Related Experiment Video
Updated: Jan 28, 2026
09:20
Isolation of Next-Generation Gene Therapy Vectors through Engineering, Barcoding, and Screening of Adeno-Associated Virus AAV Capsid Variants
Published on: October 18, 2022
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AAV Vectors for Efficient Gene Delivery to Rodent Hearts
Estrella Lopez-Gordo1, Erik Kohlbrenner1, Michael G Katz1
1Cardiovascular Institute, Icahn School of Medicine at Mount Sinai, New York City, NY, USA.
Methods in Molecular Biology (Clifton, N.J.)
|February 21, 2019
Summary
Gene therapy using adeno-associated viral (AAV) vectors shows promise for treating heart diseases. This chapter details methods for producing and delivering AAV serotype 9 vectors for efficient gene transfer to the rodent heart.
Area of Science:
- Biomedicine
- Gene Therapy
- Cardiology
Background:
- Gene therapy offers significant therapeutic potential for various diseases.
- Adeno-associated viral (AAV) vectors are effective tools for targeted gene delivery.
- AAV serotype 9 exhibits high cardiotropism in systemic delivery to rodents.
Purpose of the Study:
- To describe the production and quality control of recombinant AAV (rAAV) serotype 9 vectors.
- To detail methods for systemic and local administration of rAAV vectors in rodents.
- To enable efficient therapeutic gene delivery to the rodent heart.
Main Methods:
- Production and quality control of recombinant AAV serotype 9 vectors.
- Systemic administration of rAAV vectors.
- Local delivery of rAAV vectors via intramyocardial injection.
Main Results:
- Established protocols for rAAV serotype 9 production and quality assessment.
- Demonstrated effective systemic and local delivery of rAAV vectors to the rodent heart.
- Facilitated efficient gene transfer to the cardiac tissue.
Conclusions:
- The described methods allow for efficient global and regional delivery of therapeutic genes to the rodent heart using rAAV vectors.
- This work provides a valuable resource for researchers studying cardiac gene therapy in preclinical models.
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