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Production, Purification, and Quality Control for Adeno-associated Virus-based Vectors
Published on: January 29, 2019
38.4K
Adeno-associated virus: fit to serve
Eric Zinn1, Luk H Vandenberghe1
1Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary, Harvard Medical School, Boston, MA 02114, United States.
Current Opinion in Virology
|August 17, 2014
Summary
Adeno-associated virus (AAV) is a safe gene therapy vector due to its capsid. We explore AAV evolution and engineering strategies to improve its therapeutic applications.
Area of Science:
- Virology
- Gene Therapy
- Evolutionary Biology
Background:
- Adeno-associated virus (AAV) is a parvovirus with no known human disease association.
- Its broad tropism and limited host response make it a promising gene therapy vector.
- The viral capsid determines these traits and is subject to evolutionary pressures.
Purpose of the Study:
- To explore the evolutionary path of AAV and related parvoviruses.
- To examine how natural selection has shaped AAV phenotypes relevant to gene therapy.
- To discuss directed evolution and engineering strategies for improving AAV vectors.
Main Methods:
- Review of evolutionary pathways of AAV and other parvoviruses.
- Analysis of capsid protein evolution in relation to host interaction.
- Examination of directed evolution and engineering techniques.
Main Results:
- AAV's evolutionary history reveals key adaptations for its use in gene therapy.
- Capsid variability is a significant factor shaped by replication pressures.
- Natural selection provides insights into optimizing AAV for therapeutic gene transfer.
Conclusions:
- Understanding AAV evolution is crucial for advancing gene therapy.
- Engineering strategies based on evolutionary principles can overcome current AAV limitations.
- AAV remains a highly attractive platform for future gene transfer applications.
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