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For: Scott LJ. Givosiran: First Approval. Drugs 2020;80:335-339. [PMID: 32034693 DOI: 10.1007/s40265-020-01269-0] [Citation(s) in RCA: 260] [Impact Index Per Article: 52.0] [Reference Citation Analysis] [What about the content of this article? (0)] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 01/01/2023]
Number Cited by Other Article(s)
1
Naeem S, Zhang J, Zhang Y, Wang Y. Nucleic acid therapeutics: Past, present, and future. MOLECULAR THERAPY. NUCLEIC ACIDS 2025;36:102440. [PMID: 39897578 PMCID: PMC11786870 DOI: 10.1016/j.omtn.2024.102440] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Subscribe] [Scholar Register] [Indexed: 02/04/2025]
2
Xiao B, Wang S, Pan Y, Zhi W, Gu C, Guo T, Zhai J, Li C, Chen YQ, Wang R. Development, opportunities, and challenges of siRNA nucleic acid drugs. MOLECULAR THERAPY. NUCLEIC ACIDS 2025;36:102437. [PMID: 39897581 PMCID: PMC11787428 DOI: 10.1016/j.omtn.2024.102437] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Subscribe] [Scholar Register] [Indexed: 02/04/2025]
3
Vosoughi P, Naghib SM, Kangarshahi BM, Mozafari MR. A review of RNA nanoparticles for drug/gene/protein delivery in advanced therapies: Current state and future prospects. Int J Biol Macromol 2025;295:139532. [PMID: 39765293 DOI: 10.1016/j.ijbiomac.2025.139532] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 09/22/2024] [Revised: 01/02/2025] [Accepted: 01/03/2025] [Indexed: 01/13/2025]
4
Boutary S, Khalaf G, Landesman Y, Madani ME, Desmaële D, Piguet F, Alonso R, Banchi EG, Adams D, Massaad C, Massaad-Massade L. Therapeutic potential of siRNA PMP22-SQ nanoparticles for Charcot-Marie-Tooth 1A neuropathy in rodents and non-human primates. Int J Pharm 2025;671:125234. [PMID: 39855282 DOI: 10.1016/j.ijpharm.2025.125234] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 10/15/2024] [Revised: 01/13/2025] [Accepted: 01/14/2025] [Indexed: 01/27/2025]
5
Ramírez-Cortés F, Ménová P. Hepatocyte targeting via the asialoglycoprotein receptor. RSC Med Chem 2025;16:525-544. [PMID: 39628900 PMCID: PMC11609720 DOI: 10.1039/d4md00652f] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 08/22/2024] [Accepted: 11/19/2024] [Indexed: 12/06/2024]  Open
6
Harumoto T, Kawai R, Motosawa K, Iwano J, Koda Y, Hirata Y, Uehara K. Effect of pH-Responsive Ligands on mRNA Knockdown in EGFR-Targeting Ligand-Conjugated siRNAs. ACS Chem Biol 2025. [PMID: 39898496 DOI: 10.1021/acschembio.4c00507] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 02/04/2025]
7
Kulus M, Farzaneh M, Sheykhi-Sabzehpoush M, Ghaedrahmati F, Mehravar F, Józkowiak M, Piotrowska-Kempisty H, Bukowska D, Antosik P, Podhorska-Okołów M, Zabel M, Mozdziak P, Dzięgiel P, Kempisty B. Exosomes and non-coding RNAs: Exploring their roles in human myocardial dysfunction. Biomed Pharmacother 2025;183:117853. [PMID: 39827809 DOI: 10.1016/j.biopha.2025.117853] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 09/07/2024] [Revised: 12/29/2024] [Accepted: 01/13/2025] [Indexed: 01/22/2025]  Open
8
Fan J, Xiao Z, Dong Y, Ye F, Qiu Y, Zhang C, Yin X, Li Y, Wang T. Nanocarrier-Mediated RNA Delivery Platform as a Frontier Strategy for Hepatic Disease Treatment: Challenges and Opportunities. Adv Healthc Mater 2025;14:e2402933. [PMID: 39723654 DOI: 10.1002/adhm.202402933] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 08/07/2024] [Revised: 12/13/2024] [Indexed: 12/28/2024]
9
Fortuna A, Sorarù G. Cervical lower motor neuron syndromes: A diagnostic challenge. J Neurol Sci 2025;468:123357. [PMID: 39740575 DOI: 10.1016/j.jns.2024.123357] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 10/14/2024] [Revised: 12/10/2024] [Accepted: 12/13/2024] [Indexed: 01/02/2025]
10
Ullah A, Khan M, Zhang Y, Shafiq M, Ullah M, Abbas A, Xianxiang X, Chen G, Diao Y. Advancing Therapeutic Strategies with Polymeric Drug Conjugates for Nucleic Acid Delivery and Treatment. Int J Nanomedicine 2025;20:25-52. [PMID: 39802382 PMCID: PMC11717654 DOI: 10.2147/ijn.s429279] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 09/13/2024] [Accepted: 11/26/2024] [Indexed: 01/16/2025]  Open
11
Bhasuran B, Schmolly K, Kapoor Y, Jayakumar NL, Doan R, Amin J, Meninger S, Cheng N, Deering R, Anderson K, Beaven SW, Wang B, Rudrapatna VA. Reducing diagnostic delays in acute hepatic porphyria using health records data and machine learning. J Am Med Inform Assoc 2025;32:63-70. [PMID: 38946554 PMCID: PMC11648717 DOI: 10.1093/jamia/ocae141] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 01/19/2024] [Revised: 05/01/2024] [Accepted: 05/28/2024] [Indexed: 07/02/2024]  Open
12
Klabenkova K, Zakhryamina A, Burakova E, Bizyaev S, Fokina A, Stetsenko D. Synthesis of New Polyfluoro Oligonucleotides via Staudinger Reaction. Int J Mol Sci 2024;26:300. [PMID: 39796153 PMCID: PMC11719919 DOI: 10.3390/ijms26010300] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 12/10/2024] [Revised: 12/28/2024] [Accepted: 12/30/2024] [Indexed: 01/13/2025]  Open
13
Meinhard S, Erdmann F, Lucas H, Krabbes M, Krüger S, Wölk C, Mäder K. T14diLys/DOPE Liposomes: An Innovative Option for siRNA-Based Gene Knockdown? Pharmaceutics 2024;17:25. [PMID: 39861674 PMCID: PMC11769127 DOI: 10.3390/pharmaceutics17010025] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 11/23/2024] [Revised: 12/14/2024] [Accepted: 12/20/2024] [Indexed: 01/27/2025]  Open
14
Anvari S, Nikbakht M, Vaezi M, Amini-Kafiabad S, Ahmadvand M. Immune checkpoints and ncRNAs: pioneering immunotherapy approaches for hematological malignancies. Cancer Cell Int 2024;24:410. [PMID: 39702293 DOI: 10.1186/s12935-024-03596-8] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 03/19/2024] [Accepted: 12/03/2024] [Indexed: 12/21/2024]  Open
15
Barber HM, Pater AA, Gagnon KT, Damha MJ, O'Reilly D. Chemical engineering of CRISPR-Cas systems for therapeutic application. Nat Rev Drug Discov 2024:10.1038/s41573-024-01086-0. [PMID: 39690326 DOI: 10.1038/s41573-024-01086-0] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Accepted: 10/28/2024] [Indexed: 12/19/2024]
16
Wahane A, Kasina V, Pathuri M, Marro-Wilson C, Gupta A, Slack FJ, Bahal R. Development of bioconjugate-based delivery systems for nucleic acids. RNA (NEW YORK, N.Y.) 2024;31:1-13. [PMID: 39477529 DOI: 10.1261/rna.080273.124] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Subscribe] [Scholar Register] [Indexed: 12/18/2024]
17
Purewal JS, Doshi GM. RNAi in psoriasis: A melodic exploration of miRNA, shRNA, and amiRNA with a spotlight on siRNA. Eur J Pharmacol 2024;985:177083. [PMID: 39481628 DOI: 10.1016/j.ejphar.2024.177083] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 06/25/2024] [Revised: 10/28/2024] [Accepted: 10/29/2024] [Indexed: 11/02/2024]
18
Mikutis S, Bernardes GJL. Technologies for Targeted RNA Degradation and Induced RNA Decay. Chem Rev 2024;124:13301-13330. [PMID: 39499674 PMCID: PMC11638902 DOI: 10.1021/acs.chemrev.4c00472] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 06/21/2024] [Revised: 10/03/2024] [Accepted: 10/29/2024] [Indexed: 11/07/2024]
19
Abbasi Dezfouli S, Michailides ME, Uludag H. Delivery Aspects for Implementing siRNA Therapeutics for Blood Diseases. Biochemistry 2024;63:3059-3077. [PMID: 39388611 DOI: 10.1021/acs.biochem.4c00327] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 10/12/2024]
20
Tan YF, Hii LW, Lim WM, Cheong SK, Leong CO, Yee MSL, Mai CW. Polyethylene glycol-phospholipid functionalized single-walled carbon nanotubes for enhanced siRNA systemic delivery. Sci Rep 2024;14:30098. [PMID: 39627280 PMCID: PMC11615393 DOI: 10.1038/s41598-024-80646-1] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 08/05/2024] [Accepted: 11/21/2024] [Indexed: 12/06/2024]  Open
21
Repkova M, Mazurkov O, Filippova E, Protsenko M, Mazurkova N, Meschaninova M, Levina A, Zarytova V. Effect of modification of siRNA molecules delivered with aminopropylsilanol nanoparticles on suppression of A/H5N1 virus in cell culture. Biochim Biophys Acta Gen Subj 2024;1868:130727. [PMID: 39437973 DOI: 10.1016/j.bbagen.2024.130727] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 07/02/2024] [Revised: 10/16/2024] [Accepted: 10/18/2024] [Indexed: 10/25/2024]
22
Liu J, Xi Z, Fan C, Mei Y, Zhao J, Jiang Y, Zhao M, Xu L. Hydrogels for Nucleic Acid Drugs Delivery. Adv Healthc Mater 2024;13:e2401895. [PMID: 39152918 DOI: 10.1002/adhm.202401895] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 05/22/2024] [Revised: 07/05/2024] [Indexed: 08/19/2024]
23
Bauer IA, Dmitrienko EV. Amphiphilic Oligonucleotide Derivatives-Promising Tools for Therapeutics. Pharmaceutics 2024;16:1447. [PMID: 39598570 PMCID: PMC11597563 DOI: 10.3390/pharmaceutics16111447] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 10/18/2024] [Revised: 11/07/2024] [Accepted: 11/11/2024] [Indexed: 11/29/2024]  Open
24
Prabhakaran R, Thamarai R, Sivasamy S, Dhandayuthapani S, Batra J, Kamaraj C, Karthik K, Shah MA, Mallik S. Epigenetic frontiers: miRNAs, long non-coding RNAs and nanomaterials are pioneering to cancer therapy. Epigenetics Chromatin 2024;17:31. [PMID: 39415281 PMCID: PMC11484394 DOI: 10.1186/s13072-024-00554-6] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 07/23/2024] [Accepted: 09/25/2024] [Indexed: 10/18/2024]  Open
25
Ban J, Seo BK, Yu Y, Kim M, Choe J, Park JH, Park SY, Lee DK, Kim SH. Nonclinical Pharmacokinetics Study of OLX702A-075-16, N-Acetylgalactosamine Conjugated Asymmetric Small Interfering RNA (GalNAc-asiRNA). Drug Metab Dispos 2024;52:1262-1270. [PMID: 39168524 DOI: 10.1124/dmd.124.001805] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 05/17/2024] [Revised: 08/14/2024] [Accepted: 08/14/2024] [Indexed: 08/23/2024]  Open
26
Wang TT, Hirons A, Doerflinger M, Morris KV, Ledger S, Purcell DFJ, Kelleher AD, Ahlenstiel CL. Current State of Therapeutics for HTLV-1. Viruses 2024;16:1616. [PMID: 39459949 PMCID: PMC11512412 DOI: 10.3390/v16101616] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 09/02/2024] [Revised: 10/07/2024] [Accepted: 10/11/2024] [Indexed: 10/28/2024]  Open
27
Nomura K, An S, Kobayashi Y, Kondo J, Shi T, Murase H, Nakamoto K, Kimura Y, Abe N, Ui-Tei K, Abe H. Synthesis of 2'-formamidonucleoside phosphoramidites for suppressing the seed-based off-target effects of siRNAs. Nucleic Acids Res 2024;52:10754-10774. [PMID: 39231537 PMCID: PMC11472056 DOI: 10.1093/nar/gkae741] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 02/11/2024] [Revised: 07/31/2024] [Accepted: 08/18/2024] [Indexed: 09/06/2024]  Open
28
Panda K, Alagarasu K, Tagore R, Paingankar M, Kumar S, Jeengar MK, Cherian S, Parashar D. RNAi-Induced Gene Silencing against Chikungunya and COVID-19: What Have We Learned So Far, and What Is the Way Forward? Viruses 2024;16:1489. [PMID: 39339965 PMCID: PMC11437507 DOI: 10.3390/v16091489] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 07/19/2024] [Revised: 08/27/2024] [Accepted: 08/29/2024] [Indexed: 09/30/2024]  Open
29
Alameh MG, Tavakoli Naeini A, Dwivedi G, Lesage F, Buschmann MD, Lavertu M. Chitosan siRNA Nanoparticles Produce Significant Non-Toxic Functional Gene Silencing in Kidney Cortices. Polymers (Basel) 2024;16:2547. [PMID: 39274180 PMCID: PMC11398103 DOI: 10.3390/polym16172547] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 07/30/2024] [Revised: 08/30/2024] [Accepted: 09/02/2024] [Indexed: 09/16/2024]  Open
30
Bakrania A, Mo Y, Zheng G, Bhat M. RNA nanomedicine in liver diseases. Hepatology 2024:01515467-990000000-00569. [PMID: 37725757 DOI: 10.1097/hep.0000000000000606] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 06/12/2023] [Accepted: 09/08/2023] [Indexed: 09/21/2023]
31
Qiao M, Zeng C, Liu C, Lei Z, Liu B, Xie H. The advancement of siRNA-based nanomedicine for tumor therapy. Nanomedicine (Lond) 2024;19:1841-1862. [PMID: 39145477 PMCID: PMC11418284 DOI: 10.1080/17435889.2024.2377062] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 03/05/2024] [Accepted: 07/03/2024] [Indexed: 08/16/2024]  Open
32
Lei JJ, Li S, Dong BX, Yang J, Ren Y. Acute intermittent porphyria: a disease with low penetrance and high heterogeneity. Front Genet 2024;15:1374965. [PMID: 39188285 PMCID: PMC11345236 DOI: 10.3389/fgene.2024.1374965] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 01/23/2024] [Accepted: 07/29/2024] [Indexed: 08/28/2024]  Open
33
Ferraresso F, Leung J, Kastrup CJ. RNA therapeutics to control fibrinolysis: review on applications in biology and medicine. J Thromb Haemost 2024;22:2103-2114. [PMID: 38663489 PMCID: PMC11269028 DOI: 10.1016/j.jtha.2024.04.006] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 02/13/2024] [Revised: 04/11/2024] [Accepted: 04/11/2024] [Indexed: 05/26/2024]
34
Sun H, Wang X, Li Y, Shen Y, Zhang L, Xu Y, Liu J, Fan X. Targeting Liver Xor by GalNAc-siRNA Is an Effective Strategy for Hyperuricemia Therapy. Pharmaceutics 2024;16:938. [PMID: 39065635 PMCID: PMC11279964 DOI: 10.3390/pharmaceutics16070938] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 04/09/2024] [Revised: 06/25/2024] [Accepted: 07/05/2024] [Indexed: 07/28/2024]  Open
35
Sun J, Lu S, Xiao J, Xu N, Li Y, Xu J, Deng M, Xuanyuan H, Zhang Y, Wu F, Jin W, Liu K. Inhibition of SARS-CoV-2 Replication by Self-Assembled siRNA Nanoparticles Targeting Multiple Highly Conserved Viral Sequences. Viruses 2024;16:1072. [PMID: 39066234 PMCID: PMC11281333 DOI: 10.3390/v16071072] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 04/28/2024] [Revised: 05/26/2024] [Accepted: 05/30/2024] [Indexed: 07/28/2024]  Open
36
Traber GM, Yu AM. The Growing Class of Novel RNAi Therapeutics. Mol Pharmacol 2024;106:13-20. [PMID: 38719476 PMCID: PMC11187687 DOI: 10.1124/molpharm.124.000895] [Citation(s) in RCA: 1] [Impact Index Per Article: 1.0] [Reference Citation Analysis] [Abstract] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 02/15/2024] [Accepted: 04/15/2024] [Indexed: 06/20/2024]  Open
37
Motamedi H, Ari MM, Alvandi A, Abiri R. Principle, application and challenges of development siRNA-based therapeutics against bacterial and viral infections: a comprehensive review. Front Microbiol 2024;15:1393646. [PMID: 38939184 PMCID: PMC11208694 DOI: 10.3389/fmicb.2024.1393646] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 02/29/2024] [Accepted: 05/28/2024] [Indexed: 06/29/2024]  Open
38
Hariharan VN, Nakamura T, Shin M, Tang Q, Sontakke V, Caiazzi J, Hildebrand S, Khvorova A, Yamada K. Phosphatidylcholine head group chemistry alters the extrahepatic accumulation of lipid-conjugated siRNA. MOLECULAR THERAPY. NUCLEIC ACIDS 2024;35:102230. [PMID: 38938759 PMCID: PMC11209015 DOI: 10.1016/j.omtn.2024.102230] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Subscribe] [Scholar Register] [Received: 02/07/2024] [Accepted: 05/22/2024] [Indexed: 06/29/2024]
39
Hwang G, Kwon M, Seo D, Kim DH, Lee D, Lee K, Kim E, Kang M, Ryu JH. ASOptimizer: Optimizing antisense oligonucleotides through deep learning for IDO1 gene regulation. MOLECULAR THERAPY. NUCLEIC ACIDS 2024;35:102186. [PMID: 38706632 PMCID: PMC11066473 DOI: 10.1016/j.omtn.2024.102186] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Subscribe] [Scholar Register] [Received: 10/17/2023] [Accepted: 04/03/2024] [Indexed: 05/07/2024]
40
Sun N, Lei Q, Wu M, Gao S, Yang Z, Lv X, Wei R, Yan F, Cai L. Metal-organic framework-mediated siRNA delivery and sonodynamic therapy for precisely triggering ferroptosis and augmenting ICD in osteosarcoma. Mater Today Bio 2024;26:101053. [PMID: 38654934 PMCID: PMC11035110 DOI: 10.1016/j.mtbio.2024.101053] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 01/30/2024] [Revised: 03/27/2024] [Accepted: 04/09/2024] [Indexed: 04/26/2024]  Open
41
Saw PE, Song E. Advancements in clinical RNA therapeutics: Present developments and prospective outlooks. Cell Rep Med 2024;5:101555. [PMID: 38744276 PMCID: PMC11148805 DOI: 10.1016/j.xcrm.2024.101555] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 01/16/2024] [Revised: 03/05/2024] [Accepted: 04/15/2024] [Indexed: 05/16/2024]
42
Xie R, Yuan S, Hu G, Zhan J, Jin K, Tang Y, Fan J, Zhao Y, Wang F, Chen C, Wang DW, Li H. Nuclear AGO2 promotes myocardial remodeling by activating ANKRD1 transcription in failing hearts. Mol Ther 2024;32:1578-1594. [PMID: 38475992 PMCID: PMC11081878 DOI: 10.1016/j.ymthe.2024.03.018] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 10/13/2023] [Revised: 02/01/2024] [Accepted: 03/08/2024] [Indexed: 03/14/2024]  Open
43
Soufizadeh P, Mansouri V, Ahmadbeigi N. A review of animal models utilized in preclinical studies of approved gene therapy products: trends and insights. Lab Anim Res 2024;40:17. [PMID: 38649954 PMCID: PMC11034049 DOI: 10.1186/s42826-024-00195-6] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 11/09/2023] [Revised: 02/18/2024] [Accepted: 02/23/2024] [Indexed: 04/25/2024]  Open
44
Karthik S, Mohan S, Magesh I, Bharathy A, Kolipaka R, Ganesamoorthi S, Sathiya K, Shanmugavadivu A, Gurunathan R, Selvamurugan N. Chitosan nanocarriers for non-coding RNA therapeutics: A review. Int J Biol Macromol 2024;263:130361. [PMID: 38395284 DOI: 10.1016/j.ijbiomac.2024.130361] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 10/05/2023] [Revised: 02/02/2024] [Accepted: 02/19/2024] [Indexed: 02/25/2024]
45
Chen Y, Li Y, Li C, Zhang D, Liu Y, Zhang J, Guan S, Ding X, Xiao Q. The current perspective and opportunities of small nucleic acid-based therapeutics. Drug Dev Res 2024;85:e22164. [PMID: 38411296 DOI: 10.1002/ddr.22164] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 08/27/2023] [Revised: 02/03/2024] [Accepted: 02/13/2024] [Indexed: 02/28/2024]
46
Shuai Q, Xie W, Chen S, Su H, Yan Y. Novel aromatic moieties-modified poly(glycidyl amine)s with potent siRNA delivery and cancer treatment effect. J Mater Chem B 2024;12:3115-3128. [PMID: 38451094 DOI: 10.1039/d3tb02876c] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 03/08/2024]
47
Sawada H, Kakisawa Y, Ueno Y. Properties and synergistic effects of a nonionic backbone and aminoalkyl modified nucleosides in RNAs. Bioorg Chem 2024;144:107143. [PMID: 38309000 DOI: 10.1016/j.bioorg.2024.107143] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 10/30/2023] [Revised: 01/04/2024] [Accepted: 01/18/2024] [Indexed: 02/05/2024]
48
Pérez-Carrión MD, Posadas I, Ceña V. Nanoparticles and siRNA: A new era in therapeutics? Pharmacol Res 2024;201:107102. [PMID: 38331236 DOI: 10.1016/j.phrs.2024.107102] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 12/10/2023] [Revised: 02/03/2024] [Accepted: 02/05/2024] [Indexed: 02/10/2024]
49
Li X, Jin K, Cheng TC, Liao YC, Lee WJ, Bhullar AS, Chen LC, Rychahou P, Phelps MA, Ho YS, Guo P. RNA four-way junction (4WJ) for spontaneous cancer-targeting, effective tumor-regression, metastasis suppression, fast renal excretion and undetectable toxicity. Biomaterials 2024;305:122432. [PMID: 38176263 PMCID: PMC10994150 DOI: 10.1016/j.biomaterials.2023.122432] [Citation(s) in RCA: 1] [Impact Index Per Article: 1.0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 08/12/2023] [Revised: 11/14/2023] [Accepted: 12/14/2023] [Indexed: 01/06/2024]
50
Khoushab S, Aghmiuni MH, Esfandiari N, Sarvandani MRR, Rashidi M, Taheriazam A, Entezari M, Hashemi M. Unlocking the potential of exosomes in cancer research: A paradigm shift in diagnosis, treatment, and prevention. Pathol Res Pract 2024;255:155214. [PMID: 38430814 DOI: 10.1016/j.prp.2024.155214] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 12/16/2023] [Revised: 02/11/2024] [Accepted: 02/15/2024] [Indexed: 03/05/2024]
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