Request for Proposals
Gene Therapy Strategies for Neurofibromatosis Type 1
January 5, 2021
The Gilbert Family Foundation’s (GFF) Gene Therapy Initiative
is pleased to announce a Request for Proposal (RFP) for high-impact research on gene therapy strategies aimed at addressing the loss of neurofibromin 1 (NF1) gene
function in neurofibromatosis type 1 (NF1)
patients. Proposals will be accepted for Team Science Awards
defined as collaborative research amongst investigators with experience in gene-targeting strategies in or outside the NF1 field.
NF1 results from mutations or deletion of the NF1
gene, which encodes a tumor suppressor protein called neurofibromin that is a negative regulator of the Ras signal transduction pathway. NF1
is a large gene, comprised of 60 exons and spanning approximately 350 kb of genomic DNA. Its transcript is between 11 and 13 kb long with an approximate 8,500 bp open reading frame, encoding a protein of 2,818 amino acids. Thousands of different pathogenic mutations distributed along the entire NF1
gene have been identified in NF1 patients. While haploinsufficiency is necessary to cause the disease, tumor formation requires alterations in both copies of the NF1
gene. The somatic mutation (the ‘second hit’) can occur in various cell types and at various timepoints during an individual’s lifetime and contributes to the pathologic variability observed in patients.
This RFP will focus on the development of therapeutic strategies, including drug delivery systems, designed to address the mutant NF1
allele or protect and/or enhance the functioning of the normal NF1
There will be a two-stage peer-reviewed application process:
- In the first stage, Letters of Intent (LOIs) are due by February 28, 2021.
- GFF will select LOIs to advance and notify applicants of their application status by April 1, 2021. For those applications selected to advance, full-length proposals will be due by May 31, 2021.
The full Request for Proposal, including the application instructions, can be accessed here
GFF utilizes the proposalCENTRAL online application tool. LOIs can be submitted here