Current status of development of oligonucleotide therapeutics
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- Inoue Takao
- National Institute of Health Sciences
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- Sasaki Kiyomi
- National Institute of Health Sciences
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- Yoshida Tokuyuki
- National Institute of Health Sciences
Bibliographic Information
- Other Title
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- 核酸医薬開発の現状と今後の展望
- カクサン イヤク カイハツ ノ ゲンジョウ ト コンゴ ノ テンボウ
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Abstract
Oligonucleotide therapeutics are single- or double-stranded DNA or RNA-based molecules consisting of short strand of nucleotides (generally 10-50 nucleotides). They are manufactured by chemical synthesis and act directly on RNA or protein. In recent years, oligonucleotide therapeutics, such as antisense oligonucleotides (ASOs), siRNAs, miRNAs, aptamers or CpG-motif oligodeoxynucleotides (CpG ODN), are active areas of drug development designed to treat a variety of genetic and/or intractable diseases. So far, eight oligonucleotide therapeutics have achieved marketing authorization in USA or Europe, i.e. fomivirsen (Vitravene®), pegaptanib (Macugen®), mipomersen (Kynamro®), eteplirsen (Exondys 51®), nusinersen (Spinraza®), inotersen (Tegsedi®), patisiran (Onpattro®), and hepatitis B vaccine containing CpG ODN (CpG1018) as adjuvants (HEPLISAV-B®), and more than 150 oligonucleotide therapeutics are currently in clinical development. In this review, I outline classification, modifications and mechanism of actions of oligonucleotide therapeutics, and introduce the current status of development of RNA-targeted oligonucleotide therapeutics (ASOs, siRNAs and miRNAs).
Journal
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- Drug Delivery System
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Drug Delivery System 34 (2), 86-98, 2019-03-25
THE JAPAN SOCIETY OF DRUG DELIVERY SYSTEM
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Details 詳細情報について
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- CRID
- 1390564238101182592
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- NII Article ID
- 130007666846
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- NII Book ID
- AN10084591
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- ISSN
- 18812732
- 09135006
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- NDL BIB ID
- 029592144
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- Text Lang
- ja
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- Data Source
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- JaLC
- NDL
- Crossref
- CiNii Articles
- KAKEN
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- Abstract License Flag
- Disallowed