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Predicting candidate small molecules for neurofibromatosis type 1 using a disease signature reversion-based approach

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posted on 2021-07-08, 19:11 authored by Jineta Banerjee, Robert AllawayRobert Allaway, Diogo M. Camacho, Christine A. Pratilas, James J Collins, Jaishri O. Blakeley, Justin Guinney
• Very few therapeutic strategies exist for malignant peripheral nerve sheath tumors (MPNST) and plexiform neurofibromas (pNF) with the recent exception of Koselugo (selumetinib) for pNF
• pNFs sometimes undergo malignant transitions to MPNSTs, potentially triggered by changes in gene expression
• We applied a new pattern recognition-based method called DRUID (DRUg Indication Discoverer) on publicly available gene expression data from human tumor samples banked at the Johns Hopkins NF1 Biospecimen Repository (JHU Biobank)

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Neurofibromatosis Therapeutic Acceleration Program

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