Explore how precise cell-specific gene manipulation using viral vectors transforms disease modeling and therapeutic development. This webinar will highlight how targeted gene knockdown and overexpression strategies enable researchers to efficiently induce and study disease states both in vitro and in vivo, streamlining the path from discovery to preclinical validation. Attendees will gain insights on designing long-term genetic tools that accelerate early research, reduce time and cost, and generate actionable data for vector development.
The speaker will demonstrate how engineered AAV capsids and tailored promotor selection enable reliable microglial targeting, highlighting when and why expression strategies must differ between in vitro and in vivo applications for optimal expression. Real-world examples will illustrate how modifying capsids and promoter combinations can enhance microglial transduction and specificity, enabling deeper understanding of neuroinflammation and neurodegeration.
The session will also explore a dual-vector AAV case study demonstrating successful intranasal delivery of CRISPR tools to the central nervous system. Additionally, the webinar will offer guidance on how to incorporate control viruses and collect strong preliminary data for project-specific vectors.
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