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A Simple and Efficient Method for In Vivo Cardiac-specific Gene Manipulation by Intramyocardial Injection in Mice
Published on: April 16, 2018
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Surgical Methods for Cardiac Gene Delivery in Large Animals
Michael G Katz1,2, Yoav Hadas3, Adam S Vincek3
1Department of Genetics and Genomic Sciences, Icahn Institute for Genomics and Multiscale Biology, Icahn School of Medicine at Mount Sinai, New York, NY, USA. michael.katz1@mssm.edu.
Methods in Molecular Biology (Clifton, N.J.)
|August 30, 2022
Summary
Surgical gene delivery in large animals faces challenges in translating small animal success to clinical efficacy. Optimized methods are needed to improve myocyte transduction for long-term therapeutic benefits in gene therapy.
Area of Science:
- Cardiovascular Research
- Gene Therapy
- Surgical Innovation
Background:
- Gene therapy shows promise but clinical benefits often lag behind animal study expectations.
- Current gene delivery methods in small animals do not scale effectively to larger species.
- Insufficient myocyte transduction limits long-term efficacy in large animal models.
Purpose of the Study:
- To describe main strategies for surgical gene delivery in large animals.
- To identify limitations of current cardiac gene transfer methods.
- To outline ideal characteristics for optimized gene transfer in large animals.
Main Methods:
- Classification of cardiac gene transfer by injection site, interventional approach, and cardiac circulation.
- Review of randomized clinical trials and animal study discrepancies.
- Discussion of strategies for enhanced myocardial transduction.
Main Results:
- Therapeutic benefits in clinical trials are less substantial than anticipated from preclinical studies.
- Scalability of gene delivery methods from small to large animals is a major hurdle.
- Effective gene transfer requires methods to increase myocyte transduction efficiency.
Conclusions:
- Bridging the gap between small and large animal models is crucial for clinical gene therapy success.
- Optimized surgical gene delivery strategies are essential for achieving therapeutic efficacy.
- Future approaches should focus on extended transgene residence time and targeted delivery.
