Nanodelivery of nucleic acids

Mendes, Bárbara B.; Conniot, João; Avital, Aviram; Yao, Dongbao; Jiang, Xingya; Zhou, Xiang; Sharf-Pauker, Noga; Xiao, Yuling; Adir, Omer; Liang, Haojun; Shi, Jinjun; Schroeder, Avi; Conde, João

doi:10.1038/s43586-022-00104-y

Cited by 719RNA Interference and Gene DeliveryNanoparticle-Based Drug DeliveryDendrimers and Hyperbranched Polymers

Nanodelivery of nucleic acids

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Mendes, Bárbara B., Conniot, João, Avital, Aviram, Yao, Dongbao, Jiang, Xingya, Zhou, Xiang, Sharf-Pauker, Noga, Xiao, Yuling +5 more·2022·Nature Reviews Methods Primers

DOI 10.1038/s43586-022-00104-yPMC PMC9038125

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Abstract

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 characteristics may help to improve transduction and reduce the dose required, thereby reducing the risk of GTAU.
“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
The Targeting section (page unavailable) says surface chemistry and surface-conjugated ligands can increase nanomaterials’ affinity for specific cells or tissues, supporting the delivery-improvement part of the claim. The cited passages do not establish that such changes reduce the required dose or the risk of GTAU; searches for uveitis, dose reduction, and surface modification did not find that link in our copy. (LLM, conf=0.84)
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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: Mendes, Bárbara B.
Conniot, João
Avital, Aviram
Yao, Dongbao
Jiang, Xingya
Zhou, Xiang
Xiao, Yuling
Adir, Omer
Liang, Haojun
Shi, Jinjun
Schroeder, Avi
Conde, João
Venue: Nature Reviews Methods Primers
Publisher: Springer Science and Business Media LLC
Published: 2022-04-14
Volume: 2
Issue: 1
ISSN: 2662-8449 (ISSN-L)
DOI: 10.1038/s43586-022-00104-y
PMC: PMC9038125
OpenAlex: W4223968391
Cited by: 719
References: 230
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
Licence: www.springer.com/tdm (tdm)
www.springer.com/tdm (vor)
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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