- Updates to SFARI’s 2021 requests for grant applications
- SFARI workshop explores challenges and opportunities of gene therapies for autism spectrum disorder
- Seeing through a forest of SCN2A gene variation
- Making sense out of missense mutations
- A joint variant call-set for all Simons Simplex Collection whole-genome sequences is now available
On May 20, 2021, the SFARI science team held an informational session on the 2021 Genomics of ASD: Pathways to Genetic Therapies request for applications (RFA).
The SFARI 2021 Genomics of ASD: Pathways to Genetic Therapies RFA opened on May 3, 2021. Grants awarded through this targeted RFA are intended to advance our understanding of the genetic basis of autism spectrum disorder (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. Special emphasis will be placed on the use of scalable methods, especially as applied to genes that are suitable targets for genetic therapies.
On May 20, 2021, SFARI scientists Pamela Feliciano, Alan Packer and Julia Sommer presented goals and objectives of this new funding opportunity and answered questions. Stanley T. Crooke, founder and former CEO of Ionis Pharmaceuticals and founder, CEO and Chairman of the Board of the n-Lorem Foundation, also joined the session to discuss current use of antisense oligonucleotides (ASOs) to treat rare genetic conditions as well as future prospects, and answered questions about potential collaborations between n-Lorem and other researchers.
Pamela Feliciano is scientific director of SPARK and a senior scientist at SFARI. She oversees the return of genetic results for participants in SPARK and helps guide the overall research priorities of the program. She also focuses on grants within autism genetics and clinical investigations in SFARI’s portfolio, with a particular emphasis on the development of objective, quantitative methods for measuring clinically relevant autism behaviors.
Alan Packer is a senior scientist at SFARI. His role involves overseeing autism grants in the area of genetics and cell and developmental biology. He also serves as a liaison with the SFARI Gene curation team at MindSpec, helping to lead the development of the site’s Gene Scoring module. He has also participated in the administration of the Simons Simplex Collection exome- and genome-sequencing programs and closely follows efforts to build on these genetic insights in the service of a better understanding of the biology of autism.
Julia Sommer is a senior scientist at SFARI. She mainly focuses on grants of cellular and molecular studies in autism, including projects that make use of induced pluripotent stem cells (iPSCs). She also manages SFARI’s iPSC resources and helps oversees the administration of SFARI’s Pilot and Research Award applications.
Stanely T. Crooke is the founder, chief executive officer and chairman of n-Lorem Foundation. He is also the founder and executive chairman of the board of directors for Ionis Pharmaceuticals, the leader in RNA-targeted therapeutics. Under his leadership of Ionis, he pioneered development of the revolutionary antisense technology platform and created one of the largest, most advanced pipelines in the biotechnology industry.
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