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Modulating intracellular pathways to improve non-viral delivery of RNA therapeutics

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Advances in lipid nanoparticle mRNA therapeutics beyond COVID-19 vaccines

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Bundling of mRNA strands inside polyion complexes improves mRNA delivery efficiency in vitro and in vivo

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Comprehensive analysis of lipid nanoparticle formulation and preparation for RNA delivery

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Delivery of RNAs to Specific Organs by Lipid Nanoparticles for Gene Therapy

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Cellular Trafficking of Nanotechnology‐Mediated mRNA Delivery

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Advances in functional lipid nanoparticles: from drug delivery platforms to clinical applications

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CNS Delivery of Nucleic Acid Therapeutics: Beyond the Blood–Brain Barrier and Towards Specific Cellular Targeting

D’souza, A. A., Nozohouri, S., Bleier, B. S., & Amiji, M. M. (2022) · Pharmaceutical Research · doi:10.1007/s11095-022-03433-5 · Cited by 47 · see citing works

Delivery and Expression of mRNA in the Secondary Lymphoid Organs Drive Immune Responses to Lipid Nanoparticle-mRNA Vaccines after Intramuscular Injection

Takanashi, A., Pouton, C. W., & Al-Wassiti, H. A. (2023) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.2c01024 · Cited by 47 · see citing works

Treatment of Intervertebral Disk Disease by the Administration of mRNA Encoding a Cartilage-Anabolic Transcription Factor

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Lipid nanoparticle (LNP) mediated mRNA delivery in neurodegenerative diseases

Moosavi, S. G., Rahiman, N., Jaafari, M. R., & Arabi, L. (2025) · Journal of Controlled Release · doi:10.1016/j.jconrel.2025.113641 · Cited by 52 · see citing works

Fluorescence Correlation Spectroscopy to find the critical balance between extracellular association and intracellular dissociation of mRNA complexes

Zhang, H., Smedt, S. C. D., & Remaut, K. (2018) · Acta Biomaterialia · doi:10.1016/j.actbio.2018.05.016 · Cited by 46 · see citing works

Therapeutic Prospects for Friedreich’s Ataxia

Zhang, S., Напиерала, М., & Napierala, J. S. (2019) · Trends in Pharmacological Sciences · doi:10.1016/j.tips.2019.02.001 · Cited by 45 · see citing works

Multifaceted nanoparticles: emerging mechanisms and therapies in neurodegenerative diseases

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Engineered mRNA Delivery Systems for Biomedical Applications

Wang, J., Zhu, H., Gan, J., Liang, G., Li, L., & Zhao, Y. (2023) · Advanced Materials · doi:10.1002/adma.202308029 · Cited by 44 · see citing works

Delivery of synthetic mRNAs for tissue regeneration

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Nonviral nanoparticle gene delivery into the <scp>CNS</scp> for neurological disorders and brain cancer applications

Yang, J., Luly, K. M., & Green, J. J. (2023) · WIREs Nanomedicine and Nanobiotechnology · doi:10.1002/wnan.1853 · Cited by 40 · see citing works

Correction of half the cardiomyocytes fully rescue Friedreich ataxia mitochondrial cardiomyopathy through cell-autonomous mechanisms

Belbellaa, B., Reutenauer, L., Monassier, L., & Puccio, H. M. (2018) · Human Molecular Genetics · doi:10.1093/hmg/ddy427 · Cited by 40 · see citing works

Lipid based drug delivery systems for oral, transdermal and parenteral delivery: Recent strategies for targeted delivery consistent with different clinical application

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Modified mRNA as a therapeutic tool to induce cardiac regeneration in ischemic heart disease

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Property-Driven Design and Development of Lipids for Efficient Delivery of siRNA

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Data-balanced transformer for accelerated ionizable lipid nanoparticles screening in mRNA delivery

Wu, K., Yang, X., Wang, Z., Li, N., Zhang, J., & Liu, L. (2024) · Briefings in Bioinformatics · doi:10.1093/bib/bbae186 · Cited by 36 · see citing works

An overview of lipid constituents in lipid nanoparticle mRNA delivery systems

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mRNA delivery via non-viral carriers for biomedical applications

Shuai, Q., Zhu, F., Zhao, M., & Yan, Y. (2021) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2021.121020 · Cited by 35 · see citing works

Targeting and tracking mRNA lipid nanoparticles at the particle, transcript and protein level

Kang, D. D., Marks, A. R., Morlà‐Folch, J., Dong, Y., Brown, B. D., & Teunissen, A. J. P. (2025) · Nature Biomedical Engineering · doi:10.1038/s41551-025-01511-8 · Cited by 37 · see citing works

Targeted Therapy of Osteoarthritis via Intra‐Articular Delivery of Lipid‐Nanoparticle‐Encapsulated Recombinant Human FGF18 mRNA

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mRNA-based modalities for infectious disease management

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Next-generation materials for RNA–lipid nanoparticles: lyophilization and targeted transfection

Wang, T., Sung, T., Yu, T., Lin, H.-Y., Chen, Y.-H., Zhu, Z.-W., Gong, J., Pan, J., & Higuchi, A. (2023) · Journal of Materials Chemistry B · doi:10.1039/d3tb00308f · Cited by 29 · see citing works

Intrathecal delivery of Macromolecules: Clinical status and emerging technologies

Ajeeb, R., & Clegg, J. R. (2023) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2023.114949 · Cited by 30 · see citing works

mRNA as a Novel Treatment Strategy for Hereditary Spastic Paraplegia Type 5

Hauser, S., Poenisch, M., Schelling, Y., Höflinger, P., Schuster, S., Teegler, A., Betten, R., Gustafsson, J.-Å., Hübener‐Schmid, J., Schlake, T., et al. (2019) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2019.10.011 · Cited by 28 · see citing works

Recessive cerebellar and afferent ataxias — clinical challenges and future directions

Beaudin, M., Manto, M. U., Schmahmann, J. D., Pandolfo, M., & Dupré, N. (2022) · Nature Reviews Neurology · doi:10.1038/s41582-022-00634-9 · Cited by 29 · see citing works

Use, exposure and omics characterisation of potential hazard in nanomaterials

Ling, N., Zhang, N., Yong, W. T. L., Misbah, S., Hashim, F., Soon, C. F., Lim, G. P., Peng, W., & Sonne, C. (2023) · Materials Today Advances · doi:10.1016/j.mtadv.2023.100341 · Cited by 31 · see citing works

The Yin and Yang of nucleic acid-based therapy in the brain

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Fabry Cardiomyopathy: Current Practice and Future Directions

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Insights into the formulation of lipid nanoparticles for the optimization of mRNA therapeutics

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Synthesis and bioactivity of readily hydrolysable novel cationic lipids for potential lung delivery application of mRNAs

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Current Status of Messenger RNA Delivery Systems

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Lipid nanoparticles deliver mRNA to the blood–brain barrier

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mRNA delivery technologies: Toward clinical translation

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Adjuvants for cancer mRNA vaccines in the era of nanotechnology: strategies, applications, and future directions

Cao, L., Yu, Y.-F., Li, Z., Zhong, N., Wang, G.-R., Xiao, Y., Liu, B., Wu, Q., Feng, C., & Bu, L. (2024) · Journal of Nanobiotechnology · doi:10.1186/s12951-024-02590-6 · Cited by 23 · see citing works

Polymeric nanoparticles decorated with BDNF-derived peptide for neuron-targeted delivery of PTEN inhibitor

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Hereditary Ataxia: A Focus on Heme Metabolism and Fe-S Cluster Biogenesis

Chiabrando, D., Bertino, F., & Tolosano, E. (2020) · International Journal of Molecular Sciences · doi:10.3390/ijms21113760 · Cited by 23 · see citing works

Advancing mRNA Therapeutics: The Role and Future of Nanoparticle Delivery Systems

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mRNA-Based Approaches to Treating Liver Diseases

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Targeted Oligonucleotides for Treating Neurodegenerative Tandem Repeat Diseases

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Overexpression of Drosophila frataxin triggers cell death in an iron-dependent manner

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Topical Delivery of Four Neuroprotective Ingredients by Ethosome-Gel: Synergistic Combination for Treatment of Oxaliplatin-Induced Peripheral Neuropathy

Lin, H., Lin, L., Sun, M., Liu, J., & Wu, Q. (2020) · International Journal of Nanomedicine · doi:10.2147/ijn.s233747 · Cited by 21 · see citing works

Material Design for Next-Generation mRNA Vaccines Using Lipid Nanoparticles

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Unleashing the potential of mRNA therapeutics for inherited neurological diseases

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Current knowledge on the tissue distribution of mRNA nanocarriers for therapeutic protein expression

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Ionic liquid-coated lipid nanoparticles increase siRNA uptake into CNS targets

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Emerging therapeutics for the treatment of Friedreich’s ataxia

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Nucleic acid immunotherapeutics and vaccines: A promising approach to glioblastoma multiforme treatment

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Frataxin mRNA Replacement in Friedreich Ataxia: An Evidence-Gated Translational Framework Integrating Molecular Evidence, Reproducible Computational Analyses, and Conditional Virtual-Trial Simulations

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Particle-mediated delivery of frataxin plasmid to a human sensory neuronal model of Friedreich's ataxia

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Sustained FXN expression in dorsal root ganglia from a nonreplicative genomic HSV‐1 vector

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Potential RNA therapeutics for Friedreich’s ataxia

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Therapeutic activity of a hematopoietic stem cell-delivered cell-penetrating frataxin in Friedreich’s ataxia models

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