表达式(计算机科学)
计算生物学
生物
神经科学
计算机科学
程序设计语言
作者
Katie N. Clements,Trevor J. Gonzalez,Aravind Asokan
出处
期刊:Methods in molecular biology
日期:2024-01-01
卷期号:: 227-246
标识
DOI:10.1007/978-1-0716-3678-7_13
摘要
Circular RNAs (circRNAs) have recently emerged as a promising modality for gene and RNA-based therapies. They are more stable than their linear counterpart and can be designed for efficient expression in different cell and tissue types. In this chapter, we developed different backsplicing circRNA cassettes that can enable efficient gene expression in various cell and tissue types. Furthermore, we packaged cassettes encoding circRNAs into adeno-associated viral (AAV) vectors that can be delivered via intracerebroventricular (ICV) injections to achieve expression in murine brain tissue. We provide detailed methods for the design of backsplicing circRNAs, circRNA detection, and generation of AAV-circRNA vectors for CNS dosing and expression in mice.
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