Death and Disease · doi:10.1038/s41419-026-08879-8 · Cited by 2 · see citing works

CD98hc-targeted antibody shuttles for central nervous system delivery with broad cross-species reactivity

WU, W., Sivaneri, N. S., Akin, E., Spesia, C. J., Huang, Y. J., Bond, L. G., Doi, D. L. Q., Stad, J., Djouadi, A., Xie, Y., et al. (2026) · Nature Biomedical Engineering · doi:10.1038/s41551-026-01718-3 · Cited by 3 · see citing works

Blocking somatic repeat expansion and lowering huntingtin by RNAi synergize to attenuate Huntington’s disease pathogenesis in mice

Belgrad, J., Summers, A., Landles, C., Greene, J. R., Hildebrand, S. R., Knox, E. G., Sapp, E., Yamada, N., Furgal, R. C., Miller, R., et al. (2026) · Science Translational Medicine · doi:10.1126/scitranslmed.aea3104 · Cited by 2 · see citing works

The Emergence of Oligonucleotide Building Blocks in the Multispecific Proximity-Inducing Drug Toolbox of Destruction

Zhou, K. X. T., & Bujold, K. E. (2024) · ACS Chemical Biology · doi:10.1021/acschembio.4c00311 · 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

Beyond muscle: Delivering RNA therapeutics to the CNS in Duchenne muscular dystrophy

Vacca, O., Nagy, C., & Goyenvalle, A. (2025) · Journal of Neuromuscular Diseases · doi:10.1177/22143602251360668 · Cited by 2 · see citing works

Exploring the pleiotropic effects of lncRNA in different repeat expansion disorders

Das, S., Khandelwal, S., Shukla, S., & Kumar, A. (2025) · Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease · doi:10.1016/j.bbadis.2025.168089 · Cited by 2 · see citing works

Breaking Barriers: Delivering Therapeutics to the Brain in Alzheimer’s Disease

Strooper, B. D., Scheltens, P., & Salloway, S. (2025) · The Journal of Prevention of Alzheimer s Disease · doi:10.1016/j.tjpad.2025.100229 · Cited by 2 · see citing works

A BBB-penetrated, microglia-targeted, and hypoxia-responsive nanogel for treating ischemic stroke

Xia, Y., Li, R., Ni, Y., Zhang, X., Liu, Y., Kang, H., Liu, Y., Yu, C., Deng, Y., Jiang, Z., et al. (2026) · Materials Today Advances · doi:10.1016/j.mtadv.2026.100738 · Cited by 1 · see citing works

Genetic medicines for epilepsy: unlocking new avenues for seizure control

Wee, I. C., Przybyla, M., Touma, J., Ittner, L. M., & van Eersel, J. (2026) · Frontiers in Bioengineering and Biotechnology · doi:10.3389/fbioe.2026.1768550 · Cited by 1 · see citing works

Nanoparticle-Based Drug Delivery Across the Blood–Brain Barrier: Current In Vivo Evidence, Translational Challenges, and Future Perspectives

Al‐allaq, A. A., Hassan, H. A., Hidayet, H. M., Abdulhakeem, A. A., & Ahmad, Z. A.-A. Q. (2026) · Micro · doi:10.3390/micro6030065 · Cited by 1 · see citing works

TogoPhosTAC as a delivery-ready platform for targeted protein dephosphorylation

Chen, Y.-Y., Ke, D.-T., Lee, Y., Chen, H., Lin, H.-Y., Yeh, C.-L., Ko, Y.-C., Tu, M., Yu, C., Hu, Z., et al. (2026) · Nature Communications · doi:10.1038/s41467-026-76725-8 · Cited by 1 · see citing works

CA-IV-directed small-molecule shuttle enables targeted brain delivery of biologics

Ding, X., Chen, X., Boehm, P., Carretero‐Cerdán, A., Jang, S., Sullivan, E. E., Arokiaraj, C. M., Ristic, F., Kavvathas, B., Campos, L., et al. (2026) · Nature Chemical Biology · doi:10.1038/s41589-026-02294-y · Cited by 1 · see citing works

Harnessing TfR1 for Cross-Species Systemic Delivery of siRNAs to Deep Brain Regions Using Single-Domain AntibodiesPreprint

Jacquot, G., David, M., Pecqueux, B., Mechioukhi, Y., Girard, S. D., Godard, M., Varini, K., Boursery, C., FRAPOLLI, C., Sebastien, R. O. U. X., et al. (2026) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.64898/2026.05.20.726486 · Cited by 1 · see citing works

Running Up that Hill: An Interview with Carole Ho, Denali Therapeutics

Ho, C., & Davies, K. (2025) · GEN Biotechnology · doi:10.1089/genbio.2025.0006

Targeting Transferrin Receptor to Transport Antisense Oligonucleotides Across the Blood-Brain BarrierPreprint

Barker, S. J., Thayer, M. B., Kim, C., Tatarakis, D., Simon, M. J., Dial, R. L., Nilewski, L., Wells, R. C., Zhou, Y., Afetian, M. E., et al. (2023) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2023.04.25.538145 · Cited by 21 · see citing works

Delivery of small interfering RNA and antisense oligonucleotides across the blood-brain barrier with monovalent transferrin receptor 1 binding VHH-Fc fusion proteinsPreprint

Huggins, I. J., Carrer, M., Santos, J. A., Fazio, M., Holguin, B., Phi, S., Prakash, T. P., Afetian, M. E., Bakooshli, M. A., Klein, S. K., et al. (2026) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.64898/2026.08.13.744307

Transferrin-Functionalized Liposomes Enhance MAPT-ASO Transport Across a 3D Blood–Brain Barrier Microvascular Network Model

Konig, S., Shen, X., Mantovani, G., Winkler, G. S., Zhu, Z., & Moeendarbary, E. (2025) · International Journal of Molecular Sciences · doi:10.3390/ijms262311347 · Cited by 3 · see citing works

Angubindin-1 opens the blood–brain barrier in vivo for delivery of antisense oligonucleotide to the central nervous system (S14.009)

Kuwahara, H., Zeniya, S., Nagata, T., & Yokota, T. (2023) · Neurology · doi:10.1212/wnl.0000000000204102

Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System

Chen, Y., Mazur, C., Luo, Y., Sun, L., Zhang, M., McCampbell, A., & Tomassy, G. S. (2019) · Journal of Visualized Experiments · doi:10.3791/60274-v · Cited by 2 · see citing works