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Liposome-Mediated Gene Delivery Development Service

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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.

Advantages of Liposome-Mediated Gene Delivery Development

  • The structure can be regulated. The structure of liposomes can be altered by adjusting the lipid composition and ratio to meet different application requirements, thus realizing precise regulation of gene delivery.
  • Biocompatible and safe. Liposomes are composed of natural lipids, similar to cell membranes in living organisms, which can effectively avoid immune reactions and cytotoxicity and are easily metabolized and cleared by the body.
  • Efficient gene delivery. Liposomes form stable nanoparticles by wrapping aqueous nucleic acid drugs through their hydrophobic lipid layer. Such a structure enables liposomes to evade enzymatic degradation in vivo effectively and to deliver genetic material into cells via cell membrane fusion or endocytosis pathways.
  • Easy to prepare on a large scale. The preparation process of liposomes is relatively simple and easy to industrialize production, which is conducive to cost reduction.

What We Offer to Develop Liposome-Mediated Gene Delivery?

Designing liposome formulations

Selection of suitable lipid composition and determination of liposome size and surface properties.

Preparing liposomes

Preparation of liposomes by appropriate methods such as thin-film, emulsification or micellar methods.

Analysis and optimization

Characterization of prepared liposomes using physicochemical methods and continuous optimization of the preparation process to improve delivery efficiency.

Packaging of gene drugs

Packaging of target genes by mutual adsorption and covalent binding of negatively charged and cationic liposomes.

In vitro testing

Evaluate the delivery effect of liposomes through cytotoxicity assay, transfection efficiency and stability test.

Animal studies and preclinical studies

Validate the safety and efficacy of liposome-mediated gene delivery in animal models, and perform dose optimization and effect evaluation.

Applications of Our Liposome-Mediated Gene Delivery Development Service

  • Tumor gene therapy. Inhibit the proliferation and metastasis of tumor cells and induce apoptosis of tumor cells by delivering oncogenes or siRNA.
  • Genetic disease treatment. Using liposomes to deliver normal genes or gene repair tools to correct the defects of disease-causing genes and realize gene replacement therapy.
  • Vaccine for infectious diseases. Delivery of vaccine genes in liposomes to enhance the immune response to pathogens.
  • Other diseases in gene therapy. Such as neurological diseases, cardiovascular diseases, etc., there are also attempts to deliver therapeutic genes by liposomes.

Our Platforms for Liposome-Mediated Gene Delivery Development

Technologies & Platforms Content Description
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.
  • Reversed-phase evaporation. Lipid molecules and substances to be loaded are dissolved in the organic phase, ultrasonicated to form a reversed-phase microemulsion, and then vacuum evaporated to remove the organic solvent to obtain liposomes.
  • Extrusion. Monolayers of liposomes of uniform size can be prepared by mechanical extrusion of polycarbonate membranes.
  • Ultrasonication. Smaller sized monolayers can be prepared by sonication of the lipid dispersion.
Liposome observation technology platform
  • Electron microscopy. Used to observe the morphology and structural features of liposomes.
  • Transmission electron microscopy (TEM). Allows observation of the morphology and size distribution of liposomes.
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.

Highlights of Our Liposome-Mediated Gene Delivery Development Service

  • Expertise. Our team has deep expertise in designing and optimizing liposome formulations for effective gene delivery. This can help ensure that liposomes are stable, have the proper size and surface properties for efficient cellular uptake, and can effectively encapsulate and protect genetic material.
  • Transfection optimization guidance. We can provide technical support and guidance on transfection optimization to help customers determine the optimal liposome-to-DNA ratio, cell type, and other parameters for reliable and efficient gene transfection.
  • Customized solutions. We can customize our offerings to meet the unique demands and specifications of any client, assisting them in creating the best liposome gene delivery system for their intended use.

Published Data

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 using a lipid nanoparticle.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.

Reference

  1. Johanna Walther. Lipid Nanoparticle-Mediated Delivery of CRISPR-Cas9 for Therapeutic Gene Correction. null. 2023;0 (0):0-0.

CD Formulation is a leading biotechnology company focused on developing gene therapy formulations. With a strong focus on innovation and scientific excellence...

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