A New Approach for Large DNA Insertions Using rAAV and CRISPR

Inserting large DNA fragments into the genome presents challenges in efficiency and precision. Traditional plasmid-based approaches require microinjection, which is time-consuming, inefficient, and results in low success rates in animal models. This method, utilizing recombinant AAV (rAAV) alongside CRISPR, offers a more effective alternative.

Key Features of This Method
  • Improved Efficiency – Electroporation of rAAV provides better success rates compared to microinjection.
  • Scalability – Previously, projects required 6-8 microinjection sessions; this method typically requires only 1-2.
  • Expanded Insert Size – Uses multiple rAAV vectors to insert up to 9 kb of genetic material, surpassing the 4.7 kb limit of a single rAAV.
  • Consistent Results – Reduces variability by avoiding random integration and concatemer formation.
  • Multiplexing Capabilities – Allows for simultaneous modification of multiple targets.

This method provides a scalable approach to verifying transgene integration, ensuring that edits are made as intended and minimizing the risk of unexpected genomic alterations.

-Monica F. Sentmanat, Lead Author

How It Works

Electroporation-Based Delivery

  • rAAV donors are incubated with embryos, removing the need for microinjection.
  • CRISPR components are electroporated, improving targeting precision.

Sequential Integration with Guide RNAs

  • The first rAAV inserts a portion of the DNA and introduces a guide RNA cut site.
  • A second rAAV inserts additional DNA, followed by a third, allowing integration of up to 9 kb.

Genotyping with Long-Read Sequencing

  • This approach enables precise mapping of insertions, off-target effects, and structural variations.

Hear from Xiaoxia Cui: Insights on This Technique

Xiaoxia Cui, PhD, the director of our lab, discusses the rationale behind this approach, its applications, and how it enhances genotyping accuracy.

Interested in starting a project? Contact us.