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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...

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Related Experiment Video

Updated: Apr 20, 2026

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

Composite nanoparticles for gene delivery.

Yuhua Wang¹, Leaf Huang¹

¹Division of Molecular Pharmaceutics and Center for Nanotechnology in Drug Delivery, Eshelman School of Pharmacy, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.

Advances in Genetics

|November 21, 2014

Summary

This summary is machine-generated.

Researchers are developing advanced composite nanoparticles for efficient gene and siRNA delivery. These multifunctional nanomaterials overcome biological barriers, advancing gene therapy from the lab to clinical applications.

Keywords:

Composite nanoparticle Gene therapy Lipid

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Area of Science:

Biomaterials Science
Gene Therapy
Nanotechnology

Background:

Nanoparticle-mediated gene and siRNA delivery is crucial for treating genetic diseases.
Current materials science limits the development of multifunctional nanomaterials for efficient delivery.
Composite nanoparticles offer a solution by integrating multiple materials and functions.

Purpose of the Study:

To highlight fabrication methods for translating nanoparticles from research to clinical use.
To review lipid-based composite nanoparticles, a major class of delivery vehicles.
To address challenges in achieving desired gene delivery efficiency.

Main Methods:

Fabrication of composite nanoparticles incorporating diverse materials.
Classification of lipid-based composite nanoparticles based on their components.
Review of advanced methods for nanoparticle synthesis and characterization.

Main Results:

Composite nanoparticles enable defined nanostructures and multiple functionalities.
Fabrication methods are advancing the potential for clinical translation of gene delivery systems.
Lipid-based composite nanoparticles show significant promise for therapeutic applications.

Conclusions:

Advanced fabrication techniques are key to overcoming barriers in gene therapy.
Composite nanoparticles, particularly lipid-based ones, are vital for efficient genetic manipulation.
Further research in nanoparticle design and fabrication will accelerate clinical translation.