FAST is excited to announce funding for its first Innovative Seed Grant, entitled “Hematopoietic stem cell mobilization, engineering, and engraftment without myeloablative conditioning”, awarded to Dr. Rachel Cunningham and Dr. Jennifer Adair. Dr. Adair is a Professor and Vice Chair in the Department of Genetic & Cellular Medicine at the University of Massachusetts, developing innovative, globally accessible treatments that engineer blood stem cells to combat genetic disorders, HIV, and cancers.
This grant is part of FAST’s commitment to Pillar 1, which aims to replace the missing or non-functional maternal copy of the UBE3A gene or protein in neurons of the brain. The Innovation Seed Grant program is designed to spark bold, groundbreaking research in Angelman syndrome by supporting high-risk, high-reward ideas that have the potential to transform both understanding and treatment of the disorder.
This project focuses on improving an approach that utilizes a patient’s own blood-forming stem cells (hematopoietic stem cells, or HSCs), which are modified outside the body to correct the genetic defect and then reinfused. Researchers will first test combinations of stem cell mobilization agents to identify the most effective and rapid way to isolate a high number of HSCs. Then, they will track these cells to ensure that these specialized cells, called microglia, are making it to the brain. Current HSC gene therapies require chemotherapy-based conditioning and complex and multi-day hospital stays that carry risks like immune suppression and are inaccessible to some patients.
If the experiments funded by the ISG are successful, this strategy could eliminate major barriers to current HSC therapy and lay the foundation for a next-generation, accessible treatment approach.