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RNA therapy: Are we using the right molecules?

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

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

E-ISSN: 1879-016X ; PMID: 30521885 Version:1 ; 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: Biotechnology ; Cancer ; Rnai ; Therapy ; Mirna ; Ncrna ; RNA Interference ; RNA -- Therapeutic Use
  • Là 1 phần của: Pharmacology & therapeutics, April 2019, Vol.196, pp.91-104
  • Mô tả: 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...
  • Ngôn ngữ: English
  • Số nhận dạng: E-ISSN: 1879-016X ; PMID: 30521885 Version:1 ; DOI: 10.1016/j.pharmthera.2018.11.011

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