Call for proposals: gene-based therapeutic approaches for treatment of neurofibromatosis(2x $240.000 available)

The Children’s Tumor Foundation (CTF) has announced a research program in gene-based therapeutic approaches for the treatment of neurofibromatosis type 1 (NF1). There is 2 times $240.000 availble to fund two proof-of-principle studies in NF1 gene editing, for a duration of two years.

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.

More information is available on the CTF website.