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Progress in clinical pharmacology of oligonucleotides
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Xian-ge TANG, Xiao-duo GUAN, Rui CHEN*, Pei HU*
Acta Pharmaceutica Sinica | 2020, 55(2) : 218 - 225
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Acta Pharmaceutica Sinica | 2020, 55(2): 218-225
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Progress in clinical pharmacology of oligonucleotides
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Xian-ge TANG, Xiao-duo GUAN, Rui CHEN*, Pei HU*
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  • Clinical Pharmacology Research Center, Peking Union Medical College Hospital, Beijing Key Laboratory of Clinical PK and PD Investigation for Innovative Drugs, Beijing 100032, China
Published: 2020-02-12 doi: 10.16438/j.0513-4870.2019-0834
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Following small molecules and monoclonal antibodies, oligonucleotides are expected to overcome the rare and refractory human diseases. It has been attracted the attention of the pharmaceutical industry since the approval of six oligonucleotides in recent years because of their unique mechanism of regulating disease gene transcription at the RNA level. As a new class of drug molecules, oligonucleotides are highly polar, charged, and need to be improved by means of chemical modification and drug delivery systems. And therefore, they have different clinical pharmacology properties compared with chemical molecules and monoclonal antibodies, which pose new challenges for early clinical development. This paper reviews the characteristics of oligonucleotides from the perspective of technological development, mechanism of action, human pharmacokinetics, efficacy and safety.

oligonucleotides  /  chemical modification  /  drug delivery  /  pharmacokinetics  /  efficacy  /  safety
Xian-ge TANG, Xiao-duo GUAN, Rui CHEN, Pei HU. Progress in clinical pharmacology of oligonucleotides[J]. Acta Pharmaceutica Sinica, 2020 , 55 (2) : 218 -225 . DOI: 10.16438/j.0513-4870.2019-0834
Year 2020 volume 55 Issue 2
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Article Info
doi: 10.16438/j.0513-4870.2019-0834
  • Receive Date:2019-10-23
  • Online Date:2026-01-20
  • Published:2020-02-12
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  • Received:2019-10-23
  • Revised:2019-12-17
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    Clinical Pharmacology Research Center, Peking Union Medical College Hospital, Beijing Key Laboratory of Clinical PK and PD Investigation for Innovative Drugs, Beijing 100032, China
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表12种不同金属材料的力学参数

Family
属数
Number of
genus
种数
Number of
species
占总种数比例
Percentage of
total species (%)

Genus
种数
Number of
species
占总种数比例
Percentage of total
species (%)
鹅膏菌科Amanitaceae 2 11 5.26 鹅膏菌属 Amanita 10 4.78
小菇科 Mycenaceae 2 12 5.74 丝盖伞属 Inocybe 5 2.39
多孔菌科 Polyporaceae 8 14 6.70 蜡蘑属 Laccaria 5 2.39
红菇科 Russulaceae 3 23 11.00 小皮伞属 Marasmius 6 2.87
小菇属 Mycena 11 5.26
光柄菇属 Pluteus 5 2.39
红菇属 Russula 17 8.13
栓菌属 Trametes 5 2.39
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