Recent examples of FAST grants include powerful work from Dr. Jim Wilson (hUBE3a, miRNA, CRISPR AAV delivery for gene replacement or gene-editing); Dr. Bryce Reeve for the development of a novel communication endpoint for Angelman syndrome; Dr. Yong-Hui Jiang, who created a biorepository to include all genotypes for AS cell lines; Dr. Albert Keung, who developed landing pads in AS organoids to test drugs for all genotypes including large deletion (genes outside of UBE3A), mutation, UPD/ICD and mosaic; Drs. Jill Silverman, Dave Segal and Kyle Fink at UC Davis to create an AS animal testing facility for all pharma to utilize in early stage POC studies; and more.
In addition to grants funded by FAST, in October 2023, the National Institutes of Health awarded the first phase of a roughly $40m grant to Yale University to advance a novel CRISPR-based gene-editing delivery platform for the targeted treatment of neurogenetic diseases. The two diseases being targeted in the project are Angelman syndrome and H1-4 (HIST1H1E) syndrome.