Modeling NF2 Tumors for Drug Screening Using Induced Pluripotent Stem Cells
Abstract
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 VEGF)3. 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.
Document Details
- Document Type
- Technical Report
- Publication Date
- Dec 01, 2019
- Accession Number
- AD1094800
Entities
People
- Sally Temple