AAV Vectors for Efficient Gene Delivery to Rodent Hearts.
Methods in molecular biology (Clifton, N.J.)(2019)
Abstract
Currently, gene therapy is one of the most promising fields in biomedicine, with great therapeutic potential for an array of inherited and acquired diseases. Adeno-associated viral (AAV) vectors have emerged as promising tools to deliver selectively a therapeutic payload to target organs, including the heart. In this chapter, we describe the production and quality control of recombinant AAV (rAAV) vectors of the serotype 9, the most cardiotropic AAV serotype when delivered systemically in rodents. We also describe the systemic administration of rAAV vectors and the local delivery of rAAV vectors by direct intramyocardial injection. Taken together, the methods described in this chapter will allow the reader to deliver efficiently therapeutic genes to the rodent heart, both globally and regionally.
MoreTranslated text
Key words
AAV characterization,AAV delivery,AAV production,AAV9,Adeno-associated virus,Mouse,Rat
AI Read Science
Must-Reading Tree
Example
![](https://originalfileserver.aminer.cn/sys/aminer/pubs/mrt_preview.jpeg)
Generate MRT to find the research sequence of this paper
Chat Paper
Summary is being generated by the instructions you defined