Jay R. Lieberman, MD, chair and professor of orthopaedic surgery at the Keck School of Medicine of USC, has received a five-year, $2.2 million grant from the National Institutes of Health’s National Institute of Arthritis and Musculoskeletal and Skin Diseases to research gene therapy to enhance repair of extensive bone injuries. Examples of these types of injuries include fractures with extensive bone loss, non-healing fractures, failed spinal fusion and revision total joint replacement — a procedure performed when the original joint replacement fails.
Lieberman will genetically manipulate human bone marrow cells to overexpress bone morphogenetic protein (BMP), which is a protein that spurs progenitor cells to produce bone.
“There are a number of bone injuries that are very difficult to repair and lack satisfactory solutions,” Lieberman said. “My goal with this grant is to determine whether genetically modifying human bone marrow cells to overproduce BMP will help heal large bone defects in an animal model and, ultimately, provide a better alternative for repairs in humans.”
Lieberman’s study will determine the efficacy and safety of the gene therapy as well as establish a cellular dose of the genetically manipulated cells that can be scaled up for potential use in humans.
An abstract of the grant, 2R01AR057076-06A1, is available on the NIH RePORTER website. One hundred percent of the project’s funding — $2,284,028 — will be federally funded.
— Erica Rheinschild