RNA therapy: rich history, various applications and unlimited future prospects

참고문헌 (76)

1. 김영국, "RNA Therapy: Current Status and Future Potential", 전남의대학술지, 56(2), pp.87~93 (2020), 의과학연구소[DOI]
2. Crick, FH, "On protein synthesis", Symp Soc Exp Biol, 12, pp.138~163 (1958)
3. Brenner, S, "An unstable intermediate carrying information from genes to ribosomes for protein synthesis", Nature, 190, pp.576~581 (1961)
4. Gros, F, "Unstable ribonucleic acid revealed by pulse labelling of Escherichia coli", Nature, 190, pp.581~585 (1961)
5. Rich, A, "A new two stranded helical structure: polyadenylic acid and polyuridylic acid", J Am Chem Soc, 78, pp.3548~3549 (1956)
6. Lee, RC, "The C. elegans heterochronic gene lin-4encodes small RNAs with antisense complementarity to lin-14", Cell, 75, pp.843~854 (1993)
7. Wightman, B, "Posttranscriptional regulation of the heterochronic gene lin-14 by lin-4 mediates temporal pattern formation in C. elegans", Cell, 75, pp.855~862 (1993)
8. Fire, A, "Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans", Nature, 391, pp.806~811 (1998)
9. Zamecnik, PC, "Inhibition of Rous sarcoma virus replication and cell transformation by a specific oligodeoxynucleotide", Proc Natl Acad Sci USA, 75, pp.280~284 (1978)
10. Roehr, B, "Fomivirsen approved for CMV retinitis", J Int Assoc Phys AIDS Care, 4, pp.14~16 (1998)
11. Berget, SM, "Spliced segments at the 5’ terminus of adenovirus 2 late mRNA", Proc Natl Acad Sci USA, 74, pp.3171~3175 (1977)
12. Chow, LT, "An amazing sequence arrangement at the 5’ ends of adenovirus 2 messenger RNA", Cell, 12, pp.1~8 (1977)
13. Faustino, NA, "Pre-mRNA splicing and human disease", Genes Dev, 17, pp.419~437 (2003)
14. Scotti, MM, "RNA mis-splicing in disease", Nat Rev Genet, 17, pp.19~32 (2016)
15. Dominski, Z, "Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides", Proc Natl Acad Sci USA, 90, pp.8673~8677 (1993)
16. McCaffrey, AP, "RNA interference in adult mice", Nature, 418, pp.38~39 (2002)
17. Davis, ME, "Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles", Nature, 464, pp.1067~1070 (2010)
18. Adams, D, "Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis", N Engl J Med, 379, pp.11~21 (2018)
19. Baltimore, D, "Preliminary data on a virus-specific enzyme system responsible for the synthesis of viral RNA", Biochem Biophys Res Commun, 9, pp.388~392 (1962)
20. Reich, E, "Tatumel Action of actinomycin D on animal cells and viruses", Proc Natl Acad Sci USA, 48, pp.1238~1245 (1962)
21. Bloom, K, "Self-amplifying RNA vaccines for infectious diseases", Gene Ther, 28, pp.117~129 (2021)
22. Baltimore, D, "RNA-dependent D. N. A. polymerase in virions of RNA tumour viruses", Nature, 226, pp.1209~1211 (1970)
23. Temin, HM, "RNA-dependent DNA polymerase in virions of Rous sarcoma virus", Nature, 226, pp.1211~1213 (1970)
24. Wolff, JA, "Direct gene transfer into mouse muscle in vivo", Science, 247, pp.1465~1468 (1990)
25. Martinon, F, "Induction of virus-specific cytotoxic T lymphocytes in vivo by liposome-entrapped mRNA", Eur J Immunol, 23, pp.1719~1722 (1993)
26. Conry, RM, "Characterization of a messenger RNA polynucleotide vaccine vector", Cancer Res, 55, pp.1397~1400 (1995)
27. Weide, B, "Results of the first phase I", II clinical vaccination trial with direct injection of mRNA, 2008, pp.31, 180~188
28. Weide, B, "Direct injection of protamine-protected mRNA: results of a phase 1", 2vaccination trial in metastatic melanoma patients, 2009, pp.32, 498~507
29. Alberer, M, "Safety and immunogenicity of a mRNA rabies vaccine in healthy adults: an open-label, non-randomised, prospective, first-in-human phase 1clinical trial", Lancet, 390, pp.1511~1520 (2017)
30. Frankish, A, "GENCODE reference annotation for the human and mouse genomes", Nucleic Acids Res, 47, pp.D766~D773 (2019)
31. Esteller, M, "Non-coding RNAs in human disease", Nat Rev Genet, 12, pp.861~874 (2011)
32. Beermann, J, "Non-coding RNAs in development and disease: background, mechanisms, and therapeutic approaches", Physiol Rev, 96, pp.1297~1325 (2016)
33. Song, J, "Targeting non-coding RNAs for the treatment of retinal diseases", Mol Ther Nucleic Acids, 24, pp.284~293 (2021)
34. Fauman, EB, "Structure-based druggability assessment–identifying suitable targets for small molecule therapeutics", Curr Opin Chem Biol, 15, pp.463~468 (2011)
35. Finan, C, "The druggable genome and support for target identification and validation in drug development", Sci Transl Med, 9, pp.eaag1166~ (2017)
36. Springer, AD, "GalNAc-siRNA Conjugates: leading the way for delivery of RNAi therapeutics", Nucleic Acid Ther, 28, pp.109~118 (2018)
37. Jackson, LA, "An mRNA vaccine against SARS-CoV-2- preliminary report", N Engl J Med, 383, pp.1920~1931 (2020)
38. Mulligan, MJ, "Phase I", II study of COVID-19 RNA vaccine BNT162b1 in adults, 2020, pp.586, 589~593
39. Roberts, TC, "Advances in oligonucleotide drug delivery", Nat Rev Drug Discov, 19, pp.673~694 (2020)
40. Thi, TTH, "Lipid-based nanoparticles in the clinic and clinical trials: from cancer nanomedicine to COVID-19 vaccines", Vaccines, 9, pp.359~ (2021)
41. Ray, KK, "Inclisiran in patients at high cardiovascular risk with elevated LDL cholesterol", N Engl J Med, 376, pp.1430~1440 (2017)
42. Kim, J, "Patient-customized oligonucleotide therapy for a rare genetic disease", N Engl J Med, 381, pp.1644~1652 (2019)
43. Liang, XH, "RNase H1-dependent antisense oligonucleotides are robustly active in directing RNA Cleavage in both the cytoplasm and the nucleus", Mol Ther, 25, pp.2075~2092 (2017)
44. Lennox, KA, "Cellular localization of long non-coding RNAs affects silencing by RNAi more than by antisense oligonucleotides", Nucleic Acids Res, 44, pp.863~877 (2016)
45. Carthew, RW, "Origins and Mechanisms of miRNAs and siRNAs", Cell, 136, pp.642~655 (2009)
46. Wong, E, "Mipomersen (kynamro): a novel antisense oligonucleotide inhibitor for the management of homozygous familial hypercholesterolemia", P T, 39, pp.119~122 (2014)
47. Chen, M, "Mechanisms of alternative splicing regulation: insights from molecular and genomics approaches", Nat Rev Mol Cell Biol, 10, pp.741~754 (2009)
48. Lee, Y, "Mechanisms and regulation of alternative Pre-mRNA splicing", Annu Rev Biochem, 84, pp.291~323 (2015)
49. Lunn, MR, "Spinal muscular atrophy", Lancet, 371, pp.2120~2133 (2008)
50. Wurster, CD, "Nusinersen for spinal muscular atrophy", Ther Adv Neurol Disord, 11, pp.1756285618754459~ (2018)
51. Nowak, KJ, "Duchenne muscular dystrophy and dystrophin: pathogenesis and opportunities for treatment", EMBO Rep, 5, pp.872~876 (2004)
52. Lim, KR, "Eteplirsen in the treatment of Duchenne muscular dystrophy", Drug Des Dev Ther, 11, pp.533~545 (2017)
53. Frank, DE, "Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy", Neurology, 94, pp.e2270~82 (2020)
54. Tabrizi, SJ, "Targeting Huntingtin expression in patients with Huntington’s disease", N Engl J Med, 380, pp.2307~2316 (2019)
55. Miller, T, "Phase 1-2 trial of antisense oligonucleotide Tofersen for SOD1 ALS", N Engl J Med, 383, pp.109~119 (2020)
56. Ha, M, "Regulation of microRNA biogenesis", Nat Rev Mol Cell Biol, 15, pp.509~524 (2014)
57. Adams, D, "Hereditary transthyretin amyloidosis: a model of medical progress for a fatal disease", Nat Rev Neurol, 15, pp.387~404 (2019)
58. Zhang, X, "Pharmacokinetics of Patisiran, the first approved RNA interference therapy in patients with hereditary transthyretin-mediated amyloidosis", J Clin Pharmacol, 60, pp.573~585 (2019)
59. Balwani, M, "Phase 3 trial of RNAi therapeutic Givosiran for acute intermittent Porphyria", N Engl J Med, 382, pp.2289~2301 (2020)
60. Anderson, KE, "Acute hepatic porphyrias: current diagnosis & management", Mol Genet Metab, 128, pp.219~227 (2019)
61. D’Souza, AA, "Asialoglycoprotein receptor mediated hepatocyte targeting - strategies and applications", J Control Rel, 203, pp.126~139 (2015)
62. Lamb, YN, "Inclisiran: first approval", Drugs, 81, pp.389~395 (2021)
63. Cariou, B, "Clinical aspects of PCSK9", Atherosclerosis, 216, pp.258~265 (2011)
64. Gragoudas, ES, "Pegaptanib for neovascular age-related macular degeneration", N Engl J Med, 351, pp.2805~2816 (2004)
65. Apte, RS, "VEGF in signaling and disease: beyond discovery and development", Cell, 176, pp.1248~1264 (2019)
66. Zhou, J, "Current progress of RNA aptamerbased therapeutics", Front Genet, 3, pp.234~ (2012)
67. Germer, K, "RNA aptamers and their therapeutic and diagnostic applications", Int J Biochem Mol Biol, 4, pp.27~40 (2013)
68. Jani, B., "In vitro transcription and capping of Gaussia luciferase mRNA followed by HeLa cell transfection", J Vis Exp (2012)[DOI]
69. Dalpke, A, "RNA mediated Toll-like receptor stimulation in health and disease", RNA Biol, 9, pp.828~842 (2012)
70. Kariko, K, "Suppression of RNA recognition by Toll-like receptors: the impact of nucleoside modification and the evolutionary origin of RNA", Immunity, 23, pp.165~175 (2005)
71. An, D, "Systemic messenger RNA therapy as a treatment for methylmalonic acidemia", Cell Re, 21, pp.3548~3558 (2017)
72. Pardi, N, "Recent advances in mRNA vaccine technology", Curr Opin Immunol, 65, pp.14~20 (2020)
73. Sahin, U, "An RNA vaccine drives immunity in checkpoint-inhibitor-treated melanoma", Nature, 585, pp.107~112 (2020)
74. Habtemariam, BA, "Single-Dose pharmacokinetics and pharmacodynamics of transthyretin targeting N-acetylgalactosamine-small interfering ribonucleic acid conjugate, vutrisiran, in healthy subjects", Clin Pharmacol Ther, 109, pp.372~382 (2021)
75. Ray, P, "The impact of nanoparticles on the immune system: a gray zone of nanomedicine", J Immunol Sci, 5, pp.19~33 (2021)
76. Haute, DV, "Challenges in realizing selectivity for nanoparticle biodistribution and clearance: lessons from gold nanoparticles", Ther Deliv, 8, pp.763~774 (2017)

관련 논문AI추천