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Papers, explained in our own words

Every entry summarises what the study set out to test, what it found and why it changes how we design delivery systems. Browse research and reviews or search the collection.

586 articles

Showing 1–22 of 22 articles · Keyword: viral vectors Clear keyword

International Journal of Nanomedicine2022ResearchNon-viral Gene Delivery

1. Ionizable Lipid Nanoparticle-Mediated Delivery of Plasmid DNA in Cardiomyocytes

Sérgio Scalzo, Anderson K. Santos, Heloisa A. S. Ferreira, Pedro A. Costa, Pedro H. D. M. Prazeres, Natalia J. A. Da Silva, Lays C. Guimarães, Mário De Morais E Silva, Marco T. R. Rodrigues Alves, Celso T. R. Viana, Itamar C. G. Jesus, Alice P. Rodrigues, Alexander Birbrair, Anderson O. Lobo, Freder

Cardiomyocytes are hard-to-transfect cells, and gene therapy for cardiovascular disease is limited by insufficient delivery to cardiac tissue, nucleic acid degradation, and safety concerns with viral vectors. A safe, effective non-viral platform for pDNA delivery to cardiomyocytes is needed. LNP4 was the top…

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Bioengineering2021ResearchNon-viral Gene Delivery

2. Stabilization of Poly (β-Amino Ester) Nanoparticles for the Efficient Intracellular Delivery of PiggyBac Transposon

Rodgers, T., Muzzio, N., Watson, C., & Romero, G. (2021).

Gene-editing tools are large plasmid constructs that cannot spontaneously enter mammalian cells, and viral vectors are limited by cargo size, immunogenicity, and manufacturing challenges. PBAE nanoparticles are promising nonviral carriers, but unmodified PEG-PDHA nanoparticles release encapsulated plasmid DNA too…

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Applied Materials Today2020ResearchNon-viral Gene Delivery

3. Bispecific T-cell Engager (BiTE) Immunotherapy of Ovarian Cancer Based on MIL-88A MOF/MC Gene Delivery System

Jing Zhao, Danping Lu, Sergio Moya, Haoying Yan, Miaojuan Qiu, Junzong Chen, Xincheng Wang, Yang Li, Haobo Pan, Guochuang Chen, Guocheng Wang

Bispecific T-cell engager (BiTE) immunotherapy is a promising cancer treatment, but clinical application is limited by high production costs and short in vivo half-life of BiTE proteins. A gene delivery system that enables sustained in vivo expression of BiTE could overcome these limitations, but existing non-viral…

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Bioconjugate Chemistry2020ResearchNon-viral Gene Delivery

4. Ionizable Lipid Nanoparticle-Mediated mRNA Delivery for Human CAR T Cell Engineering

Margaret M. Billingsley, Nathan Singh, Pranali Ravikumar, Rui Zhang, Carl H. June, Michael J. Mitchell

CAR T cell therapy currently relies on viral vectors or electroporation for T cell engineering. Viral vectors cause permanent CAR expression and carry safety/manufacturing limitations, while electroporation is cytotoxic and requires specialised equipment. A safer, efficient non-viral mRNA delivery platform for human…

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American Journal of Translational Research (Am J Transl Res)2019ResearchNon-viral Gene Delivery

5. PiggyBac Transposon System with Polymeric Gene Carrier Transfected into Human T Cells

CAR-T cell therapy relies mainly on retroviral/lentiviral vectors, which carry risks of carcinogenicity and complex manufacturing. PiggyBac transposon systems offer a safer, simpler non-viral alternative for stable transgene expression, but primary T lymphocytes are difficult to transfect efficiently. A polymeric…

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International Journal of Pharmaceutics2018ResearchNon-viral Gene Delivery

6. ε-Poly-L-Lysine/Plasmid DNA Nanoplexes for Efficient Gene Delivery In Vivo

Haimanti Mandal, Sameer S. Katiyar, Rajan Swami, Varun Kushwah, Parmeshwar B. Katare, Anand Kumar Meka, Sanjay K. Banerjee, Amirali Popat, Sanyog Jain

Non-viral gene delivery vectors are safer than viral vectors but often suffer from lower transfection efficiency and cytotoxicity. ε-Poly-L-lysine (ε-PLL) is a naturally occurring, FDA GRAS cationic polymer with low molecular weight and potential for low toxicity, but its use as a pDNA nanocarrier for efficient in…

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International Journal of Pharmaceutics2017ResearchNon-viral Gene Delivery

7. Nasal Vaccination with Poly(β-amino ester)–Poly(D,L-lactide-co-glycolide) Hybrid Nanoparticles

Genada Sinani, Melike Sessevmez, M. Koray Gök, Saadet Özgümüş, Alper Okyar, H. Oya Alpar, Erdal Cevher

Mucosal vaccination can stimulate both mucosal and systemic immunity, but free protein antigens generally induce poor systemic immune responses and require adjuvants. There is a need for a safe, effective nasal vaccine carrier that improves antigen stability, uptake, and immunogenicity, especially for poorly…

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International Journal of Nanomedicine2015ResearchNon-viral Gene Delivery

8. An Efficient Method for In Vitro Gene Delivery via Regulation of Cellular Endocytosis Pathway

Jing Luo, Caixia Li, Jianlin Chen, Gang Wang, Rong Gao, Zhongwei Gu

Nonviral gene carriers (cationic polymers and lipids) are promising alternatives to viral vectors, but their transfection efficiencies are much lower. While extensive efforts have focused on chemical modification of gene delivery materials, simple optimisation of the transfection procedure itself—beyond standard…

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ACS Nano2015ResearchNon-viral Gene Delivery

9. Polymeric Nanoparticles for Non-Viral Gene Therapy Extend Brain Tumor Survival In Vivo

Antonella Mangraviti, Stephany Yi Tzeng, Kristen Lynn Kozielski, Yuan Wang, Yike Jin, David Gullotti, Mariangela Pedone, Nitsa Buaron, Ann Liu, David R. Wilson, Sarah K. Hansen, Fausto J. Rodriguez, Guo-Dong Gao, Francesco Dimeco, Henry Brem, Alessandro Olivi, Betty Tyler, Jordan J. Green

Glioblastoma remains highly lethal with poor prognosis despite surgery, radiation, and chemotherapy. Gene therapy is a promising alternative, but viral vectors carry safety risks. Non-viral polymeric nanoparticles are safer but have historically shown poor in vivo efficacy. A biodegradable poly(beta-amino ester)…

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Small2012ResearchNon-viral Gene Delivery

10. A novel assay for quantifying the number of plasmids encapsulated by polymer nanoparticles

Nupura S. Bhise, Ron B. Shmueli, Jose Gonzalez, Jordan J. Green

Non-viral polymeric nanoparticles are promising for gene therapy and stem cell reprogramming, but the number of plasmids complexed per particle affects transfection efficiency and co-delivery of multiple plasmids. Existing methods for quantifying plasmids per particle have significant drawbacks, and few studies have…

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Journal of Controlled Release2012ResearchNon-viral Gene Delivery

11. Transfecting Hard-to-Transfect Lymphoma/Leukemia Cells Using a Simple Cationic Polymer Nanocomplex

Nianxi Zhao, Jianjun Qi, Zihua Zeng, Parag Parekh, Chung-Che Chang, Ching-Hsuan Tung, Youli Zu

Suspension lymphoma/leukemia cells are notoriously hard to transfect with non-viral vectors. Existing methods such as Lipofectamine give poor plasmid DNA delivery in these cells, and viral methods raise safety concerns. A simple, efficient, biodegradable cationic polymer nanocomplex is needed for gene delivery to…

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International Journal of Nanomedicine (Dove Press open-access article; exact citation details not fully provided in the 2011ResearchNon-viral Gene Delivery

12. Synthetic Poly(ester amine) and Poly(amido amine) Nanoparticles for Efficient DNA and siRNA Delivery to Human Endothelial Cells

Stephany Y. Tzeng, Peter H. Yang, Warren L. Grayson, Jordan J. Green

Human umbilical vein endothelial cells (HUVECs) are relevant to tissue engineering, cancer therapy, and angiogenesis-related diseases. Non-viral vectors are safer than viruses but often less efficient. Biodegradable poly(ester amine) (PEA) and poly(amido amine) (PAA) nanoparticles were developed and screened for…

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Journal of Controlled Release2011ResearchNon-viral Gene Delivery

13. Systemic gene transfer of polyethylenimine (PEI)–plasmid DNA complexes to neonatal mice

Wong Sp, Argyros O, Howe Sj, Harbottle Rp

Nonviral vectors have rarely been tested in neonatal mice because of poor delivery efficiency, yet early gene therapy could prevent irreversible disease, requires lower vector doses due to small body size, and may avoid immune reactions before immune maturation. Viral vectors carry risks such as insertional…

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2010ResearchNon-viral Gene Delivery

14. Cationic Lipid–Nucleic Acid Complexes for Gene Delivery and Silencing: Pathways and Mechanisms for Plasmid DNA and siRNA

Kai K. Ewert, Alexandra Zidovska, Ayesha Ahmad, Nathan F. Bouxsein, Heather M. Evans, Christopher S. Mcallister, Charles E. Samuel, Cyrus R. Safinya

Cationic lipid–nucleic acid (CL-NA) complexes are promising non-viral vectors for gene delivery and siRNA-mediated silencing, but their transfection and silencing efficiencies remain low compared with viral vectors. A major gap is poor mechanistic understanding of how complex structure, membrane charge density, and…

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International Journal of Pharmaceutics2010ResearchNon-viral Gene Delivery

15. DC-CholDOPE cationic liposomes A comparative study of the influence factors on plasmid pDNA and siRNA gene delivery

Zhang Y, Li H, Sun J, Gao J, Liu W, Li B, Guo Y, Chen J

DC-Chol/DOPE cationic liposomes are efficient non-viral vectors, but the effects of DC-Chol/DOPE molar ratio, PEGylation, and serum on pDNA versus siRNA delivery were unclear. The study also aimed to test whether pDNA and siRNA have different transfection mechanisms in these liposomes. Optimal DC-Chol/DOPE molar…

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Advanced Materials2009ResearchNon-viral Gene Delivery

16. Small Molecule End Group of Linear Polymer Determines Cell-Type Gene Delivery Efficacy

Joel Sunshine, Jordan J. Green, Kerry P. Mahon, Fan Yang, Ahmed A. Eltoukhy, David N. Nguyen, Robert Langer, Daniel G. Anderson

Viral vectors are efficient but face safety, manufacturing, and cargo-capacity limitations. Cationic polymers such as poly(β-amino ester)s (PBAEs) are promising non-viral alternatives, but their gene delivery efficacy varies across cell types. The study asked whether small-molecule end groups on a linear PBAE could…

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Bioconjugate Chemistry2006ResearchNon-viral Gene Delivery

17. Biodegradable Polymeric Vectors for Gene Delivery to Human Endothelial Cells

Jordan J. Green, Julie Shi, Eugene Chiu, Elizaveta S. Leshchiner, Robert Langer, Daniel G. Anderson

Effective and safe gene delivery to primary endothelial cells in the presence of serum proteins is particularly challenging. Endothelial cells are important targets for cardiovascular disease and cancer, but existing nonviral vectors have low efficacy and/or cytotoxicity under serum-containing conditions. C32…

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Gene Therapy2001ResearchNon-viral Gene Delivery

18. Receptor-mediated gene delivery to human peripheral blood mononuclear cells using anti-CD3 antibody coupled to polyethylenimine

Primary lymphoid cells, especially human peripheral blood mononuclear cells (PBMC), are very difficult to transfect with viral and nonviral vectors. There is a need for efficient nonviral, receptor-mediated gene delivery to PBMC for cell-based therapies and for studying gene function in primary lymphocytes. Naive vs…

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Gene Therapy1999ResearchNon-viral Gene Delivery

19. Cell-specific nuclear import of plasmid DNA

J Vacik, Bs Dean, We Zimmer, Da Dean

Non-viral gene therapy vectors lack efficient cell-specific targeting, with most approaches focusing only on cell surface internalisation rather than nuclear delivery. The nuclear import of plasmid DNA is a critical barrier to gene expression, yet it has been largely overlooked in vector design. A method to achieve…

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Gene Therapy1999ResearchNon-viral Gene Delivery

20. Mechanisms of gene transfer mediated by lipoplexes associated with targeting ligands or pH-sensitive peptides

Sérgio Simões, Vladimir Slepushkin, Pedro Pires, Rogério Gaspar, Maria C. Pedroso De Lima & Nejat Düzgüneş

Cationic liposome–DNA complexes (lipoplexes) are safer than viral vectors but have limited transfection efficiency, toxicity at high doses, and sensitivity to serum/extracellular negatively charged macromolecules. The study asks whether targeting ligands or pH-sensitive fusogenic peptides can improve…

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Journal of Controlled Release1998ResearchNon-viral Gene Delivery

21. Polyethylene Glycol-Grafted Poly-L-Lysine as Polymeric Gene Carrier

Young Hun Choi, Feng Liu, Jin-Seok Kim, Young Kweon Choi, Jong Sang Park, Sung Wan Kim

Non-viral gene delivery systems such as poly-L-lysine (PLL) have low immunogenicity compared with viral vectors, but PLL suffers from poor biocompatibility, rapid degradation, and low transfection efficiency. PEGylation was explored to improve solubility, reduce cytotoxicity, and enhance cell permeability for gene…

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ResearchNon-viral Gene Delivery

22. Efficient gene delivery by EGF-lipoplexes in vitro and in vivo

Maria Buñuales, Nejat Düzgüneş, Sara Zalba, María J. Garrido & Conchita Tros De Ilarduya

Nonviral gene delivery vectors are safer than viral vectors but generally suffer from low and nonspecific transfection efficiency. EGFR is overexpressed on many tumour cells, making EGF-mediated targeting an attractive strategy to improve selectivity and gene delivery into cancer cells. EGF-lipoplexes were…

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