Grant: Operating Grant: ECRs in Human Development, Child and Youth Health

The overarching goal of my lab is to develop therapeutic strategies for hereditary benign tumor syndrome afflicting pediatric patients. NF is one of those syndromes with an urgent unmet clinical need. 99% of NF patients present with multiple incurable neurofibromas, typically developing during puberty. NF gene therapy, where re-expression of a functional copy of the mutated NF1 gene aims at regressing/curing these neurofibromas, will be a game changer for NF patients. Here, and building on strong preliminary results, we propose two independent strategies to evaluate the conceptual feasibility of NF gene therapy. This new knowledge will cover a significant gap in pediatric research and we are very well positionned to accelerate and promote research to address the critical needs of youth health.