A chemical biology approach identified PI3K as a potential therapeutic target for neurofibromatosis type 2.

ABSTRACT: Mutations in the merlin tumor suppressor gene cause Neurofibromatosis type 2 (NF2), which is a disease characterized by development of multiple benign tumors in the nervous system. The current standard of care for NF2 calls for surgical resection of the characteristic tumors, often with devastating neurological consequences. There are currently no approved non-surgical therapies for NF2. In an attempt to identify much needed targets and therapeutically active compounds for NF2 treatment, we employed a chemical biology approach using ultra-high-throughput screening. To support this goal, we created a merlin-null mouse Schwann cell (MSC) line to screen for compounds that selectively decrease their viability and proliferation. We optimized conditions for 384-well plate assays and executed a p

SUBMITTER: Petrilli AM 

PROVIDER: S-EPMC4212923 | biostudies-literature | 2014

REPOSITORIES: biostudies-literature