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 a novel approach using 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, without the need for chemotherapy-based conditioning. Current HSC gene therapies require complex, multi-day hospital stays and carry risks like immune suppression, which limits their use. This study aims to overcome those barriers by developing a same-day, non-chemotherapy, non-viral HSC gene therapy platform that is safer, faster, and more scalable.
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 use specially engineered gold nanoparticles carrying CRISPR gene-editing tools to edit the cells without the need for purification or freezing, simplifying the process and lowering costs. These treated cells will be reintroduced into mice to evaluate long-term safety and gene-editing effectiveness.
If successful, this strategy could eliminate major barriers to current HSC gene therapy delivery and lay the foundation for a next-generation, accessible treatment approach.