This $454,050 Project Grant award from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846) aims to develop a novel, clinically expedient gene therapy for bone healing. The key products and services to be delivered include:
Developing an adeno-associated virus (AAV)-based gene therapy to deliver bone morphogenetic protein-2 (BMP-2) and potentially an interleukin-1 (IL-1) receptor antagonist to critical-size bone defects in a validated rat model. This will involve optimizing the AAV8-RGD vector for efficient transduction and transgene expression.
Assessing the quality and characteristics of the regenerated bone through mechanical testing, micro-computed tomography, and histological analysis to determine if the new bone forms through intramembranous or endochondral ossification.
Utilizing single-cell RNA sequencing and in situ hybridization to identify and localize transduced cells within the bone defect site.
This 5-year project, awarded to the Mayo Clinic in Rochester, Minnesota, on February 1, 2025, seeks to advance the development of an effective, affordable, and clinically expedient gene therapy for healing critical-size bone defects.