Cited by 1,139RNA Interference and Gene DeliveryNanoparticle-Based Drug DeliveryRNA and protein synthesis mechanisms

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Overcoming cellular barriers for RNA therapeutics

Dowdy, Steven F·2017·Nature Biotechnology

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Abstract

RNA-based therapeutics, such as small-interfering (siRNAs), microRNAs (miRNAs), antisense oligonucleotides (ASOs), aptamers, synthetic mRNAs and CRISPR-Cas9, have great potential to target a large part of the currently undruggable genes and gene products and to generate entirely new therapeutic paradigms in disease, ranging from cancer to pandemic influenza to Alzheimer's disease. However, for these RNA modalities to reach their full potential, they first need to overcome a billion years of evolutionary defenses that have kept RNAs on the outside of cells from invading the inside of cells. Overcoming the lipid bilayer to deliver RNA into cells has remained the major problem to solve for widespread development of RNA therapeutics, but recent chemistry advances have begun to penetrate this evolutionary armor.

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Authors: Dowdy, S. F.
Affiliations: University of California San Diego (US)
Corresponding author: Dowdy, Steven F
ORCID: Dowdy, Steven F
Venue: Nature Biotechnology
Publisher: Springer Science and Business Media LLC
Published: 2017-02-27
Volume: 35
Issue: 3
Pages: 222-229
ISSN: 0733-222X (ISSN-L)
1087-0156
1546-1696
DOI: 10.1038/nbt.3802
OpenAlex: W2594133976
Cited by: 1,139
References: 105
Citations by year: 2026: 103 · 2025: 130 · 2024: 147 · 2023: 133 · 2022: 130 · 2021: 141 · 2020: 104 · 2019: 88 · 2018: 120 · 2017: 41
FWCI: 9.04
Type: journal article
Language: en
Open access: closed
Licence: www.springer.com/tdm (tdm)
www.springer.com/tdm (vor)
Locations: Nature Biotechnology (publishedVersion)
PubMed (publishedVersion)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA and protein synthesis mechanisms
Keywords: RNA therapeutics, RNA delivery, lipid bilayers, siRNA, microRNA, antisense oligonucleotides, aptamers, synthetic mRNA, CRISPR/Cas9
MeSH terms: RNAi Therapeutics, Animals, Cell Membrane, Humans, RNA, RNA Interference, Nanocapsules, Molecular Targeted Therapy

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