Personalizing the care of children with NF1
Our ability to predict what medical problems will arise in any given individual with NF1 remains limited. In order to define risk factors for brain tumors and autism, Dr. Gutmann and his team will employ a unique series of Nf1 mouse strains and patient-derived induced pluripotent stem cells, personalized with specific NF1 patient NF1 gene mutations. This initiative represents an important first step in creating prognostic bioinformatic tools that enable precision medicine approaches for people with NF1.
Discovering and translating novel therapies
Dr. Gutmann and his team will build upon their discoveries that low-grade tumor formation and growth are governed by non-cancerous cells and signals in the tumor microenvironment (stroma). This initiative involves the complementary use of electronic health records, novel Nf1 mouse strains, and human biospecimens to identify and evaluate the most promising stroma-directed treatments that reduce the growth of low-grade (benign) brain and nerve tumors.
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