PEDF and Derivative Peptide for Treatment of Osteogenesis Imperfecta

• Absence of pigment epithelium- derived factor (PEDF) causes Osteogenesis Imperfecta (OI) in humans.
• OI Type VI is an autosomal recessive disease manifested by severely impaired bone mineralization and fractures in early childhood.
• PEDF is a regulator of MSC differentiation to the osteoblast lineage. PEDF modulates Wnt/β- catenin signaling to direct MSC fate toward osteoblasts. Restoration of PEDF in this PEDF KO mice corrected the bone phenotype (figure).
• Recently it was shown that PEDF treatment restores bone elasticity and reduces bone brittleness in the PEDF-KO mouse model (Unpublished data).
• Reference: Gattu et al. "Determination of mesenchymal stem cell fate by pigment epithelium- derived factor (PEDF) results in increased adiposity and reduced bone mineral content." The FASEB Journal 27.11 (2013): 4384-4394.
• Intellectual Property: US patent issued No. 10,357,549