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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: Aug 2, 2025

Isolation of Chondrocytes and Chondroprogenitors Using Fibronectin Adhesion and Migratory Assay

Isolation of Chondrocytes and Chondroprogenitors Using Fibronectin Adhesion and Migratory Assay

Published on: October 4, 2024

Gene Delivery to Chondrocytes.

Christopher V Nagelli¹, Christopher H Evans², Rodolfo E De la Vega¹

¹Musculoskeletal Gene Therapy Laboratory, Mayo Clinic, Rochester, MN, USA.

Advances in Experimental Medicine and Biology

|April 13, 2023

Summary

This summary is machine-generated.

Gene therapy offers new ways to treat cartilage conditions and study chondrocytes. Adeno-associated virus (AAV) shows promise for in vivo gene delivery to cartilage, with ongoing clinical trials for arthritis.

Keywords:

Cartilage Chondrocyte Gene therapy Osteoarthritis

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Area of Science:

Biotechnology and Biomedical Engineering
Gene Therapy and Regenerative Medicine

Background:

Gene delivery to chondrocytes is crucial for treating cartilage disorders and understanding chondrocyte biology.
Viral and non-viral vectors have been developed for both ex vivo and in vivo gene therapy applications.
Ex vivo gene delivery is often integrated with cell-based therapies for cartilage repair.

Purpose of the Study:

To review the current state and challenges of gene delivery to chondrocytes, particularly for in vivo applications.
To highlight the potential of adeno-associated virus (AAV) for intra-articular gene therapy.

Main Methods:

Review of existing literature on gene therapy vectors and their application to chondrocytes.
Discussion of the physical barriers within the cartilage extracellular matrix affecting vector diffusion.
Comparison of different viral vector sizes and their efficacy in chondrocyte transduction.

Main Results:

The dense cartilage matrix significantly limits the penetration of larger viral vectors like adenovirus.
Adeno-associated virus (AAV), with its smaller size, has demonstrated successful transduction of chondrocytes in equine joints.
In vivo delivery to articular chondrocytes remains challenging due to matrix properties, while ex vivo methods are more established.

Conclusions:

Adeno-associated virus (AAV) represents a promising vector for in vivo gene therapy in cartilage, currently under investigation in clinical trials for arthritis.
Further research is needed to confirm AAV's efficacy in transducing human chondrocytes throughout cartilage depth after intra-articular injection.
Limited research exists on gene transfer to chondrocytes in non-articular cartilaginous tissues.