NF1 results from mutations or deletion of the neurofibromin 1 (NF1) gene, which encodes a negative regulator of the Ras signal transduction pathway called neurofibromin. To date, drug development has primarily focused on addressing the molecular abnormalities that arise downstream of the NF1 gene mutation/deletion. There are no therapies that address the underlying issue of this genetic disease; present therapies do not cure the disease. Thus the mission of the Gilbert Family Foundation’s Gene Therapy Initiative (GTI) is to develop curative therapies that address the underlying genetic abnormalities in NF1 patients.
In its first phase, the Gene Therapy Initiative will explore the feasibility of various gene-targeting therapeutic strategies for NF1 as well as novel or enhanced in vivo gene delivery systems. Areas of interest thus include:
- Gene replacement
- Gene editing
- RNA editing
- Exon skipping
- Mutation suppression
- Gene delivery systems
Initial Gene Therapy Initiative awards were announced in December 2018 and the second cycle will be announced in January 2022.
GTI Advisory Board
Barry Byrne, MD, PhD
University of Florida
Leah Byrne, PhD
University of Pittsburgh
School of Medicine
Jeffrey Chamberlain, PhD
University of Washington
Manuela Corti, PhD
University of Florida
Kleopas Kleopa, MD
Cyrus Institute of Neurology and Genetics
Verena Staedtke, MD, PhD
Johns Hopkins University
School of Medicine
Barry Byrne, MD, PhD
University of Florida
Leah Byrne, PhD
University of Pittsburgh
School of Medicine
Jeffrey Chamberlain, PhD
University of Washington
Manuela Corti, PhD
University of Florida
Kleopas Kleopa, MD
Cyrus Institute of Neurology and Genetics
Verena Staedtke, MD, PhD
Johns Hopkins University
School of Medicine