Omar Abudayyeh
Assistant Professor Harvard Medical School
Seminars
Thursday 31st July 2025
Round Table Discussion: Employing Machine Learning & Computational Biology to Validate Novel Therapeutic Targets, Design Robust Guide RNA & Predict Functional Effect of Editing, Driving the Development of Safe, Selective Editors
12:30 pm
• Utilizing machine learning and computational biology to predict the functional effect of editing to assist preclinical development and safeguard patient safety
• Designing high-quality, durable, and ultra-specific guide RNA using machine learning tools to improve editing magnitude and safety
• Employing computational tools to apply RNA editing to common diseases by targeting multiple mutations and identifying novel therapeutic targets
Wednesday 30th July 2025
Programmable RNA Writing with Trans-Splicing
2:30 pm
- Utilizing trans-splicing technology to replace large RNA segments, enabling the correction of multiple mutations at once
- Demonstrated correction for transcripts of therapeutically relevant genes, including those implicated in Huntington's Disease and Rett Syndrome
- Protein-free system design allows for efficient AAV delivery, facilitating in vivo RNA correction strategies
