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Figure 1. The Gilbert Family Foundation’s Gene Therapy Initiative (GTI) Three-Pronged Strategy for Advancing Novel NF1 Therapeutic Approaches. The GTI framework integrates supporting discovery research with technology and infrastructure development to propel progress in NF1 gene therapy. Discovery research identifies key molecular mechanisms and therapeutic targets within the heterogeneous NF1 disease landscape, while technology and infrastructure development provide essential tools such as assays and preclinical models that support both discovery and the evaluation of novel therapeutic strategies.