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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: Nov 17, 2025

A Multilayer Microfluidic Platform for the Conduction of Prolonged Cell-Free Gene Expression

A Multilayer Microfluidic Platform for the Conduction of Prolonged Cell-Free Gene Expression

Published on: October 6, 2019

Layer-by-Layer technique as a versatile tool for gene delivery applications.

Dmitrii S Linnik¹, Yana V Tarakanchikova^1,2, Mikhail V Zyuzin³

¹Laboratory of Micro-Encapsulation and Targeted Delivery of Biologically Active Compounds, Peter The Great St. Petersburg Polytechnic University, St. Petersburg, Russia.

Expert Opinion on Drug Delivery

|February 18, 2021

Summary

This summary is machine-generated.

Layer-by-Layer (LbL) technology offers a promising non-viral alternative for gene therapy delivery. This method efficiently transports genetic material like DNA and mRNA, overcoming key challenges in clinical applications.

Keywords:

DNA Layer-by-Layer RNA clinical perspective ex vivo cell modification gene delivery multilayer capsules templates

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Last Updated: Nov 17, 2025

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Area of Science:

Biotechnology
Molecular Biology
Nanotechnology

Background:

Gene therapy holds promise for treating diseases but faces delivery challenges.
Current viral vectors have limitations, necessitating safer, efficient non-viral alternatives.
Efficient in vivo gene delivery requires advanced carrier systems with minimal off-target effects.

Purpose of the Study:

To review Layer-by-Layer (LbL) assembly as a gene delivery platform.
To explore LbL applications for various nucleic acid types (pDNA, mRNA, siRNA, genome-editing tools).
To discuss ex vivo gene modification and clinical potential of LbL systems.

Main Methods:

Literature review of Layer-by-Layer (LbL) gene delivery strategies.
Analysis of LbL system performance for different genetic materials.
Examination of ex vivo applications and clinical considerations.

Main Results:

LbL techniques demonstrate versatility in delivering diverse genetic payloads.
Successful examples of LbL-mediated pDNA, mRNA, and siRNA transfer are presented.
LbL systems show potential for ex vivo cell modification and future clinical gene therapy.

Conclusions:

Layer-by-Layer technology presents a viable and adaptable non-viral approach for gene therapy.
LbL offers significant opportunities for advancing gene delivery and clinical applications.
Further development of LbL systems could overcome current gene therapy delivery bottlenecks.