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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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Direct Protein Delivery to Mammalian Cells Using Cell-permeable Cys2-His2 Zinc-finger Domains

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Cationic α-Helical Peptides for Gene Delivery into Cells.

T Niidome¹, H Aoyagi

¹Department of Applied Chemistry, Nagasaki University, Nagasaki, Japan.

Methods in Molecular Medicine

|February 15, 2011

Summary

Advancements in nonviral gene transfer utilize cationic lipids and polymers to complex with DNA for cellular uptake. Targeted delivery is achieved by modifying carriers with specific cell-targeting ligands.

Area of Science:

Biotechnology
Molecular Biology
Gene Therapy

Background:

Nonviral gene transfer methods are crucial for therapeutic applications.
Development of efficient and safe gene delivery vectors remains a key challenge.

Purpose of the Study:

To review the progress in nonviral gene transfer techniques.
To highlight the role of cationic lipids and polymers in gene delivery.
To discuss the advancements in targeted gene transfer.

Main Methods:

Utilizing cationic lipids and polymers (e.g., polylysine derivatives, polyethyleneimines, polyamidoamine dendrimers) to form complexes with DNA.
Employing cell-targeting ligands (e.g., asialoorosomucoid, transferrin, insulin, galactose) for directed gene delivery.

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Last Updated: Jun 4, 2026

Direct Protein Delivery to Mammalian Cells Using Cell-permeable Cys2-His2 Zinc-finger Domains

Direct Protein Delivery to Mammalian Cells Using Cell-permeable Cys₂-His₂ Zinc-finger Domains

Published on: March 25, 2015

Peptide-derived Method to Transport Genes and Proteins Across Cellular and Organellar Barriers in Plants

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Published on: December 16, 2016

Construction of Cyclic Cell-Penetrating Peptides for Enhanced Penetration of Biological Barriers

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Published on: September 19, 2022

Main Results:

Cationic nonviral vectors effectively complex with negatively charged DNA.
These complexes facilitate DNA uptake into cells via electrostatic interactions.
Modification of carriers with targeting ligands enables specific cell targeting for enhanced gene transfer.

Conclusions:

Nonviral gene transfer techniques have significantly advanced.
Cationic lipids and polymers are effective carriers for gene delivery.
Targeted gene transfer strategies improve the specificity and efficacy of nonviral vectors.