International Journal of Nanomedicine2023ResearchNon-viral Gene Delivery
Hannah J Vaughan, Camila G Zamboni, Kathryn M Luly, Ling Li, Kathleen L Gabrielson, Laboni F Hassan, Nicholas P Radant, Pranshu Bhardwaj, Florin M Selaru, Martin G Pomper, Jordan J Green
Hepatocellular carcinoma (HCC) has limited treatment options, with modest survival after systemic chemotherapy or transarterial chemoembolization (TACE). Gene therapies hold promise for treating HCC, but delivery remains a critical hurdle. While poly(beta-amino ester) (PBAE) nanoparticles have shown efficacy in…
Read the article →Frontiers in Oncology2022ReviewNon-viral Gene Delivery
Tianqing Xin, Li Cheng, Chuchao Zhou, Yimeng Zhao, Zhenhua Hu, Xiaoyan Wu
CAR-T cell therapy has shown impressive success in haematological malignancies, but systemic toxicity (CRS, ICANS, on-target/off-tumour effects) and the complex, costly, individualized manufacturing process of autologous CAR-T cells hinder broader application. Universal allogeneic CAR-T cells have encountered safety…
Read the article →International Journal of Nanomedicine2022ResearchNon-viral Gene Delivery
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…
Read the article →Frontiers in Pharmacology2022ResearchNon-viral Gene Delivery
Sajid Iqbal, Alessandro Francisco Martins, Muhammad Sohail, Jingjing Zhao, Qi Deng, Muhan Li, Zhongxi Zhao
Polymeric gene delivery vectors require detailed physicochemical characterisation to ensure reproducible formulation and efficient transfection. PBAE-447 is a promising biodegradable cationic polymer, but its simple nanoparticles can degrade or aggregate in aqueous/physiological conditions. This study characterises…
Read the article →Advanced Healthcare Materials2022ReviewNon-viral Gene Delivery
Erin W. Kavanagh And Jordan J. Green
Viral vectors dominate gene therapy but suffer from immunogenicity, insertional mutagenesis risk, cargo-size limits, and manufacturing challenges. Non-viral nanoparticles are safer, more economical, and easier to scale, but their clinical translation has been limited by inefficient delivery, especially for DNA and…
Read the article →2022ReviewNon-viral Gene Delivery
Maeki M, Uno S, Niwa A, Okada Y, Tokeshi M
RNA-loaded lipid nanoparticles (LNPs) are clinically validated for siRNA and mRNA delivery, but their therapeutic performance depends critically on reproducible control of size, size distribution, zeta potential, and RNA encapsulation efficiency. Microfluidic devices offer precise mixing, rapid ethanol dilution, high…
Read the article →Poly (beta-amino ester) as an in vivo nanocarrier for therapeutic nucleic acids.2022ReviewNon-viral Gene Delivery
Sadeqi Nezhad, M
Therapeutic nucleic acids require safe and effective in vivo delivery vectors. Most gene and cell therapies rely on ex vivo gene delivery, which is laborious, time-consuming, and costly. PBAE is a promising biodegradable synthetic cationic polymer for in vivo gene delivery due to its transfection efficiency…
Read the article →Chemical Society Reviews* (as indicated by review format; exact volume/pages and DOI not included in the supplied file)2021ReviewNon-viral Gene Delivery
Yingshu Guo, Xiuping Cao, Xiaofei Zheng, Sk Jahir Abbas, Juan Li, Weihong Tan
Cancer treatment still relies heavily on chemotherapy, which causes severe damage to normal cells and systemic side effects. Safer, more selective drug delivery strategies are needed. Nanocarriers based on nucleic acids (NCNAs) are attractive because nucleic acids offer biocompatibility, low toxicity, programmable…
Read the article →EBioMedicine* (Elsevier)2021ReviewNon-viral Gene Delivery
Ella S. Atsavapranee, Margaret M. Billingsley, Michael J. Mitchell
Adoptive T cell therapy has been revolutionized by genetic engineering, but viral transduction—the standard delivery method—has limitations including small cargo capacity, high immunogenicity, high manufacturing cost, and risks of genotoxicity/insertional mutagenesis. There is a need for improved non-viral delivery…
Read the article →Trends in Biotechnology* (as indicated in the article’s Key Figure footer)2021ReviewNon-viral Gene Delivery
Qurrat Ul Ain, Estefania V.R. Campos, Ariel Huynh, Dominik Witzigmann, Sarah Hedtrich
Gene therapies are powerful tools to prevent, treat, and cure human diseases, but their application to skin diseases has received little attention despite the easy accessibility of skin and urgent medical need. The main obstacle is the unique barrier properties of human skin, which severely limit absorption of…
Read the article →Journal of Materials Chemistry B2021ReviewNon-viral Gene Delivery
Rih-Yang Huang, Zhuo-Hao Liu, Wei-Han Weng, Chien-Wen Chang
Gene therapy requires safe, precise, efficient, and cost-effective delivery. Viral vectors are efficient but face manufacturing, cost, and safety challenges; non-viral vectors are safer and more scalable but often have low delivery efficiency. Magnetic nanoparticles (MNPs) have emerged as a promising strategy to…
Read the article →Advanced Materials2021ReviewNon-viral Gene Delivery
Arun R. K. Kumar, Yufeng Shou, Brian Chan, Krishna L., Andy Tay
This review examines how viral vectors, electroporation, microfluidic systems, nanoparticles and high-aspect-ratio nanostructures can improve the efficiency, viability and scalability of transfecting primary immune cells for applications such as CAR-T, CAR-NK and gene editing. It highlights the trade-offs between…
Read the article →Biotechnology and Bioengineering (accepted manuscript)2021ResearchNon-viral Gene Delivery
Emily Harris, Devon Zimmerman, Eric Warga, Anil Bamezai, Jacob Elmer
Retroviral gene delivery for T cell therapies is expensive, semi-randomly integrating, and variable between patients. Non-viral alternatives are needed, but T cells are notoriously hard to transfect, especially primary T cells. Lipofectamine LTX was the best vehicle in Jurkat cells; optimised conditions in X-VIVO…
Read the article →Nanomaterials2021ResearchNon-viral Gene Delivery
Tresa Lopez-Royo, Victor Sebastian, Laura Moreno-Martinez, Laura Uson, Cristina Yus, Teresa Alejo, Pilar Zaragoza, Rosario Osta, Manuel Arruebo, Raquel Manzano
CRISPR/Cas gene-editing components are often encoded on large plasmids (9–19 kb), which are difficult to encapsulate and transfect. Most PLGA nanoparticle (NP) gene-delivery studies use small nucleic acids (siRNA) or small plasmids (<6 kb), and the structural integrity and functional performance of large plasmids in…
Read the article →Bioengineering2021ResearchNon-viral Gene Delivery
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…
Read the article →Biomaterials2021ReviewDrug Delivery
Shepherd Sj, Issadore D, Mitchell Mj.
Nanomedicine translation is limited by non-scalable, batch-variable bulk NP formulation methods that often produce large, polydisperse particles. Microfluidics offers precise control over microscale mixing and nanoprecipitation, enabling more reproducible NPs with controlled size, size distribution, and loading. This…
Read the article →2021ReviewDrug Delivery
Cancer immunotherapy has shown efficacy but is limited by serious adverse effects, nonspecific inflammation, and challenges in spatiotemporal control of immune responses. Nanomedicine and functionalised nanosystems offer programmable pharmacokinetics, co-delivery of immunomodulators, and targeted delivery…
Read the article →2021ReviewDrug Delivery
Venkatas J, Singh M.
Cervical cancer is immunologically relevant because retained high-risk HPV antigens can be recognised as foreign, making immunotherapy attractive. However, conventional immunotherapy is limited by variable efficacy, immune evasion, cytokine toxicity, and poor targeting. Nanomedicine may improve delivery, targeting…
Read the article →Advanced Healthcare Materials2021ReviewNon-viral Gene Delivery
Ho W, Gao M, Li F, Li Z, Zhang X-Q, Xu X.
Nucleic acid vaccines (DNA and mRNA) offer rapid design, adaptability to changing pathogen strains, and stimulation of both humoral and cellular immunity, but their clinical translation is limited by inefficient delivery—nucleic acids are rapidly degraded by endogenous nucleases, have poor cellular uptake due to…
Read the article →ACS Nano.2021ReviewNon-viral Gene Delivery
Rumiana Tenchov, Robert E. Bird, Allison E. Curtze, Qiongqiong Angela Zhou
Lipid nanoparticles (LNPs) have become key delivery vehicles across pharmaceuticals, with renewed prominence as the delivery platform for COVID-19 mRNA vaccines. The field lacks a broad, quantitative landscape of LNP research across liposomes, solid lipid nanoparticles, nanostructured lipid carriers, cationic…
Read the article →Frontiers in Bioengineering and Biotechnology2021ReviewNon-viral Gene Delivery
Chang D, Ma Y, Xu X, Xie J, Ju S
Polymeric nanoparticles are widely used for cancer theranostics because of their biocompatibility, biodegradability, and structural versatility, but conventional delivery still faces poor target specificity, uncontrolled release, and systemic toxicity. Stimuli-responsive polymeric nanoplatforms are proposed to…
Read the article →Journal of Drug Delivery Science and Technology2021ReviewNon-viral Gene Delivery
Ashok B, Peppas Na, Wechsler Me
CRISPR/Cas9 genome editing holds major therapeutic promise, but in vivo delivery is arguably the most difficult barrier to clinical translation. Efficient, selective transport of editing components to target cells/tissues while minimising off-target effects remains unresolved. This review critiques lipid- and…
Read the article →Biochemical Pharmacology*, Volume 173, 2020, Article 113648 DOI/2020ReviewDrug Delivery
Anne Vejux, Dehbia Abed-Vieillard, Khadija Hajji, Amira Zarrouk, John J. Mackrill, Shubhrima Ghosh, Thomas Nury, Aline Yamminse, Mohamed Zaibih, Wafa Mihoubi, Habiba Bouchabi, Boubker Nasser, Yael Grosjean, Gérard Lizard
7-Ketocholesterol (7KC) and 7β-hydroxycholesterol (7β-OHC) are major auto-oxidation products of cholesterol that accumulate in cardiovascular disease, age-related macular degeneration (ARMD), neurodegenerative disorders, and inflammatory bowel diseases. While their cytotoxicity (oxidative stress, organelle…
Read the article →Immunotherapy* (Future Science Group)2020ReviewNon-viral Gene Delivery
Mona Yazdani, Mahmoud Reza Jaafari, Javad Verdi, Behrang Alani, Mahdi Noureddini, Ali Badiee
Melanoma is a poorly immunogenic and highly aggressive skin cancer with limited long-term survival for advanced disease. Dendritic cell (DC)-based vaccines are promising immunotherapies, but their efficacy relies on critical factors including DC maturation state and efficient antigen delivery. Nanoparticulate…
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