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Altering AAV tropism with mosaic viral capsids
Laure Gigout1, Patricia Rebollo, Nathalie Clement
1Department of Gene and Cell Medicine, Cell and Developmental Biology, Mount Sinai School of Medicine, 1425 Madison Avenue, New York, NY 10029-6574, USA.
Summary
Adeno-associated virus (AAV) mosaics enhance gene therapy vector infectivity. This novel approach, using AAV2 mosaics with protein A fragments, enables targeted transduction of specific cell types for improved gene delivery.
Area of Science:
- Gene Therapy
- Molecular Biology
- Virology
Background:
- Adeno-associated virus (AAV)-based vectors are key for gene therapy.
- Limitations exist in AAV tropism, with some cells being resistant to transduction.
- Targeting specific cell types is crucial for many gene therapy applications.
Purpose of the Study:
- To develop improved methods for altering AAV2 tropism.
- To enhance the infectivity and targeting capabilities of AAV vectors.
- To demonstrate the utility of AAV2 mosaics for selective cell transduction.
Main Methods:
- Construction and analysis of AAV2 mosaic vectors.
- Incorporation of the immunoglobulin-binding Z34C fragment of protein A into AAV2 capsids.
- Testing transduction efficiency in MO7e and Jurkat cell lines.
Main Results:
- AAV2 mosaics exhibited improved infectivity over all-mutant virions.
- AAV2 mosaics with a protein A fragment facilitated selective and efficient transduction.
- The system demonstrated versatility in targeting various cell types.
Conclusions:
- AAV2 mosaics represent a significant advancement in modifying AAV tropism.
- The engineered AAV2 vectors with protein A fragments offer precise control over gene delivery.
- This versatile method broadens the potential applications of AAV-based gene therapy.