Progress on RNA-based therapeutics for genetic diseases
RNA therapeutics inhibit the expression of specific proteins/RNAs by targeting complementary sequences of corresponding genes or encode proteins for the synthesis desired genes to treat genetic diseases. RNA-based therapeutics are categorized as oligonucleotide drugs (antisense oligonucleotides, small interfering RNA, RNA aptamers), and mRNA drugs. The antisense oligonucleotides and small interfering RNA for treatment of genetic diseases have been approved by the FDA in the United States, while RNA aptamers and mRNA drugs are still in clinical trials. Chemical modifications can be applied to RNA drugs, such as pseudouridine modification of mRNA, to reduce immunogenicity and improve the efficacy. The secure and effective delivery systems such as lipid-based nanoparticles, extracellular vesicles, and virus-like particles are under development to address stability, specificity, and safety issues of RNA drugs. This article provides an overview of the specific molecular mechanisms of eleven RNA drugs currently used for treating genetic diseases, and discusses the research progress of chemical modifications and delivery systems of RNA drugs.
| Medienart: |
E-Artikel |
|---|
| Erscheinungsjahr: |
2023 |
|---|---|
| Erschienen: |
2023 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:52 |
|---|---|
| Enthalten in: |
Zhejiang da xue xue bao. Yi xue ban = Journal of Zhejiang University. Medical sciences - 52(2023), 4 vom: 25. Aug., Seite 406-416 |
| Sprache: |
Englisch |
|---|
| Weiterer Titel: |
治疗遗传病的RNA药物研究进展 |
|---|
| Beteiligte Personen: |
Luo, Ting [VerfasserIn] |
|---|
| Links: |
|---|
| Anmerkungen: |
Date Completed 31.08.2023 Date Revised 14.09.2023 published: Print Citation Status MEDLINE |
|---|
| doi: |
10.3724/zdxbyxb-2023-0190 |
|---|
| funding: |
|
|---|---|
| Förderinstitution / Projekttitel: |
|
| PPN (Katalog-ID): |
NLM361418817 |
|---|
| LEADER | 01000naa a22002652 4500 | ||
|---|---|---|---|
| 001 | NLM361418817 | ||
| 003 | DE-627 | ||
| 005 | 20231226085109.0 | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 231226s2023 xx |||||o 00| ||eng c | ||
| 024 | 7 | |a 10.3724/zdxbyxb-2023-0190 |2 doi | |
| 028 | 5 | 2 | |a pubmed24n1204.xml |
| 035 | |a (DE-627)NLM361418817 | ||
| 035 | |a (NLM)37643975 | ||
| 035 | |a (PII)1008-9292(2023)04-0406-11 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rakwb | ||
| 041 | |a eng | ||
| 100 | 1 | |a Luo, Ting |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Progress on RNA-based therapeutics for genetic diseases |
| 246 | 3 | 3 | |a 治疗遗传病的RNA药物研究进展 |
| 264 | 1 | |c 2023 | |
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a ƒaComputermedien |b c |2 rdamedia | ||
| 338 | |a ƒa Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Date Completed 31.08.2023 | ||
| 500 | |a Date Revised 14.09.2023 | ||
| 500 | |a published: Print | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a RNA therapeutics inhibit the expression of specific proteins/RNAs by targeting complementary sequences of corresponding genes or encode proteins for the synthesis desired genes to treat genetic diseases. RNA-based therapeutics are categorized as oligonucleotide drugs (antisense oligonucleotides, small interfering RNA, RNA aptamers), and mRNA drugs. The antisense oligonucleotides and small interfering RNA for treatment of genetic diseases have been approved by the FDA in the United States, while RNA aptamers and mRNA drugs are still in clinical trials. Chemical modifications can be applied to RNA drugs, such as pseudouridine modification of mRNA, to reduce immunogenicity and improve the efficacy. The secure and effective delivery systems such as lipid-based nanoparticles, extracellular vesicles, and virus-like particles are under development to address stability, specificity, and safety issues of RNA drugs. This article provides an overview of the specific molecular mechanisms of eleven RNA drugs currently used for treating genetic diseases, and discusses the research progress of chemical modifications and delivery systems of RNA drugs | ||
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a Antisense oligonucleotides | |
| 650 | 4 | |a Genetic disease | |
| 650 | 4 | |a Messenger RNA drugs | |
| 650 | 4 | |a RNA aptamers | |
| 650 | 4 | |a RNA, drug | |
| 650 | 4 | |a RNA, medicine | |
| 650 | 4 | |a Review | |
| 650 | 4 | |a Small interfering RNA | |
| 650 | 7 | |a Aptamers, Nucleotide |2 NLM | |
| 650 | 7 | |a RNA, Small Interfering |2 NLM | |
| 650 | 7 | |a RNA, Messenger |2 NLM | |
| 650 | 7 | |a Oligonucleotides, Antisense |2 NLM | |
| 700 | 1 | |a Huo, Chunxiao |e verfasserin |4 aut | |
| 700 | 1 | |a Zhou, Tianhua |e verfasserin |4 aut | |
| 700 | 1 | |a Xie, Shanshan |e verfasserin |4 aut | |
| 773 | 0 | 8 | |i Enthalten in |t Zhejiang da xue xue bao. Yi xue ban = Journal of Zhejiang University. Medical sciences |d 2002 |g 52(2023), 4 vom: 25. Aug., Seite 406-416 |w (DE-627)NLM123207037 |x 1008-9292 |7 nnns |
| 773 | 1 | 8 | |g volume:52 |g year:2023 |g number:4 |g day:25 |g month:08 |g pages:406-416 |
| 856 | 4 | 0 | |u http://dx.doi.org/10.3724/zdxbyxb-2023-0190 |3 Volltext |
| 912 | |a GBV_USEFLAG_A | ||
| 912 | |a GBV_NLM | ||
| 951 | |a AR | ||
| 952 | |d 52 |j 2023 |e 4 |b 25 |c 08 |h 406-416 | ||