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RNA therapy: Are we using the right molecules?

Yu, Ai-Ming ; Jian, Chao ; Yu, Allan H ; Tu, Mei-Juan

Pharmacology and Therapeutics, April 2019, Vol.196, pp.91-104 [Tạp chí có phản biện]

ISSN: 0163-7258 ; E-ISSN: 1879-016X ; DOI: 10.1016/j.pharmthera.2018.11.011

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  • Nhan đề:
    RNA therapy: Are we using the right molecules?
  • Tác giả: Yu, Ai-Ming ; Jian, Chao ; Yu, Allan H ; Tu, Mei-Juan
  • Chủ đề: Therapy ; Rnai ; Mirna ; Ncrna ; Biotechnology ; Cancer ; Pharmacy, Therapeutics, & Pharmacology
  • Là 1 phần của: Pharmacology and Therapeutics, April 2019, Vol.196, pp.91-104
  • Mô tả: Keywords Therapy; RNAi; miRNA; ncRNA; Biotechnology; Cancer Abstract Small-molecule and protein/antibody drugs mainly act on genome-derived proteins to exert pharmacological effects. RNA based therapies hold the promise to expand the range of druggable targets from proteins to RNAs and the genome, as evidenced by several RNA drugs approved for clinical practice and many others under active trials. While chemo-engineered RNA mimics have found their success in marketed drugs and continue dominating basic research and drug development, these molecules are usually conjugated with extensive and various modifications. This makes them completely different from cellular RNAs transcribed from the genome that usually consist of unmodified ribonucleotides or just contain a few posttranscriptional modifications. The use of synthetic RNA mimics for RNA research and drug development is also in contrast with the ultimate success of protein research and therapy utilizing biologic or recombinant proteins produced and folded in living cells instead of polypeptides or proteins synthesized in vitro. Indeed, efforts have been made recently to develop RNA bioengineering technologies for cost-effective and large-scale production of biologic RNA molecules that may better capture the structures, functions, and safety profiles of natural RNAs. In this article, we provide an overview on RNA therapeutics for the treatment of human diseases via RNA interference mechanisms. By illustrating the structural differences between natural RNAs and chemo-engineered RNA mimics, we focus on discussion of a novel class of bioengineered/biologic RNA agents produced through fermentation and their potential applications to RNA research and drug development. Abbreviations A, adenine; ASO, antisense oligonucleotide; asRNA, antisense RNA; BERA, bioengineered/biologic RNA agents; C, cytosine; CMV, cytomegalovirus; dsRNA, double-stranded RNA; FPLC, fast protein liquid chromatography; FDA, Food and Drug Administration; G, guanine; GalNAc, N-acetylgalactosamine; GFP, green fluorescent protein; gRNA, guide RNA; HPLC, high performance liquid chromatography; lncRNA, long noncoding RNA; MGA, malachite green aptamer; miRNA, microRNA; ncRNA, noncoding RNA; NSCLC, non-small cell lung cancer; [PSI], pseudouridine; PMO, phosphorodiamidate morpholine oligonucleotide; PNA, peptide nucleic acid; pre-miRNA, precursor miRNA; pri-miRNA, primary miRNA; PS, phosphorothioate; RNAi, RNA interference; RNase, ribonuclease; rRNA, ribosomal RNA; shRNA, short-hairpin RNA; siRNA, small interfering RNA; sRNA, small RNA; T, thymine; tRNA, transfer RNA; U, uracil; VEGF, vascular endothelial growth factor Author Affiliation: Department of Biochemistry & Molecular Medicine, UC Davis School of Medicine, Sacramento, CA 95817, USA * Corresponding author at: Department of Biochemistry & Molecular Medicine, UC Davis School of Medicine, 2700, Stockton Blvd., Suite 2132, Sacramento, CA 95817, USA. Byline: Ai-Ming Yu [aimyu@ucdavis.edu] (*), Chao Jian, Allan H. Yu, Mei-Juan Tu
  • Ngôn ngữ: English
  • Số nhận dạng: ISSN: 0163-7258 ; E-ISSN: 1879-016X ; DOI: 10.1016/j.pharmthera.2018.11.011

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