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Nanodelivery of nucleic acids
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There is growing need for a safe, efficient, specific and non-pathogenic means for delivery of gene therapy materials. Nanomaterials for nucleic acid delivery offer an unprecedented opportunity to overcome these drawbacks; owing to their tunability with diverse physico-chemical properties, they can readily be functionalized with any type of biomolecules/moieties for selective targeting. Nucleic acid therapeutics such as antisense DNA, mRNA, small interfering RNA (siRNA) or microRNA (miRNA) have been widely explored to modulate DNA or RNA expression Strikingly, gene therapies combined with nanoscale delivery systems have broadened the therapeutic and biomedical applications of these molecules, such as bioanalysis, gene silencing, protein replacement and vaccines. Here, we overview how to design smart nucleic acid delivery methods, which provide functionality and efficacy in the layout of molecular diagnostics and therapeutic systems. It is crucial to outline some of the general design considerations of nucleic acid delivery nanoparticles, their extraordinary properties and the structure-function relationships of these nanomaterials with biological systems and diseased cells and tissues.
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“Modulation of surface characteris tics may help to improve transduction and reduce the dose required, thereby reducing the risk of GTAU (Mendes et al., 2022; Carvalho et al., 2023).”
From Gene Therapy-Associated Uveitis (GTAU): Understanding and mitigating the adverse immune response in retinal gene therapy (2025) · cites this paper as Mendes et al., 2022
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Metadata
- Authors
- Mendes, B. B., Conniot, J., Avital, A., Yao, D., Jiang, X., Zhou, X., Sharf-Pauker, N., Xiao, Y., Adir, O., Liang, H., Shi, J., Schroeder, A., & Conde, J.
- Affiliations
- Ningbo University (CN)
- University of Science and Technology of China (CN)
- Brigham and Women's Hospital (US)
- Technion – Israel Institute of Technology (IL)
- Universidade Nova de Lisboa (PT)
- Corresponding author
- Liang, Haojun
- Shi, Jinjun
- Schroeder, Avi
- Conde, João
- ORCID
- Venue
- Nature Reviews Methods Primers
- Published
- 2022-04-14
- Volume
- 2
- Issue
- 1
- ISSN
- 2662-8449 (ISSN-L)
- PMC
- PMC9038125
- OpenAlex
- W4223968391
- Cited by
- 719
- References
- 228
- Citations by year
- 2026: 166 · 2025: 233 · 2024: 196 · 2023: 103 · 2022: 21
- FWCI
- 14.40
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- Nature Reviews Methods Primers (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Portuguese National Funding Agency for Science, Research and Technology (RCAAP Project by FCT) (submittedVersion, other-oa)
- Funders
- U.S. Department of Veterans Affairs (I01 BX004426)
- American Lung Association
- Massachusetts General Hospital
- National Natural Science Foundation of China (52003264, 21991132, 52033010, 2020YFA0710700, 52021002)
- National Institutes of Health
- Fundação para a Ciência e a Tecnologia (PTDC/BTM-MAT/4738/2020)
- Fundamental Research Funds for the Central Universities
- National Heart, Lung, and Blood Institute (R01HL159012, R01HL156362, R01HL137229, R01HL158097, R01HL162367)
- National Cancer Institute (R01CA200900)
- H2020 European Research Council (848325)
- DOD Peer Reviewed Cancer Research Program
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
- Physical Sciences › Materials Science › Polymers and Plastics › Dendrimers and Hyperbranched Polymers
- Keywords
- nucleic acid delivery, nanodelivery, gene therapy, nanoparticles, siRNA, microRNA, antisense DNA, mRNA therapeutics, targeted delivery, structure-function relationship, molecular diagnosis, gene silencing, vaccines, protein replacement
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