Gene delivery vectors can be categorized into viral and non-viral systems based on their origin. Viral-mediated gene delivery systems employ replication-defective modified viruses but can deliver DNA expression, including adenoviruses, retroviruses, and lentiviruses. Traditional non-viral vectors include micelles, liposomes, dendritic polymers and carbon nanotubes. In recent years, cell-secreted extracellular vesicles, especially exosomes, have been used as nonviral vectors for gene delivery, showing good biocompatibility, low clearance, and desirable cell-targeted delivery capabilities.
CD Formulation offers the development of liposome-mediated gene delivery systems, which can accelerate gene therapy research by directing gene vectors to the cell membrane that can carry genes for expression or inhibit siRNAs and efficiently introduce genetic information into specific cells.
Selection of suitable lipid composition and determination of liposome size and surface properties.
Preparation of liposomes by appropriate methods such as thin-film, emulsification or micellar methods.
Characterization of prepared liposomes using physicochemical methods and continuous optimization of the preparation process to improve delivery efficiency.
Packaging of target genes by mutual adsorption and covalent binding of negatively charged and cationic liposomes.
Evaluate the delivery effect of liposomes through cytotoxicity assay, transfection efficiency and stability test.
Validate the safety and efficacy of liposome-mediated gene delivery in animal models, and perform dose optimization and effect evaluation.
Technologies & Platforms | Content Description |
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Liposome synthesis technology platform | Film hydration method. Lipid molecules are dissolved in an organic solvent, dried to form a lipid film, and then added to the aqueous phase to hydrate to form multilayer liposomes.
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Liposome observation technology platform |
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CRISPR-Cas9 technology platform | There are two approaches to gene therapy using CRISPR-Cas9, the first is to repair damaged genes through the homologous recombination repair (HDR) mechanism, a system in which damaged target genes are corrected by replacing them with intact and healthy homologous genes. The second is the use of non-homologous end-joining (NHEJ) would result in the random insertion of DNA fragments into the gene sequence, thereby altering the gene function or silencing the gene altogether, for the treatment of genetic disorders that require the deletion or silencing of genes. |
Liposome gene transfection technology platform | Liposomal gene transfection is a potential gene therapy method. When the human body lacks normal genes or has mutations in genes, normal genes can be introduced into the patient's body and normal gene expression can be restored through the liposome gene transfection method. This is a very important means of gene therapy, which is expected to treat various hereditary diseases through gene therapy technology. |
Technology: CRISPR-Cas9 technology platform
Published: 2023
Results:Originally a defense mechanism for bacteria, CRISPR-Cas9 has been modified into a precision editing tool. It functions similarly to molecular scissors, generating specific DNA breaks that trigger a repair process that, when combined with DNA, corrects the mutation that caused the disease. How to safely and effectively deliver genes to a patient's affected organs is an important question that needs to be addressed. This paper explores the use of lipid nanoparticles (small virus-sized particles composed of naturally occurring body fat) as a viable delivery system for CRISPR-Cas9. It is shown that the designed nanoparticles can effectively deliver cultured cells and liver well into mice as well as delivery that will produce precise genome editing. Using nano-mediated gene delivery, the authors present a promising method for delivering genes into diseased cells to correct disease-causing mutations.
Fig.1 Advantages of synthetic vectors for CRISPR/Cas delivery using a lipid nanoparticle as example. (Johanna Walther, 2023)
CD Formulation is dedicated to the development of liposome delivery systems. Based on our professional technical team and advanced technology platform, we have developed liposome delivery systems that can effectively overcome the problems of low transfection efficiency, enhance the stability and efficacy of non-viral vectors, improve transfection efficiency, optimize the construction and maintenance of vectors in the nucleus of the host cell, and reduce plasmid loss and cytotoxicity. If you are interested in us, please feel free to contact us.
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