Article
Pharmacology & Pharmacy
Diviya Santhanes, Alex Wilkins, Huiming Zhang, Robert John Aitken, Mingtao Liang
Summary: Lipid/polymer hybrid nanoparticles loaded with red fluorescent protein encoded plasmid DNA were successfully formulated using microfluidics. These nanoparticles showed high encapsulation efficiency and transfection efficacy, demonstrating their potential for clinical applications in DNA therapeutics.
INTERNATIONAL JOURNAL OF PHARMACEUTICS
(2022)
Article
Biochemistry & Molecular Biology
Alex J. Anderson, Emerson Grey, Nicholas J. Bongiardina, Christopher N. Bowman, Stephanie J. Bryant
Summary: A new particulate delivery system based on step-growth thiol-Michael dispersion polymerization was reported in this study, in which synthetic nucleic acid mimic click nucleic acids (CNA) were functionalized onto low polydispersity microparticles. The results showed that CNA-functionalized microparticles exhibited controllable DNA loading and release properties, suggesting a promising platform for controlled DNA delivery.
Article
Biochemistry & Molecular Biology
Carly Boye, Sezgi Arpag, Michael Francis, Scott DeClemente, Aislin West, Richard Heller, Anna Bulysheva
Summary: Gene therapy has a wide range of applications but viral delivery methods have safety concerns. By using smaller plasmid DNA backbones, gene expression levels can be significantly increased using non-viral methods, as shown in rat tenocytes and myocardium. Delivery to the skin showed more moderate improvements.
BIOELECTROCHEMISTRY
(2022)
Article
Materials Science, Biomaterials
Randa Zoqlam, Chris J. Morris, Mohammad Akbar, Alaaldin M. Alkilany, Sherif I. Hamdallah, Peter Belton, Sheng Qi
Summary: An effective delivery vehicle is crucial for successful gene therapy, with nanoparticles often being used as the preferred choice. Despite claims of superior properties of microfluidic-prepared nanoparticles, direct comparisons with conventional methods are lacking. This study compared DNA-loaded nanoparticles prepared by microfluidic and conventional methods, finding differences in size and DNA loading efficiency. While both methods showed low cytotoxicity, conventional preparation showed higher transfection efficiency, indicating potential for plasmid delivery.
MATERIALS SCIENCE & ENGINEERING C-MATERIALS FOR BIOLOGICAL APPLICATIONS
(2021)
Article
Chemistry, Multidisciplinary
Tresa Lopez-Royo, Victor Sebastian, Laura Moreno-Martinez, Laura Uson, Cristina Yus, Teresa Alejo, Pilar Zaragoza, Rosario Osta, Manuel Arruebo, Raquel Manzano
Summary: The study evaluated the synthesis of large-size plasmid-loaded PLGA nanoparticles using two different methods and compared their effects. While the double emulsion method had high encapsulation efficiency, it couldn't preserve plasmid integrity; the nanoprecipitation method maintained plasmid integrity but did not show cell expression in vitro. Further optimization or alternative synthesis methods are needed to use PLGA nanoparticles as delivery vectors for gene editing therapeutic technologies.
Article
Engineering, Chemical
P. Ferreira, M. Riscado, S. Bernardo, M. G. Freire, J. L. Faria, APM. Tavares, CG. Silva, F. Sousa
Summary: Therapeutic approaches based on nucleic acids have gained attention, but require effective manufacturing strategies and precise quality control. This study describes a simple and efficient method using carbon nanotubes to selectively capture RNA and other impurities, resulting in the isolation and clarification of plasmid DNA (pDNA) from complex samples.
SEPARATION AND PURIFICATION TECHNOLOGY
(2023)
Review
Pharmacology & Pharmacy
Margaret Wallen, Farrukh Aqil, Wendy Spencer, Ramesh C. Gupta
Summary: Despite its introduction more than three decades ago, gene therapy has not lived up to its potential due to the lack of an effective delivery system. This review highlights the use of exosomes derived from bovine milk and colostrum as a nano-delivery platform for gene therapy. These exosomes provide an abundant, scalable, and cost-effective source for targeted delivery of nucleic acids, overcoming the limitations of current gene therapy delivery techniques.
Article
Instruments & Instrumentation
Behzad Baradaran, Ali Mohammadi, Sara Shamekhi, Nikoo Majidazar, Azita Dilmaghani, Saiedeh Razi Soofiyani, Nigel A. J. McMillan, Farzaneh Lotfipour, Somayeh Hallaj-Nezhadi
Summary: Novel nanoliposomes loaded with murine interleukin-12 (mIL-12) pDNA were successfully prepared using a freeze-drying of a monophase solution method, showing promising physicochemical characteristics and transfection efficiency. In vivo experiments demonstrated significant inhibition of mIL-12 expression in colon carcinoma cells and tumor growth after repeated injections of the nanoliposomes. This study highlights the potential of freeze-drying a monophase solution method for preparing pDNA-loaded nanoliposomes for cancer gene therapy.
DRUG DELIVERY AND TRANSLATIONAL RESEARCH
(2022)
Article
Biotechnology & Applied Microbiology
Kaamini M. Dhanabalan, Ameya A. Dravid, Smriti Agarwal, Ramanath K. Sharath, Ashok Kumar Padmanabhan, Rachit Agarwal
Summary: Trauma to the knee joint can lead to cartilage degeneration and bone erosion, causing osteoarthritis. Current treatments focus on symptom relief. Modulating autophagy and targeting senescence pathways show potential as treatment strategies. A study found that rapamycin-loaded microparticles can effectively mitigate cartilage damage and inflammation, potentially preventing the progression of post-traumatic osteoarthritis.
BIOENGINEERING & TRANSLATIONAL MEDICINE
(2023)
Article
Chemistry, Analytical
Eve Robinson, Paul Giffen, Dave Hassall, Doug Ball, Heather Reid, Diane Coe, Simon Teague, Rebecca Terry, Meredith Earl, Joseph Marchand, Brian Farrer, Rasmus Havelund, Ian S. Gilmore, Peter S. Marshall
Summary: Controlled-release formulations in the form of micro- or nanoparticles are increasingly popular in the pharmaceutical industry. The use of a multimodal approach, including MALDI MS imaging and TOF-SIMS analysis, helps monitor the distribution of drug in the lung and increases understanding of drug delivery with controlled-release microparticles.
Article
Chemistry, Multidisciplinary
Da Sun, Wenyu Sun, Song-Qi Gao, Cheng Wei, Amirreza Naderi, Andrew L. Schilb, Josef Scheidt, Sangjoon Lee, Timothy S. Kern, Krzysztof Palczewski, Zheng-Rong Lu
Summary: Developing gene replacement therapy for large gene mutations in retinal disorders, such as Stargardt disease, remains a challenge. A non-viral gene therapy using ECO/pDNA nanoparticles has shown promise, with modified ABCA4 plasmids with SV40 enhancer demonstrating increased gene expression. The addition of sucrose stabilized the nanoparticles, leading to improved transfection efficiency, while sorbitol showed fluctuating results.
JOURNAL OF CONTROLLED RELEASE
(2021)
Article
Pharmacology & Pharmacy
Hardy Mitdank, Simko Sama, Meike Troeger, Maria Francesca Testa, Mattia Ferrarese, Dario Balestra, Mirko Pinotti, Alexander Weng
Summary: Minicircle DNA is a promising tool in gene therapy with advantages such as higher transfection efficiency and longer expression time, but its cost-intensive production limits broader use. An optimized production method can lead to high-quality minicircle DNA, which has been investigated for pharmaceutical potential in vitro in a tumor cell model.
INTERNATIONAL JOURNAL OF PHARMACEUTICS
(2021)
Article
Biochemistry & Molecular Biology
Steven J. Hancock, Minh-Duy Phan, Leah W. Roberts, Thu Ngoc Minh Vu, Patrick N. A. Harris, Scott A. Beatson, Mark A. Schembri
Summary: Incompatibility group C (IncC) plasmids are large, broad host range plasmids that spread genes conferring resistance to antibiotics. Recent studies have identified novel IncC conjugation genes, including dtrJ, which has been examined in detail. The role of DtrJ in DNA transfer of IncC plasmids during conjugation has been defined through this work.
MOLECULAR MICROBIOLOGY
(2021)
Article
Pharmacology & Pharmacy
Monica-Carolina Villa-Hermosilla, Sofia Negro, Emilia Barcia, Carolina Hurtado, Consuelo Montejo, Mario Alonso, Ana Fernandez-Carballido
Summary: Inhalation therapy is a potential method for delivering drugs to treat respiratory disorders like COVID-19. This study developed celecoxib-loaded PLGA microparticles (MPs) for inhaled therapy that can be internalized by alveolar macrophages. The MPs had suitable particle size and high encapsulation efficiency, and the drug release lasted for over 40 to 70 days. The CXB-loaded MPs showed no cytotoxicity and exhibited stronger anti-inflammatory activity compared to CXB in solution.
Article
Biochemistry & Molecular Biology
Zhibin Wang, Philip J. Troilo, Thomas G. Griffiths, Laural B. Harper, Amy B. Barnum, Stephen J. Pacchione, Cindy J. Pauley, Jose A. Lebron, Jayanthi Wolf, Brian J. Ledwith
Summary: The study provides a quantitative assessment of the potential integration frequency of Ad5 vectors in mice following intravenous injection, showing up to 0.05 integrations per cell. Integration sites were found to be random and within short segments of homology.