Genomics of ASD: Pathways to Biological Convergence and Genetic Therapies — Request for Applications

Grants awarded through this request for applications (RFA) were intended to advance our understanding of the genetic basis of ASD and the molecular and cellular consequences of genetic risk, and to provide a foundation for the development of treatments for select genetically defined forms of the condition.

In recent years, the scientific community has identified more than 100 highly penetrant risk genes for ASD, owing in part to analyses of SFARI-sponsored cohorts such as SPARK, Simons Searchlight and the Simons Simplex Collection. Although important progress has been made in leveraging these genetic findings to better understand the biological underpinnings of ASD, much remains to be done.

In this RFA, we sought proposals in three different areas, with the common goal of identifying new possibilities for intervention. The first did so by identifying possible points of biological convergence among multiple genetic risk factors, which might then have suggested new therapeutic targets. The second continued SFARI-supported efforts to characterize the full range of functional variation in genes associated with neurodevelopmental disorders, again with an eye toward informing translational efforts. The third area focused on the development of genetically based therapies for select forms of ASD. We also encouraged applicants to familiarize themselves with resources that SFARI supports that may accelerate projects, such as our resource of iPS cells, mouse, rat and zebrafish models