research and DEVELOPMENT
CELLTIUM has developed PRECISION™, a state-of-the-art drug discovery platform implemented with high-throughput and high-content automated workflows. Our IRIS™ (intuitive reconn imaging system) and machine learning framework further drive our CMT BigData and A.I. architecture. Revolutionized by innovative modular technologies, our PRECISION™ platform targets Charcot-Marie-Tooth disease (CMT), diabetic peripheral neuropathy (DPN), and chemotherapy-induced peripheral neuropathy (CIPN).
CELLTIUM is testing customizable proof-of-concept for gene therapy targeting Charcot-Marie-Tooth diseases. We refine adeno-associated virus (AAV) as an impactful gene transfer system. CELLTIUM completes CRISPR (clustered regularly interspaced short palindromic repeats) techniques to repair disease-causing genetic defects and ultimately restore the normal functions of target cells.
SCHWANN CELL & MYELIN SHEATH
Schwann cell is the main non-neuronal cell-type, known as glia or glial cells, in the peripheral nervous system. Schwann cell forms the lipid-rich membranous myelin sheath surrounding peripheral axons, which functions as electrical insulator to ensure proper propagation of neural information along peripheral nerves. In addition, Schwann cell maintains environmental and metabolic homeostasis for peripheral nerves. Many genetic mutations found in Charcot-Marie-Tooth patients primarily interfere with Schwann cell development and function including myelin sheath formation and maintenance, while neuronal or axonal defects are manifested in CMT2 subtypes.