NIH R01 · 2025
A novel, clinically expedient, AAV-based gene therapy for bone healing
Abstract This project will develop a novel gene therapy for bone healing based upon new insights from our laboratory on the osteogenic properties of transgenic bone morphogenetic protein-2 (BMP-2), its modulation by inflammatory mediators, especially interleukin-1 (IL-1), and the biology of the healing of large osseous segmental defects. A validated rat model will be used in which a 5 mm critical size, segmental defect is surgically created in the femur. Both female and male animals will be used. BMP-2 is a highly osteogenic morphogen. Recombinant, human (rh) BMP-2 is used clinically in bone healing but its potency is weak, necessitating the use of milligram amounts of rhBMP-2. These are…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.