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Gene Therapy

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...

Reporter Genes

Reporter Genes

Reporter genes are a type of protein-coding gene that are often tagged to a gene of interest. Once inside a target cell, reporter genes usually produce visually identifiable characteristics like fluorescence and luminescence when expressed along with the gene of interest. Thus, reporter genes “report” the presence or absence of genes of interest in an organism, determine the gene expression pattern, or track the physical location of a DNA segment or protein in the cell.

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Facile Preparation and Photoactivation of Prodrug-Dye Nanoassemblies

Facile Preparation and Photoactivation of Prodrug-Dye Nanoassemblies

Published on: February 17, 2023

Oxidation-Responsive Nanoassemblies for Light-Enhanced Gene Therapy.

Na Zhang^1,2, Yu Wang^1,2, Rui Wu^1,2

¹State Key Laboratory of Chemical Resource Engineering and Beijing Laboratory of Biomedical Materials, Beijing University of Chemical Technology, Beijing, 100029, China.

Small (Weinheim an Der Bergstrasse, Germany)

|September 21, 2019

Summary

This summary is machine-generated.

Researchers developed oxidation-responsive supramolecular assemblies for enhanced gene delivery. These assemblies utilize reactive oxygen species and light to improve nucleic acid transfection and achieve light-enhanced antitumor effects.

Keywords:

antitumor assembly gene therapy light-enhanced oxidation-responsive

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Area of Science:

Biomedical Engineering
Materials Science
Nanotechnology

Background:

Microenvironment-responsive supramolecular assemblies are crucial for controlled drug delivery.
Developing efficient nucleic acid delivery systems remains a significant challenge in gene therapy.
Oxidation-responsive materials offer potential for targeted therapeutic agent release.

Purpose of the Study:

To prepare oxidation-responsive supramolecular polycationic assemblies (CPAs) for nucleic acid delivery.
To investigate the role of reactive oxygen species (ROS) and light in enhancing gene transfection.
To evaluate the in vitro and in vivo antitumor efficacy of p53 gene delivery using CPAs.

Main Methods:

Fabrication of CPAs using β-cyclodextrin-based polycations and a ferrocene-functionalized zinc tetraaminophthalocyanine core.
Assessment of CPA disassembly triggered by ROS (H₂O₂) and singlet oxygen (¹O₂).
Evaluation of pDNA transfection efficiency in A549, HEK 293, and 4T1 cell lines under varying conditions (ROS presence, light exposure).
In vitro and in vivo studies of p53 gene delivery for antitumor effects.

Main Results:

CPAs demonstrated accelerated disassembly in the presence of ROS, enhancing pDNA release and transfection efficiency in A549 cells.
Light irradiation, generating singlet oxygen, further amplified ROS levels and CPA disassembly, leading to improved transfection in HEK 293 and 4T1 cells.
p53 gene delivery via CPAs exhibited significant antitumor effects under illumination in both in vitro and in vivo models.

Conclusions:

Oxidation-responsive CPAs are effective nanoassemblies for nucleic acid delivery.
Light-enhanced ROS generation significantly boosts gene transfection efficiency.
This strategy offers a promising approach for developing advanced gene therapy and photodynamic cancer treatment.