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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 inserted. The...

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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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Microorganisms in Medicine and Therapeutics

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Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.

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Evaluation of Polymeric Gene Delivery Nanoparticles by Nanoparticle Tracking Analysis and High-throughput Flow Cytometry

Evaluation of Polymeric Gene Delivery Nanoparticles by Nanoparticle Tracking Analysis and High-throughput Flow Cytometry

Published on: March 1, 2013

A novel temperature-responsive polymer as a gene vector.

Yongzheng Ma¹, Sen Hou, Bin Ji

¹The Key Laboratory of Bioactive Materials, Ministry of Education, College of Life Sciences, Nankai University, Tianjin 300071, China.

Macromolecular Bioscience

|November 12, 2009

Summary

This summary is machine-generated.

A novel temperature-responsive polymer, PDMNIB, shows efficient DNA interaction and enhanced gene transfection capabilities. Its unique thermosensitive properties, with a lower critical solution temperature (LCST) at 20°C, enable temperature-controlled gene delivery.

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Published on: July 21, 2017

Area of Science:

Polymer Chemistry
Biomaterials Science
Gene Delivery Systems

Background:

Developing efficient and controllable gene delivery vectors is crucial for gene therapy.
Temperature-responsive polymers offer potential for stimuli-responsive drug and gene delivery.
Existing polymers may lack precise control over complexation and release.

Purpose of the Study:

To synthesize and characterize a novel temperature-responsive polymer, PDMNIB.
To investigate the interaction of PDMNIB with DNA.
To evaluate the temperature-dependent gene transfection efficiency of PDMNIB.

Main Methods:

Free radical polymerization was used to synthesize poly{2-(dimethylamino)ethyl methacrylate-co-[cis-butenedioic anhydride-poly[(N-isopropylacrylamide)-co-(butyl methacrylate)]]} (PDMNIB).
Lower critical solution temperature (LCST) was determined.
Gel retardation assays were performed to assess DNA binding.
Dynamic light scattering (DLS) and zeta potential measurements were used to analyze PDMNIB/DNA complexes.
Gene transfection efficiency was evaluated at different temperatures.

Main Results:

PDMNIB exhibited significant temperature-responsive behavior with an LCST of 20°C.
Efficient interaction between PDMNIB and DNA was confirmed by gel retardation assays.
Temperature variations altered the size and surface charge of PDMNIB/DNA complexes.
Gene transfection efficiency was significantly improved by temperature control.

Conclusions:

PDMNIB is a promising temperature-responsive polymer for gene delivery.
The thermosensitive interaction between PDMNIB and DNA allows for temperature-modulated gene transfection.
This polymer offers a controllable platform for enhanced gene delivery applications.