Programmable RNA Writing with Trans-Splicing

• 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

Speakers:

Jonathan Gootenberg

Assistant Professor

Harvard Medical School

Omar Abudayyeh

Assistant Professor

Harvard Medical School