Cited by 92RNA Interference and Gene DeliveryDNA and Nucleic Acid ChemistryRNA and protein synthesis mechanisms

Development of Therapeutic-Grade Small Interfering RNAs by Chemical Engineering

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Bramsen, Jesper B., Kjems, Jørgen·2012·Frontiers in Genetics

Abstract

Recent successes in clinical trials have provided important proof of concept that small interfering RNAs (siRNAs) indeed constitute a new promising class of therapeutics. Although great efforts are still needed to ensure efficient means of delivery in vivo, the siRNA molecule itself has been successfully engineered by chemical modification to meet initial challenges regarding specificity, stability, and immunogenicity. To date, a great wealth of siRNA architectures and types of chemical modification are available for promoting safe siRNA-mediated gene silencing in vivo and, consequently, the choice of design and modification types can be challenging to individual experimenters. Here we review the literature and devise how to improve siRNA performance by structural design and specific chemical modification to ensure potent and specific gene silencing without unwarranted side-effects and hereby complement the ongoing efforts to improve cell targeting and delivery by other carrier molecules.

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Authors
Bramsen, J. B., & Kjems, J.
Affiliations
Aarhus University (DK)
Corresponding author
Bramsen, Jesper B.
Venue
Frontiers in Genetics
Published
2012
Volume
3
Pages
154-154
ISSN
1664-8021 (ISSN-L)
OpenAlex
W1994555339
Cited by
92
References
266
Citations by year
2025: 3 · 2024: 4 · 2023: 4 · 2022: 6 · 2021: 13 · 2020: 4 · 2019: 11 · 2018: 8 · 2017: 3 · 2016: 9 · 2015: 10 · 2014: 7 · 2013: 9 · 2012: 1
FWCI
3.47
Type
journal article
Language
en
Open access
Licence
cc-by
Locations
  • Frontiers in Genetics (publishedVersion, cc-by)
  • Europe PMC (PubMed Central) (publishedVersion)
  • PubMed Central (publishedVersion)
  • PubMed (publishedVersion)
  • DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
Funders
  • Lundbeckfonden
  • H. Lundbeck A/S
Topics
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › DNA and Nucleic Acid Chemistry
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA and protein synthesis mechanisms
Keywords
siRNA, siRNA chemical modification, siRNA design, gene silencing, siRNA stability, siRNA specificity, siRNA delivery, siRNA therapeutics

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