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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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Enhanced Gene Delivery and Expression using Intraosseous Injection of Chitosan Nanoparticles Encapsulated Adenine Base Editor Plasmids

Enhanced Gene Delivery and Expression using Intraosseous Injection of Chitosan Nanoparticles Encapsulated Adenine Base Editor Plasmids

Published on: May 16, 2025

In situ bone tissue engineering using gene delivery nanocomplexes.

Atefeh Malek-Khatabi¹, Hamid Akbari Javar¹, Erfan Dashtimoghadam²

¹Department of Pharmaceutical Biomaterials, Faculty of Pharmacy, Tehran University of Medical Sciences, Tehran, 1417614411, Iran.

Acta Biomaterialia

|March 13, 2020

Summary

This study developed microfluidic-synthesized gene delivery nanocomplexes for bone regeneration. These platforms, utilizing plasmid DNA and chitosan on a PCL scaffold, effectively promote osteogenic differentiation of stem cells for tissue engineering.

Keywords:

Bone tissue regeneration Gene delivery Microfluidics micromixing Nanocomplexes Nanofiber scaffolds Plasmid DNA

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Published on: September 11, 2015

Area of Science:

Biomaterials Science
Regenerative Medicine
Nanotechnology

Background:

Gene delivery is crucial for tissue regeneration but requires efficient and safe carriers.
Current methods often lack the precision for localized and controlled genetic material delivery.
Developing advanced platforms is essential for enhancing functional recovery and tissue repair.

Purpose of the Study:

To create microfluidic-assisted synthesized plasmid DNA (pDNA)-based nanocomplexes (NCs) for enhanced bone tissue regeneration.
To optimize NC formation using chitosan (CS) carriers and a micromixer platform.
To immobilize NCs onto a functionalized polycaprolactone (PCL) scaffold for localized gene delivery.

Main Methods:

Microfluidic-assisted synthesis of pDNA-chitosan (CS) nanocomplexes (NCs) using a micromixer.
Tuning flow characteristics to control NC size, zeta potential, and compactness.
Immobilization of NCs onto a metalloprotease-sensitive peptide-functionalized PCL nanofibrous scaffold.
In vitro assessment of osteogenic differentiation of bone marrow-derived mesenchymal stem cells (MSCs).

Main Results:

Successfully fabricated and optimized pDNA-CS NCs using microfluidics.
Demonstrated environmental-sensitive and localized gene delivery via immobilized NCs on the PCL scaffold.
Confirmed significant osteogenic differentiation of MSCs, indicating successful gene delivery and therapeutic effect.

Conclusions:

The developed microfluidic-synthesized gene delivery nanocomplexes show great potential for in situ bone tissue engineering.
This methodology offers a promising approach for localized and controlled gene delivery in regenerative nanomedicine.
The immobilized NCs on functionalized scaffolds provide a robust platform for promoting tissue regeneration.