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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: Jul 1, 2025

Isolation of Next-Generation Gene Therapy Vectors through Engineering, Barcoding, and Screening of Adeno-Associated Virus AAV Capsid Variants

Isolation of Next-Generation Gene Therapy Vectors through Engineering, Barcoding, and Screening of Adeno-Associated Virus AAV Capsid Variants

Published on: October 18, 2022

Rationally engineered novel AAV capsids for intra-articular gene delivery.

Wenjun Li^1,2, Susi Liu Feng¹, Lizette Herrschaft¹

¹Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.

Molecular Therapy. Methods & Clinical Development

|March 4, 2024

Summary

This summary is machine-generated.

Novel adeno-associated virus (AAV) gene therapy vectors show enhanced joint transduction in mice. Engineered AAV mutants improve efficacy and reduce off-target effects, offering potential for treating joint diseases.

Keywords:

AAV Nab engineering joint rational design transduction

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Last Updated: Jul 1, 2025

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Engineering and Evolution of Synthetic Adeno-Associated Virus AAV Gene Therapy Vectors via DNA Family Shuffling

Engineering and Evolution of Synthetic Adeno-Associated Virus AAV Gene Therapy Vectors via DNA Family Shuffling

Published on: April 2, 2012

Area of Science:

Gene therapy
Orthopedics
Virology

Background:

Intra-articular adeno-associated virus (AAV) gene therapy is promising for joint diseases.
Challenges include low transduction efficiency, off-target expression, and neutralizing antibodies (Nabs).

Purpose of the Study:

To screen AAV serotypes for optimal joint transduction.
To engineer novel AAV capsids for improved intra-articular gene delivery.
To investigate the impact of specific amino acid modifications on AAV transduction and immunogenicity.

Main Methods:

Screened AAV serotypes 1-9 for intra-articular joint transduction in mice.
Engineered novel AAV capsids, including AAV62 (AAV2 VRI swap into AAV6).
Generated and tested AAV6D (T265 deletion) and AAV6M (A263 mutation) mutants.

Main Results:

AAV6 demonstrated the highest joint transduction among screened serotypes.
AAV62 showed increased transduction efficiency per genome copy number.
AAV6D exhibited a 2-fold increase in transduction compared to AAV6.
AAV6M showed a 10-fold decrease in transduction.
AAV6D efficiently transduced synoviocytes and chondrocytes with minimal off-target transduction and reduced Nab formation.

Conclusions:

Rational engineering of AAV capsids can enhance joint transduction after intra-articular administration.
Novel AAV mutants like AAV6D show potential for improved gene therapy in joint diseases.
Engineered AAV vectors may evade Nabs and minimize liver off-target effects.