Designing genome editing experiments with EditABLE

Area of Science:

• Genomics
• Bioinformatics
• Molecular Biology

Background:

• CRISPR-Cas9 technology offers powerful genome editing capabilities.
• Existing computational tools for CRISPR experiment design are fragmented.
• A centralized resource is needed to optimize genome editing strategies.

Purpose of the Study:

• To develop EditABLE, an integrated online platform for designing efficient CRISPR-Cas genome editing strategies.
• To provide a centralized resource for selecting optimal CRISPR editors and guide RNAs.
• To facilitate applications in base editing, prime editing, and integrase-mediated editing.

Main Methods:

• Developed EditABLE, an accessible web-based application (EditABLE-app.stanford.edu).
• Integrated functionalities for base editing, prime editing, and integrase-mediated editing.
• Utilized user-provided sequence data for personalized editing strategy prediction.

Main Results:

• EditABLE successfully identifies optimal CRISPR editors and guide RNAs for specific genome editing applications.
• The platform demonstrates utility in targeting complex genetic disorders, exemplified by autosomal dominant polycystic kidney disease (ADPKD).
• Specific editing tools were identified across the ADPKD mutation landscape, showcasing EditABLE's practical application.

Conclusions:

• EditABLE serves as a valuable centralized resource for designing efficient CRISPR-Cas genome editing experiments.
• The platform streamlines the selection of appropriate editing tools for diverse applications, including base editing, prime editing, and integrase-mediated editing.
• EditABLE aids in advancing research for genetic disorders by providing tailored editing strategies.