The Children’s Tumor Foundation (CTF) is pleased to announce the initiation of a research program in gene-based therapeutic approaches for the treatment of neurofibromatosis type 1 (NF1). This initiative begins through this request for applications (RFA), with the first goal to fund two proof-of-principle studies in NF1 gene editing. Funding for each study will be $240,000 total (inclusive of 10% indirect costs) for a duration of two years.
The objective of this RFA is to support proof of principle in vitro studies to investigate the feasibility of genome editing techniques, including but not limited to those based on CRISPR-Cas9, to correct pathogenic mutations in NF1 gene. The ultimate goal of this initiative is to explore the development of genome editing as a potential therapeutic tool for NF1.
Experimental outcomes should include: (1) evidence indicating whether the editing reagents can correct the mutation(s) (and characterization of off-target effects), (2) degree of success in delivering the reagents to Schwann cells, and (3) a functional assay showing efficacy.