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Brain targeting gene transfer and release system and its prepn process

A brain-targeting and gene-based technology, applied in gene therapy, nervous system diseases, pharmaceutical formulations, etc., can solve problems such as transferrin application limitations, achieve improved brain targeting, high transfection efficiency, and improved transfection efficiency and expression level effects

Inactive Publication Date: 2007-03-21
FUDAN UNIV
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

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Problems solved by technology

However, recent studies have shown that the dissociation constant (Kd) of transferrin and its receptor is 1 μM, while the concentration of free transferrin in the circulation system under physiological conditions is very high, about 25 μM, which is very important for transferrin receptors. In general, it is almost saturated, which limits the application of transferrin as a drug transporter targeting the head group

Method used

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  • Brain targeting gene transfer and release system and its prepn process
  • Brain targeting gene transfer and release system and its prepn process
  • Brain targeting gene transfer and release system and its prepn process

Examples

Experimental program
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Effect test

Embodiment 1

[0062] PAMAM and PEG containing bifunctional groups were dissolved in phosphate buffered saline (PBS) at pH 8.0 according to the molecular ratio of 1:2, stirred and reacted at room temperature for 15 minutes to generate PAMAM-PEG, and unreacted PEG was removed by ultrafiltration and replaced. The buffer was PBS pH 7.0. At the same time, lactoferrin reacted with the sulfhydryl reagent N-succinyl-S-acetylthioacetate (SATA for short) to introduce sulfhydryl groups, and after purification, it was mixed with PAMAM-PEG in a molecular ratio of 1:1, at room temperature After stirring and reacting for 12 hours, PAMAM-PEG-Lf was generated, and unreacted lactoferrin was removed by molecular exclusion chromatography to obtain PAMAM-PEG-Lf.

Embodiment 2

[0064] PAMAM and PEG containing bifunctional groups were dissolved in PBS at pH 8.0 according to the molecular ratio of 1:4, stirred at room temperature for 15 minutes to generate PAMAM-PEG, unreacted PEG was removed by ultrafiltration and the buffer was replaced with PBS at pH 7.0 . At the same time, lactoferrin reacted with the thiol reagent SATA to introduce sulfhydryl groups, and after purification, it was mixed with PAMAM-PEG in a molecular ratio of 2:1, stirred and reacted at room temperature for 12 hours to generate PAMAM-PEG-Lf, and molecular exclusion chromatography Remove unreacted lactoferrin to obtain PAMAM-PEG-Lf.

Embodiment 3

[0066] PAMAM and PEG containing bifunctional groups were dissolved in PBS at pH 8.0 according to the molecular ratio of 1:8, stirred at room temperature for 15 minutes to generate PAMAM-PEG, unreacted PEG was removed by ultrafiltration and the buffer was replaced with PBS at pH 7.0 . At the same time, lactoferrin reacted with the thiol reagent SATA to introduce sulfhydryl groups, and after purification, it was mixed with PAMAM-PEG in a molecular ratio of 4:1, stirred and reacted at room temperature for 12 hours to generate PAMAM-PEG-Lf, and molecular exclusion chromatography Remove unreacted lactoferrin to obtain PAMAM-PEG-Lf.

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Abstract

The present invention belongs to the field of biotechnology, and is one kind of brain targeting gene transfer and release system and its preparation process. Lactoferrin is first used to modify cationic polymer by means of hydrophilic polymer, and the modified cationic polymer is then compounded with plasmid DNA by means of electrostatic effect to form the gene transfer and release system. The present invention can raise the transfection efficiency and expression efficiency of gene on brain capillary endothelial cell effectively, and raise the brain targeting property of gene transfer and release system and the expression of the gene in brain obviously after the non-invasive administration via intravenous injection. Compared with available technology, the present invention has obviously raised brain targeting effect.

Description

technical field [0001] The invention belongs to the field of biological technology, and relates to a brain-targeted gene delivery system and a preparation method thereof. Background technique [0002] Many chronic brain diseases, such as Parkinson's disease and Alzheimer's disease, have a high incidence rate and have plagued human beings for a long time, seriously affecting people's quality of life. Gene therapy is a treatment with great potential, and DNA medicine has become one of the eye-catching macromolecular medicines in recent years. However, due to the existence of the blood-brain barrier, therapeutic genes cannot autonomously enter the lesions in the brain to treat central nervous system diseases. Neurosurgery methods, such as intracerebral intubation and intracerebroventricular injection, which are commonly used clinically at present, are harmful, easily cause intracranial infection, and are not conducive to long-term medication. A method of delivering a gene pro...

Claims

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Application Information

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IPC IPC(8): A61K48/00A61K47/42A61K47/32A61K47/34A61P25/16A61P25/28
Inventor 黄容琴蒋晨裴元英
Owner FUDAN UNIV
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