Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms

Akinc, Akin, Querbes, William, De, Soma, Qin, June, Frank-Kamenetsky, Maria, Jayaprakash, K. Narayanannair, Jayaraman, Muthusamy, Rajeev, Kallanthottathil G +17 more·2010·Molecular Therapy

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    Britt Delnoy, Minela Haskovic, Jo M. Vanoevelen, Laura K.M. Steinbusch, Esther Naomi Vos, Kèvin Knoops, Luc J. I. Zimmermann, Marek J. Noga, Dirk J. Lefeber, Paolo G.V. Martini et al. · 2022 · Journal of Inherited Metabolic Disease
  2. Plasma and Liver Pharmacokinetics of the N-Acetylgalactosamine Short Interfering RNA JNJ-73763989 in Recombinant Adeno-Associated–Hepatitis B Virus–Infected Mice
    Louis Sandra, Huybrecht T’jollyn, Nele Goeyvaerts, An Vermeulen, Anne‐Gaëlle Dosne, Juan José Pérez Ruixo · 2022 · Journal of Pharmacology and Experimental Therapeutics
  3. Plug-and-play assembly of biodegradable ionizable lipids for potent mRNA delivery and gene editing in vivo
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  4. Increasing polycation hydrophobicity in mRNA polyplex vaccines enhances the efficacy of humoral and cellular immunity induction
    Nan Qiao, Mao Hori, Mitsuru Naito, Heemin Chang, Satomi Ogura, Mika Suzuki, Tsuyoshi Kimura, Shigeto Fukushima, Hyun Jin Kim, Satoshi Uchida et al. · 2025 · Biomaterials
  5. Advances and Strategies in Enhancing mRNA Cancer Vaccines
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  6. Unleashing CAR-T potential in solid tumors: overcoming intrinsic and extrinsic hurdles to improve therapy
    Zhiwei Zhang, Dawei Cui, Hao Wang, Liming Wu, Xia Liu · 2026 · Cancer Immunology Immunotherapy
  7. Nanotechnology in Cancer Therapy: How Nanoparticles Are Shaping the Future of Personalized Treatment
    Indrianita Lionadi, Amir Farokh Payam · 2026 · ACS Nano Medicine
  8. Navigating the Biomolecular Corona: Biological Interactions and Therapeutic Implications of RNA–Lipid Nanoparticles
    Anamika Saini, Shiyao Li, Zhaoran Wang, Magdalena Plebanski, Yi Ju · 2026 · ACS Nano
  9. Repositioning the antihistamine ebastine as an intracellular siRNA delivery enhancer
    Cristina Muntean, Eva Blondeel, L. Harinck, Kunal P. Pednekar, Jai Prakash, Olivier De Wever, Jeanne Leblond Chain, Stefaan C. De Smedt, Katrien Remaut, Koen Raemdonck · 2023 · International Journal of Pharmaceutics
  10. Considerations on the Design of Lipid-based mRNA Vaccines Against Cancer
    Sofie Meulewaeter, Yao Zhang, Abishek Wadhwa, Kevin Fox, Ine Lentacker, Kenneth W. Harder, Pieter R. Cullis, Stefaan C. De Smedt, Miffy H. Y. Cheng, Rein Verbeke · 2023 · Journal of Molecular Biology
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    Sizhen Wang, Beibei Guo, Huan Wang, Feng Yang · 2024 · Journal of Drug Delivery Science and Technology
  12. DNAJB1-PRKACA fusion protein-regulated LINC00473 promotes tumor growth and alters mitochondrial fitness in fibrolamellar carcinoma
    Rosanna K. Ma, Pei-Yin Tsai, Alaa R. Farghli, Alexandria J. Shumway, Matt Kanke, John Dozier Gordan, Taranjit S. Gujral, Khashayar Vakili, Manabu Nukaya, Leila J. Noetzli et al. · 2024 · PLoS Genetics
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  14. Engineering Lipid Nanoparticles to Enhance Intracellular Delivery of Transforming Growth Factor-Beta siRNA (siTGF-β1) via Inhalation for Improving Pulmonary Fibrosis Post-Bleomycin Challenge
    Xu Deng, Yingjie Yang, Li‐Ming Gan, Xinliu Duan, Xiwei Wang, Jingyan Zhang, Aiping Wang, An-An Zhang, Zhizhao Yuan, Daquan Chen et al. · 2025 · Pharmaceutics
  15. Chirality-Controlled Lipid Nanoparticles for mRNA Delivery
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  16. A Molecular View of Lipid Nanoparticles: Insights into their Morphology and Structural Plasticity
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  17. Tissue-specific gene silencing monitored in circulating RNA
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  18. Development of siRNA Therapeutics for the Treatment of Liver Diseases
    Anja Holm, Marianne Bengtson Løvendorf, Sakari Kauppinen · 2021 · Methods in molecular biology
  19. Effect of Nanoparticle Synthetic Conditions on Ligand Coating Integrity and Subsequent Nano-Biointeractions
    Jessica C. Hsu, Yu Mei Du, Arjun Sengupta, Yuxi C. Dong, Katherine J. Mossburg, Mathilde Bouché, Andrew D. A. Maidment, Aalim M. Weljie, David P. Cormode · 2021 · ACS Applied Materials & Interfaces
  20. Non-viral nucleic acid therapeutics: Revolutionizing the landscape of atherosclerotic treatment
    Shuai Qu, Renfa Liu, Nisi Zhang, Yunxue Xu, Xiuli Yue, Zhifei Dai · 2022 · Nano Today
  21. Chemically modified in-vitro-transcribed mRNA encoding thrombopoietin stimulates thrombopoiesis in mice
    Yu Zhang, Xiaodong Xi, Hang Yu, Liuyan Yang, Jinzhong Lin, Wen Yang, Junling Liu, Xuemei Fan, Yingjie Xu · 2022 · Molecular Therapy — Nucleic Acids
  22. The development of highly dense highly protected surfactant ionizable lipid RNA loaded nanoparticles
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  28. Harnessing antiviral RNAi therapeutics for pandemic viruses: SARS-CoV-2 and HIV
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  30. New Approach to Develop Ultra-High Inhibitory Drug Using the Power Function of the Stoichiometry of the Targeted Nanomachine or Biocomplex
    Dan Shu, Fengmei Pi, Chi Wang, Peng Zhang, Peixuan Guo · 2015 · Nanomedicine
  31. Multifunctional Enveloped Nanodevices (MENDs)
    Yusuke Sato, Takashi Nakamura, Yuma Yamada, Hidetaka Akita, Hideyoshi Harashima · 2014 · Advances in genetics
  32. Emerging Therapies for Transthyretin Cardiac Amyloidosis
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  33. Sterol-modified PEG lipids: alteration of the bilayer anchoring moiety has an unexpected effect on liposome circulation
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  34. Involvement of Caveolin-1-mediated transcytosis in the intratumoral accumulation of liposomes
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  35. Extension in the approaches to treat cancer through siRNA system: a beacon of hope in cancer therapy
    Indra Rautela, Aditi Sharma, Pallavi Dheer, Priya Thapliyal, Shweta Sahni, Vimlendu Bhushan Sinha, Manish Dev Sharma · 2021 · Drug Delivery and Translational Research
  36. Bispecific antibody targeting of lipid nanoparticles
    Angelo Amabile, Matthew Phelan, Zhe Yu, Pedro Silva, Adam R. Marks, Judit Morlà‐Folch, Moah Sohn, Gürkan Mollaoglu, Chiara Falcomatà, Abraham J. P. Teunissen et al. · 2024 · bioRxiv (Cold Spring Harbor Laboratory)
  37. Surface Plasmon Resonance Based Binding Characterization for Screening RNA-Loaded Lipid Nanoparticles (LNPs): Exploring Species Cross-Reactivity in LNP–Apolipoprotein E Interactions
    Benjamin Lew, Sandeep Chhabra, Jacob A. Lewis, Angela C. Wagoner, Angel Hsu, Steve L. Halaby, Pooja Sharma, Justin K. Murray, Francis S. Kinderman, Deirdre Murphy Piedmonte et al. · 2025 · Molecular Pharmaceutics
  38. Endogenous Targeting of Lipid Nanoparticles to Kidney Tumors
    Amogh Vaidya, Yun‐Chieh Sung, Vanina Toffessi Tcheuyap, Cheng Cheng Zhang, Ramesh Butti, Arijit Mal, Sean A. Dilliard, Jing Liu, Eunice Song, Erick Damian Guerrero et al. · 2025 · ACS Nano
  39. Programmable lipid nanoparticles for RNA therapeutics: Design principles and clinical translation
    Mahdi Navid Talemi, Marzieh Ramezani Farani, Naiyereh Alipour Eskandani, Danial Mirzaee, Iraj Alipourfard, Yun Suk Huh · 2026 · Materials Today Bio
  40. Novel Lipid Nanoparticle (LNP) Delivery Systems Enabling the Advancement of RNA Therapeutics
    Honglei Zhang, Shaowei Han, Xueyan Zhang, Jingxuan Ma, Lijie Jin, Yanfeng Wang, Yuqing Ma, Teng Ma, Fei Yu, Gengshen Song · 2026 · Advanced Healthcare Materials
  41. A multiobjective AI model for LNP engineering enhances tissue-selective mRNA delivery
    Muye Zhou, Y Xu, Gen Li, Jingan Chen, Margarita Savguira, Breanna Y. Seto, Fanglin Gong, Tyler Thomson, Juan Chen, Rick Xing Ze Lu et al. · 2026 · Nature Biotechnology
  42. Controlling the protein corona of polymeric nanocapsules: effect of polymer shell on protein adsorption
    Germán Berrecoso, Susana B. Bravo, Iker Arriaga, Nicola G. A. Abrescia, José Crecente‐Campo, Marı́a José Alonso · 2023 · Drug Delivery and Translational Research
  43. Impact of protein corona and PEGylation on the accumulation and efficacy of lysosome-disrupting lipid nanoparticles in KRAS-mutated cancer
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  44. A novel multitargeted self-assembling peptide-siRNA complex for simultaneous inhibition of SARS-CoV-2-host cell interaction and replication
    Martina Tuttolomondo, Stephanie Thuy Duong Pham, Mikkel G. Terp, Virginia Cendán Castillo, Nazmie Kalisi, Stefan Vogel, Niels Langkjær, Ulla Melchior Hansen, Helge Thisgaard, Henrik Daa Schrøder et al. · 2024 · Molecular Therapy — Nucleic Acids
  45. The absorption, distribution, metabolism and elimination characteristics of small interfering RNA therapeutics and the opportunity to predict disposition in pregnant women
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  46. Roadmap to discovery and early development of an mRNA loaded LNP formulation for liver therapeutic genome editing
    Annette Bak, Liping Zhou, Joanna Rejman, Marianna Yanez Arteta, Gunilla Åkesson Nilsson, Marianne B. Ashford · 2025 · Expert Opinion on Drug Delivery
  47. Lipid Nanoparticles for mRNA Delivery in Cancer Immunotherapy
    Yasir Alshehry, Xiang Liu, Wenhua Li, Qiyan Wang, Janét Cole, Guizhi Zhu · 2025 · The AAPS Journal
  48. Dimethylamino-based synthetic lipidoid nanoparticles for selective mRNA delivery to splenic antigen-presenting cells
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  49. In vivo demonstration of enhanced mRNA delivery by cyclic disulfide-containing lipid nanoparticles for facilitating endosomal escape
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  50. Pharmacometric modeling of lipid nanoparticle-encapsulated mRNA therapeutics and vaccines: A systematic review
    Miao Zhang, Linh M. Van, Mansoor M. Amiji · 2025 · Molecular Therapy — Nucleic Acids
  51. Providing The Full Picture: A Mandate for Standardizing Nanoparticle-Based Drug Delivery
    Kenneth Alan Howard, Dan Peer · 2013 · Nanomedicine
  52. The Possible Uses and Challenges of Nanomaterials in Mast Cell Research
    Brett A. Duguay, Lei Lu, Narcy G Arizmendi, Larry D. Unsworth, Marianna Kulka · 2020 · The Journal of Immunology
  53. Messenger RNA as a personalized therapy: The moment of truth for rare metabolic diseases
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  56. Targeted Lipid Nanoparticle Delivery of FAP-CAR mRNA Enables Potent In Vivo T-cell Engineering against Pancreatic Tumors
    Khuloud Bajbouj, Zebin Xiao, Leslie Todd, Li Huang, Tyler Ellis Papp, Faris Halilovic, Jayalakshmi Ramani, Yanjie Bao, Matthew Butcher, Adrian Bot et al. · 2026 · Cancer Immunology Research
  57. Engineering branched ionizable lipid for hepatic delivery of clustered regularly interspaced short palindromic repeat-Cas9 ribonucleoproteins
    Haruno Onuma, Rina Shimizu, Yuichi Suzuki, Mina Sato, Hideyoshi Harashima, Yusuke Sato · 2024 · iScience
  58. Entrapment of lipid nanoparticles in peripheral endosomes but not lysosomes impairs intracellular trafficking and endosomal escape
    Yiqin Cheng, E Zhao, Xiaojuan Yang, Chengzhi Luo, Guanghui Zi, Rui Wang, Yuhong Xu, Baowei Peng · 2024 · International Journal of Pharmaceutics
  59. PEG‐Free Monosialoganglioside Lipid Nanoparticles with Spleen‐Selective mRNA Delivery for Cancer Immunotherapy
    Ziqian Wang, Xingwu Liu, Jiahui Jin, Xiaoyi Liu, Cong Liu, Fengfei Xie, Xingyun Wang, Fangjie An, Bao Xiao, Hanchao Cheng et al. · 2025 · Advanced Functional Materials
  60. Hepatocyte-targeted lipid nanoparticle for full-length ATP7B mRNA delivery in Wilson disease
    Yukui Ma, Songli Wang, Zhiwei Guo, Tianhao Ding, Kuan Jiang, Kaisong Tian, Min Jae Yang, Zhiping Cui, Bin Wang, Weiping Wang et al. · 2025 · Journal of Controlled Release
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    Purva Khare, James F. Conway, Devika Soundara Manickam · 2022 · European Journal of Pharmaceutics and Biopharmaceutics
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  63. Engineering a Nano/Biointerface for Cell and Organ-Selective Drug Delivery
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  77. Phase‐Separated Liposomes Hijack Endogenous Lipoprotein Transport and Metabolism Pathways to Target Subsets of Endothelial Cells In Vivo
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  86. Stabilization of Poly (β-Amino Ester) Nanoparticles for the Efficient Intracellular Delivery of PiggyBac Transposon
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  88. Development of Protecting Groups for Prodrug-Type Oligonucleotide Medicines
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  90. Multimodal profiling of biostabilized human skin modules reveals a coordinated ecosystem response to injected mRNA ‐1273 COVID ‐19 vaccine
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  92. Advanced Nanotechnology-Based Nucleic Acid Medicines
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  93. Electron‐Deficient Alkyne Lipids Enable Efficient Synthesis of Comparable Polymer Lipids via Copper‐Free Azide‐Alkyne Cycloaddition
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  94. Unlocking the full therapeutic potential of lipid nanoparticles through extrahepatic delivery
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  97. Delivering the Message: Translating mRNA Therapy for Liver Inherited Metabolic Diseases
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  98. Advancing Cancer Immunotherapy Using Lipid Nanoparticle-Based Approaches
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  100. mRNA-lipid nanoparticle vaccines provide protection against lethal Nipah virus infection
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