Naked DNA-mediated gene therapy refers to the delivery of naked DNA (without viral vectors) carrying target genes directly to the target cells to realize the transcription and expression of genes with the help of the cell's own genetic expression mechanism, so as to achieve the purpose of preventing or treating diseases. This method does not need to rely on viral vectors, so it is highly safe and simple. Naked DNA is injected directly into target cells by intramuscular injection, intradermal injection or intravenous injection, and the expression of target genes is accomplished in the cells, thus producing therapeutic effects. This approach can be applied to gene therapy for a variety of diseases, such as hereditary diseases, tumors, infectious diseases, and so on.
CD Formulation is a leading global biotechnology service provider, with many years of professional and rich experience, we can provide you with a full range of gene therapy vector delivery system development services. We can provide customized gene therapy non-viral vector development services according to your project requirements, and our professional knowledge and advanced technology can effectively help you promote the research and development of gene therapy.
First of all, we need to determine the therapeutic target and select suitable therapeutic genes, such as corrective genes, suicide genes or immunomodulatory genes, etc. Then we will clone the target gene into the appropriate plasmid vector and construct the expression vector. Then, the target gene should be cloned into the appropriate plasmid vector and the expression vector should be constructed.
Plasmid amplification, extraction, purification and other technical means are used to obtain high-purity naked DNA samples. The concentration, purity and structural integrity of naked DNA should be strictly controlled to ensure the quality.
According to the different indications, the appropriate route of administration, such as intramuscular injection, intradermal injection, intravenous injection, etc., should be selected. At the same time, the dosage should be optimized to ensure effective and safe treatment.
After the naked DNA enters the target cells, it can realize the expression of the target gene through the transcription and translation mechanism in the cells. The transfection efficiency can be evaluated by monitoring the gene expression level and therapeutic effect.
Technologies & Platforms | Content Description |
---|---|
Naked DNA efficient transfection technology platform | Our researchers have developed several technology platforms for different cell types and therapeutic targets. Electroshock transfection, liposome transfection and bioluminescence transfection are among the commonly used technology platforms. Electroshock transfection is achieved by applying electrical pulses to induce naked DNA to enter the cell, liposome transfection is achieved by using synthetic liposomes to wrap the DNA and disrupt the cell membrane, and bioluminescent transfection utilizes a complex of specific proteins and genes to introduce the DNA into the cell, and at the same time, generates fluorescent signals to monitor the efficiency of the transfection. |
In vivo gene editing technology platform | In vivo gene editing therapies require efficient and safe gene delivery systems to deliver therapeutic genes to relevant organs and tissues in the human body. Currently, we offer several major in vivo gene editing delivery technologies such as lipid nanoparticle (LNP) delivery and virus-like particle (VLP) delivery for non-viral vector delivery. |
Hydrodynamic retrograde intrabiliary injection (HRII) technology platform | The intrabiliary injection approach has the potential for injection of vector DNA by ERCP-guided hydrodynamic delivery in combination with sequential multilobular injections, which opens new avenues for direct gene delivery to the liver. |
Zinc finger nuclease (ZFN)technology platform | This technology platform is used to recognize and bind specific DNA sequence repeats consisting of zinc finger proteins and nuclease structural domains of restriction endonucleases that non-specifically cleave DNA by dimerization. |
Technology: Hydrodynamic retrograde intrabiliary injection (HRII) technology
Journal: Mol Ther Methods Clin Dev.
IF: 4.7
Published: 2022
Results: The study aimed to improve hepatic gene therapy in newborns by targeting periportal hepatocytes in weaned pigs. The researchers established a method for delivering non-integrating naked DNA vectors using hydrodynamic retrograde intrabiliary injection (HRII). The procedure involved a surgical approach with laparotomy and temporary liver isolation. A catheter was inserted into the common bile duct through enterotomy to deliver the vectors. The delivery was performed under optimal conditions to avoid histological abnormalities in liver tissue. The HRII method, while less efficient than intraportal delivery, presents a less invasive and potentially more practical approach for hepatic gene therapy in newborns, with the added benefit of being less distressful for the subjects and allowing for repeat treatments.
Fig.1 Direct intrabiliary injection of naked DNA vectors in a model system with newborn piglets. (Chan T, et al., 2022)
CD Formulation has many years of research service experience in gene therapy. In naked DNA development, we have established core technologies including target gene design and screening, plasmid vector construction, production processes, quality standards, and so on. Relying on our existing technology platform, we are committed to promoting the research and application of naked DNA delivery system in gene therapy. If you are interested in us, please feel free to contact us.
Reference