Modeling NF2 Tumors for Drug Screening Using Induced Pluripotent Stem Cells
Technical Report,15 Sep 2015,14 Sep 2016
Regenerative Research Foundation Rensselaer United States
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Neurofibromatosis type 2 NF2 is an autosomal dominant genetic disorder with a penetrance of nearly 100 by age 601. NF2 is characterized by multiple tumors to the nervous system and skin, with the most common being a vestibular schwannoma2. Hearing loss is the most common presenting symptom in adults, becoming life-threatening as the tumor impinges on vital structures1. Patients demonstrate considerable heterogeneity in tumor onset and growth and novel tools to explore patient variability are needed. The complex local microenvironment consists of the nerve, schwannoma and vasculature, largely endothelial cells, is not typically modeled as a composite in vitro. Animal models capture this crosstalk but are limited e.g. Avastin is specific to human, but not murine VEGF3. The development of a 3D in vitro NF2 model using iPSCs to generate both Schwann cells and endothelial cells would enable screening of novel therapeutics, as well as a better understanding of current and novel treatments.
- Medicine and Medical Research