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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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Related Experiment Video

Updated: Jun 5, 2026

Production of Double-stranded DNA Ministrings

Production of Double-stranded DNA Ministrings

Published on: February 29, 2016

Layer-by-layer self-assembly vectors for gene delivery.

Peng Li¹, Na Zhang

¹The School of Pharmaceutical Science, Shandong University, 44 Wenhua Xi Road, Ji'nan, Shandong Province, China. figolipeng@163.com

Current Gene Therapy

|December 25, 2010

Summary

This summary is machine-generated.

Layer-by-layer self-assembly vectors offer efficient gene delivery, overcoming limitations of traditional nanoparticles. These nanovectors provide enhanced nucleic acid loading, controlled release, and targeted delivery for gene therapy applications.

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Last Updated: Jun 5, 2026

Production of Double-stranded DNA Ministrings

Production of Double-stranded DNA Ministrings

Published on: February 29, 2016

Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models

Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models

Published on: March 29, 2019

Area of Science:

Biotechnology
Nanomedicine
Molecular Biology

Background:

Gene therapy success depends on efficient and safe gene delivery vectors.
Nanovector-based nucleic acid delivery is a promising strategy for genetic material transfer.
Traditional nanoparticle encapsulation methods often have low efficiency and lead to gene degradation.

Purpose of the Study:

To review the preparation, advantages, applications, and drawbacks of layer-by-layer self-assembly vectors for gene delivery.
To highlight the potential of layer-by-layer vectors as a potent gene delivery system.
To compare layer-by-layer vectors with traditional nanoparticle-based delivery systems.

Main Methods:

Utilizing the layer-by-layer self-assembly technique for vector preparation.
Surface adsorption of gene/polycation multilayered films onto colloidal particles.
Reviewing existing literature on layer-by-layer vector preparation and performance.

Main Results:

Layer-by-layer self-assembly vectors demonstrate efficient nucleic acid loading.
These vectors allow for controlled release of the loaded gene in physiological environments.
The system offers potential for targeted delivery to specific sites or cell types.

Conclusions:

Layer-by-layer self-assembly vectors represent a potent advancement in gene delivery systems.
They offer significant advantages over traditional nanoparticle encapsulation methods.
Further research into their preparation, application, and potential drawbacks is warranted.