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Polypeptide tree-shaped macromolecule as gene vector and use thereof

A technology of macromolecules and macromolecular carriers, applied in gene therapy, medical preparations of non-active ingredients, non-effective ingredients of polymer compounds, etc., can solve the problems of immunogenicity and lack of tissue specificity

Inactive Publication Date: 2009-06-24
刘湖
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

Although viral vectors have high transfection efficiency, there are also many insurmountable shortcomings, such as immunogenicity, cytotoxicity, lack of tissue specificity, etc.

Method used

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Examples

Experimental program
Comparison scheme
Effect test

preparation example Construction

[0022] The invention relates to a dendritic polypeptide macromolecular carrier system capable of forming a stable and soluble complex with nucleic acids and loading therapeutic genes and a preparation method thereof. The composition contains cationic macromolecular units such as polyethylene glycol Covalently linked dendrimer peptide macromolecules. During dissociation, the vector system complex is able to release nucleic acids for transfection of several types of cells.

[0023] The following examples should not be construed as limiting the present invention in any sense.

Embodiment 1

[0024] Example 1 Synthesis of lysine-based dendrimer polypeptide macromolecule (1)

[0025] Lysine is the most widely used branching unit in the construction of polypeptide dendrimers, with diphenylmethylamine as the core, Boc-protected L-lysine as the branching unit, DCC as the coupling agent, and trifluoroacetic acid as the desorbing unit. Protecting agent, repeated coupling-deprotection reaction. Each generation of product in the synthesis process needs to be separated and purified by column chromatography, and then the next generation of synthesis is performed, so the efficiency is low, but the structure of the obtained product is very regular, and the theoretical molecular weight is almost consistent with the results obtained by mass spectrometry. The structure of this dendritic polypeptide macromolecule is unconventional, and the number of amino groups in the outer layer can be accurately controlled. It has the typical characteristics of dendritic macromolecules such as:...

Embodiment 2

[0026] Example 2 Synthesis of Lysine-based Dendritic Polypeptide Macromolecule-Polyethylene Glycol Conjugate (1-PEG)

[0027] In a nitrogen-protected ice-water bath, polyethylene glycol 0.042M of various molecular weight ranges was dissolved in 0.5 mL of anhydrous DMF. IBCF (0.036M) dissolved in 0.5 mL NMF was added dropwise to the above solution. The mixture was stirred in an ice-water bath for 30 minutes, and then added dropwise to 1 mL of anhydrous dimethylsulfoxide (DMSO) containing 25 mg of lysine-based dendrimer (1·HBr) and 10 μL of TEA, and then Stir at room temperature for another 3 hours. Precipitate the product in 100 mL of anhydrous ether and dissolve the precipitate in 10 mL of distilled water. Dissolve it in 0.01M NaHCO 3 Dialysis was performed against solution (dialysis bag MWCO 25,000), then against 0.1 M hydrochloric acid solution, and then against distilled water. The dialyzed product was finally freeze-dried. pass 1 H-NMR calculated the molar ratio of am...

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Abstract

The invention relates to a polypeptide dendric macromolecule used as a gene vector and application thereof. The polypeptide dendric macromolecule used for preparing a dendric polypeptide macromolecule vector system for loading treating genes consists of treating genes, dendric polypeptide macromolecules or dendric polypeptide macromolecule connected with cation macromolecules. The structural unit of the dendric polypeptide macromolecule is a natural amino acid which can be single amino acid or formed by different amino acids. The dendric polypeptide macromolecule vector system can be salt compounds and ester compounds accepted in the pharmacology. The dendric polypeptide macromolecule vector system for loading treating genes can treat inherited diseases due to gene defect, immune deficiency or tumors due to inactivation of cancer inhibiting genes.

Description

technical field [0001] The present invention relates to polypeptide dendrimers as gene carriers and applications thereof, aiming to provide a composition for effectively mediating therapeutic gene delivery into target cells, a biocompatible combination for effective DNA delivery substances, resulting in non-cytotoxic transfection into target cells. The dendrimer polypeptide macromolecule carrier system, which can be used to prepare therapeutic genes, consists of therapeutic genes, dendrimer polypeptide macromolecules or dendrimer polypeptide macromolecules connected with cationic macromolecules. The structural unit of the dendritic polypeptide macromolecule is a natural amino acid, which can be a single amino acid or be composed of different amino acids. The dendrimer polypeptide macromolecule carrier system can be pharmaceutically acceptable salt compounds and ester compounds. The dendritic polypeptide macromolecule carrier system loaded with therapeutic genes can treat dis...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K47/48A61K47/42A61K48/00A61P35/00C12N15/63A61K47/60A61K47/64
Inventor 刘湖
Owner 刘湖
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