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Going beyond the liver: Progress and challenges of targeted delivery of siRNA therapeutics
Open accessAbstract
Therapeutic gene silencing promises significant progress in pharmacotherapy, including considerable expansion of the druggable target space and the possibility for treating orphan diseases. Technological hurdles have complicated the efficient use of therapeutic oligonucleotides, and siRNA agents suffer particularly from insufficient pharmacokinetic properties and poor cellular uptake. Intense development and evolution of delivery systems have resulted in efficient uptake predominantly in liver tissue, in which practically all nanoparticulate and liposomal delivery systems show the highest accumulation. The most efficacious strategies include liposomes and bioconjugations with N-acetylgalactosamine. Both are in early clinical evaluation stages for treatment of liver-associated diseases. Approaches for achieving knockdown in other tissues and tumors have been proven to be more complicated. Selective targeting to tumors may be enabled through careful modulation of physical properties, such as particle size, or by taking advantage of specific targeting ligands. Significant barriers stand between sufficient accumulation in other organs, including endothelial barriers, cellular membranes, and the endosome. The brain, which is shielded by the blood-brain barrier, is of particular interest to facilitate efficient oligonucleotide therapy of neurological diseases. Transcytosis of the blood-brain barrier through receptor-specific docking is investigated to increase accumulation in the central nervous system. In this review, the current clinical status of siRNA therapeutics is summarized, as well as innovative and promising preclinical concepts employing tissue- and tumor-targeted ligands. The requirements and the respective advantages and drawbacks of bioconjugates and ligand-decorated lipid or polymeric particles are discussed.
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“Strategies that have been applied for achieving functional siRNA delivery into the brain mainly rely on employing potent protein or peptide ligands to target the receptors, such as transferrin, insulin and LDL receptors, which are highly expressed on the blood brain barrier (BBB).[102]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [102]
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Metadata
- Authors
- Lorenzer, C., Dirin, M., Winkler, A.-M., Baumann, V., & Winkler, J.
- Affiliations
- University of Vienna (AT)
- Corresponding author
- Winkler, Johannes
- ORCID
- Winkler, Johannes
- Venue
- Journal of Controlled Release
- Publisher
- Elsevier BV
- Published
- 2015-04
- Volume
- 203
- Pages
- 1-15
- ISSN
- 0168-3659 (ISSN-L)
- OpenAlex
- W2019613864
- Cited by
- 284
- References
- 188
- Citations by year
- 2026: 7 · 2025: 16 · 2024: 17 · 2023: 17 · 2022: 22 · 2021: 25 · 2020: 23 · 2019: 35 · 2018: 34 · 2017: 33 · 2016: 37 · 2015: 18
- FWCI
- 3.94
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Locations
- Journal of Controlled Release (publishedVersion, cc-by)
- PubMed (publishedVersion)
- Funders
- FWF (I519-B11)
- Innovative Medicines Initiative Joint Undertaking (115363)
- European Union's (FP7/2007–2013)
- EFPIA companies'
- 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 › Pharmacology, Toxicology and Pharmaceutics › Pharmaceutical Science › Advanced Drug Delivery Systems
- Keywords
- siRNA therapeutics, targeted delivery, gene silencing, liposomes, nanoparticle drug delivery, tumor targeting, blood-brain barrier, transcytosis, cellular uptake, pharmacokinetics, oligonucleotide therapeutics, liver-targeted drug delivery
- MeSH terms
- RNAi Therapeutics, Animals, Blood-Brain Barrier, Endothelium, Humans, Leukocytes, Liposomes, Liver, Models, Molecular, Neoplasms, Drug Delivery Systems, Gene Transfer Techniques, RNA, Small Interfering, Nanoparticles
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
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