NF1 and neurofibromatosis type 1: However, given the 2019 report of the successful adeno-associated viral transduction of truncated forms of NF1 into human cells,114 the cloning of a full-length Nf1 cDNA that is able to modulate Ras signalling,115 and opening of a proposal for a proof-of-concept of NF1 gene therapy by the Children’s Tumor Foundation and the Gilbert Foundation, the NF1 community is optimistic about novel therapeutics aimed at restoring the function of neurofibromin to correct the disease at its root in order to ultimately reduce the tumour burden in NF1 patients.