Springer AD, Dowdy SF. GalNAc-siRNA conjugates: leading the way for delivery of RNAi therapeutics. Nucleic Acid Ther. 2018;28:109–118.
Article CAS PubMed PubMed Central Google Scholar
Debacker AJ, Voutila J, Catley M, Blakey D, Habib N. Delivery of oligonucleotides to the liver with GalNAc: from research to registered therapeutic drug. Mol Ther. 2020;28:1759–1771.
Article CAS PubMed PubMed Central Google Scholar
Wu X, Liu S, Liu D, Li X, Wang H, Han X. Global and Chinese trends in oligonucleotide drug clinical development: a comparative analysis. Pharmacol Res. 2024;210:107487.
Zhang Y, Chen H, Hong L, Wang H, Li B, Zhang M, et al. Inclisiran: a new generation of lipid-lowering siRNA therapeutic. Front Pharm. 2023;14:1260921.
Ray KK, Wright RS, Kallend D, Koenig W, Leiter LA, Raal FJ, et al. Two phase 3 trials of inclisiran in patients with elevated LDL cholesterol. N Engl J Med. 2020;382:1507–1519.
Article CAS PubMed Google Scholar
Qin Z-X, Zuo L, Zeng Z, Ma R, Xie W, Zhu X, et al. GalNac-siRNA conjugate delivery technology promotes the treatment of typical chronic liver diseases. Expert Opin Drug Deliv. 2025;22:455–469.
Article CAS PubMed Google Scholar
Kaffe E, Roulis M, Zhao J, Qu R, Sefik E, Mirza H, et al. Humanized mouse liver reveals endothelial control of essential hepatic metabolic functions. Cell. 2023;186:3793–3809.e26.
Article CAS PubMed PubMed Central Google Scholar
Malecova B, Burke RS, Cochran M, Hood MD, Johns R, Kovach PR, et al. Targeted tissue delivery of RNA therapeutics using antibody-oligonucleotide conjugates (AOCs). Nucleic Acids Res. 2023;51:5901–5910.
Article CAS PubMed PubMed Central Google Scholar
Song E, Zhu P, Lee SK, Chowdhury D, Kussman S, Dykxhoorn DM, et al. Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors. Nat Biotechnol. 2005;23:709–17.
Article CAS PubMed Google Scholar
Cuellar TL, Barnes D, Nelson C, Tanguay J, Yu SF, Wen X, et al. Systematic evaluation of antibody-mediated siRNA delivery using an industrial platform of THIOMAB-siRNA conjugates. Nucleic Acids Res. 2015;43:1189–203.
Article CAS PubMed Google Scholar
Janas MM, Schlegel MK, Harbison CE, Yilmaz VO, Jiang Y, Parmar R, et al. Selection of GalNAc-conjugated siRNAs with limited off-target-driven rat hepatotoxicity. Nat Commun. 2018;9:723.
Article PubMed PubMed Central Google Scholar
Zhang C, Dai P, Vinogradov AA, Gates ZP, Pentelute BL. Site-Selective Cysteine–Cyclooctyne Conjugation. Angew Chem Int Ed. 2018;57:6459–6463.
Song X, Wang X, Ma Y, Liang Z, Yang Z, Cao H. Site-Specific Modification Using the 2′-Methoxyethyl Group Improves the Specificity and Activity of siRNAs. Mol Ther - Nucleic Acids. 2017;9:242–250.
Article CAS PubMed PubMed Central Google Scholar
Geall AJ, Doppalapudi VR, Chu DS-H, Cochran MC, Hood M, Darimont BD, et al. Compositions and methods of treating muscle atrophy and myotonic dystrophy. PubChem Patent Summary for US-10881743-B2.
Roberts TC, Langer R, Wood MJA. Advances in oligonucleotide drug delivery. Nat Rev Drug Discov. 2020;19:673–694.
Article CAS PubMed PubMed Central Google Scholar
Meng Q, Yang M, Xing F, Xie Z, Hao Y, Jiang P, et al. Antibody-oligonucleotide conjugates in cancer therapy: potential and promise. Crit Rev Oncol/Hematol. 2025;215:104858.
Su Z, Xiao D, Xie F, Liu L, Wang Y, Fan S, et al. Antibody-drug conjugates: Recent advances in linker chemistry. Acta Pharm Sin B. 2021;11:3889–3907.
Article CAS PubMed PubMed Central Google Scholar
Rennick JJ, Johnston APR, Parton RG. Key principles and methods for studying the endocytosis of biological and nanoparticle therapeutics. Nat Nanotechnol. 2021;16:266–276.
Article CAS PubMed Google Scholar
Candelaria PV, Leoh LS, Penichet ML, Daniels-Wells TR. Antibodies targeting the transferrin receptor 1 (TfR1) as direct anti-cancer agents. Front Immunol. 2021;12:607692.
Article CAS PubMed PubMed Central Google Scholar
Aisen P. Transferrin receptor 1. Int J Biochem Cell Biol. 2004;36:2137–2143.
Article CAS PubMed Google Scholar
Etxaniz U, Diaz M, Bhardwaj R, Tyaglo O, Lemoine K, Missinato MA, et al. Antibody-oligonucleotide conjugates (AOCs) demonstrate potent and durable exon skipping and dystrophin restoration in a mouse model of duchenne muscular dystrophy (S23.003). Neurology. 2022;98:1761.
Girgenrath M, Estrella N, Kumar A, Li J, Hicks A, Brennan C, et al. P20 Endosomal Escape Vehicles (EEV™) - Oligonucleotides conjugates produce exon skipping and dystrophin production in preclinical models of Duchenne muscular dystrophy. Neuromuscul Disord. 2023;33:S102.
Alam R, Alvarado A, Andrews FH, Babb NA, Balasubramaniam D, Chauvigne-Hines L, et al. Transferrin receptor binding proteins and conjugates. Publication Number WO/2024/036096.
Lee HN, Jeong MS, Jang SB. Molecular characteristics of amyloid precursor protein (APP) and its effects in cancer. Int J Mol Sci. 2021;22:4999.
Gordon RE, Nemeth JF, Singh S, Lingham RB, Grewal IS. Harnessing SLE autoantibodies for intracellular delivery of biologic therapeutics. Trends Biotechnol. 2021;39:298–310.
Article CAS PubMed Google Scholar
Rattray Z, Deng G, Zhang S, Shirali A, May CK, Chen X, et al. ENT2 facilitates brain endothelial cell penetration and blood-brain barrier transport by a tumor-targeting anti-DNA autoantibody. JCI Insight. 2021;6:e145875.
Naes SM, Ab-Rahim S, Mazlan M, Abdul Rahman A, Zhang DM. Equilibrative nucleoside transporter 2: properties and physiological roles. BioMed Res Int. 2020;2020:5197626.
Anderson CM, Baldwin SA, Young JD, Cass CE, Parkinson FE. Distribution of mRNA encoding a nitrobenzylthioinosine-insensitive nucleoside transporter (ENT2) in rat brain. Brain Res Mol Brain Res. 1999;70:293–7.
Article CAS PubMed Google Scholar
Manso T, Sanou G, Nousias C, Maalem I, Boutin F, Giudicelli V, et al. Identification of engineered IMGT Fc variants in IMGT/mAb-DB, a database of therapeutic antibodies and fusion proteins. MAbs. 2025;17:2594260.
Article PubMed PubMed Central Google Scholar
Rackear M, Quijano E, Ianniello Z, Colón-Ríos DA, Krysztofiak A, Abdullah R, et al. Next-generation cell-penetrating antibodies for tumor targeting and RAD51 inhibition. Oncotarget. 2024;15:699–713.
Article PubMed PubMed Central Google Scholar
Chen CF, Hsu EC, Lin KT, Tu PH, Chang HW, Lin CH, et al. Overlapping high-resolution copy number alterations in cancer genomes identified putative cancer genes in hepatocellular carcinoma. Hepatology. 2010;52:1690–701.
Article CAS PubMed Google Scholar
Farré X, Guillén-Gómez E, Sánchez L, Hardisson D, Plaza Y, Lloberas J, et al. Expression of the nucleoside-derived drug transporters hCNT1, hENT1 and hENT2 in gynecologic tumors. Int J Cancer. 2004;112:959–66.
Tufail M, Jiang C-H, Li N. Immune evasion in cancer: mechanisms and cutting-edge therapeutic approaches. Signal Transduction Targeted Therapy. 2025;10:227.
Lanza F, Maffini E, Rondoni M, Massari E, Faini AC, Malavasi F. CD22 expression in B-cell acute lymphoblastic leukemia: biological significance and implications for inotuzumab therapy in adults. Cancers. 2020;12:303.
Martino M, Alati C, Canale FA, Musuraca G, Martinelli G, Cerchione C. A review of clinical outcomes of CAR T-cell therapies for B-acute lymphoblastic leukemia. Int J Mol Sci. 2021;22:2150.
Duan S, Paulson JC. Siglecs as immune cell checkpoints in disease. Annu Rev Immunol. 2020;38:365–395.
Article CAS PubMed Google Scholar
Uy N, Nadeau Nguyen M, Stahl M, Zeidan A. Inotuzumab ozogamicin in the treatment of relapsed/refractory acute B cell lymphoblastic leukemia. J Blood Med. 2018;9:67–74.
Kwong LS, Brown MH, Barclay AN, Hatherley D. Signal-regulatory protein α from the NOD mouse binds human CD47 with an exceptionally high affinity – implications for engraftment of human cells. Immunology. 2014;143:61–67.
Article CAS PubMed PubMed Central Google Scholar
Eladl E, Tremblay-LeMay R, Rastgoo N, Musani R, Chen W, Liu A, et al. Role of CD47 in hematological malignancies. J Hematol Oncol. 2020;13:96.
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