We are thrilled to announce the launch of a groundbreaking research project to develop a novel ARID1B-RD brain organoid model. Dr. Omer Revah from the Hebrew University of Jerusalem has secured generous funding from the Fritz Thyssen Foundation to create and study transplanted ARID1B-RD brain organoids. This research will also evaluate the efficacy of the FAR-funded gene therapy developed at UCL.

Brain organoids are tiny, three-dimensional structures grown in laboratories that mimic certain aspects of the human brain. These organoids provide researchers with a unique tool to study brain development and disorders in a controlled environment outside the human body.

Recently, Dr. Revah and his collaborators, including Dr.Sergiu Pasca from Stanford University, a world leader in this field, achieved ar emarkable breakthrough. They successfully transplanted human brain organoidsinto the brains of newborn rats. The results were astonishing: the transplanted organoids thrived, with human neurons developing impressively, growing largerand forming more complex connections than those grown solely in lab dishes.This innovative approach opens up new possibilities to observe how human braincells behave and interact within a living brain.

In this two-year project, Dr. Revah will first grow organoids from neurons with a mutation in the ARID1B gene, transplant them into newborn rats, and then compare their characteristics to organoids without the mutation. The differences he identifies can shed light on the mechanisms underlying ARID1B-RD. Additionally, these differences will serve as potential targets for the novel gene therapy developed at UCL with FAR's support.

This project, facilitated by FAR's collaboration with leading researchers worldwide, holds great potential to become a significant milestone in the journey toward developing effective treatments. We are deeply grateful to Dr. Revah for his hard work, to the Fritz Thyssen Foundation for their generous support, and to Dr. Gabriele Lignani for being an outstanding collaborator.

Stay tuned for more updates!