Gene-therapy drug delivery system based on cooperative assembling

Abstract

The invention relates to the field of chemistry and preparations, and specifically relates to a gene-therapy drug delivery system and preparation of two click reaction module molecules included in the gene-therapy drug delivery system. A drug liposome of the gene-therapy drug delivery system further contains a modifier and an alkynylation derivative in addition to conventional drug, liposome and cholesterol, wherein the modifier is inserted to a lipid bimolecular layer of the drug liposome by virtue of hydrophobic interaction; the alkynylation derivative is connected to the outermost layer of the drug liposome by virtue of click chemical reaction; the drug is coated with a hydrophilic core of the drug liposome by virtue of electrostatic interaction with the cationic liposome. The delivery system disclosed by the invention has certain targeting performance.

Description

technical field [0001] The present invention relates to the field of chemistry and preparation. It specifically relates to a gene therapy drug delivery system and the preparation of two types of click reaction module molecules contained therein. Background technique [0002] With the implementation of the Human Genome Project, a large number of genes related to human diseases have been discovered one after another, which has enabled people to have a deeper understanding and understanding of the molecular genetic mechanism of diseases, making gene therapy drugs an important field for the development of biotechnology drugs one. Gene therapy is the introduction of exogenous normal genes into specific tissues and cells of patients for proper expression to correct or compensate diseases caused by gene defects or abnormalities, so as to achieve the purpose of treating diseases. Gene therapy drugs are becoming an important part of biotechnology drugs. Compared with traditional pr...

AI Technical Summary

Problems solved by technology

For the surface coating of nanocomposites, this method relying on physical interactions has serious shortcomings, such as no specific binding sites on the surface of nanocomposites, complex physical interaction forces, specificity and recognition during assembly. The ability is poor, the size of nanoparticles is larger than that of molecules, and it is difficult to carry out stable and effective assembly by weak interactions between molecules, etc.

In addition to the above shortcomings, in the specific implementation of layer-by-layer assembly, negatively charged polymers are usually selected to cover through electrostatic interaction, and the electrostatic force is stronger than other physical forces such as hydrogen bonds, hydrophobic bonds, etc., but many studies have pointed out that in the process of Negatively charged polyanions can easily compete with the cationic carriers in the binding complex during layer-by-layer assembly, displacing the gene drug from the binary complex, greatly reducing the binding rate or drug loading capacity

Moreover, the stability of the coating layer formed by electrostatic interaction is still poor, and it is easy to detach during in vivo transport or be disassembled by competition from negatively charged components in blood such as plasma proteins, and the amount of coating is not easy to quantify and control , the excess free material present in the product may even interfere or affect the targeting function of the delivery system

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