Recent Advances and Future of Gene Therapy for Bone Regeneration


Shapiro G, Lieber R, Gazit D, Pelled G. Recent Advances and Future of Gene Therapy for Bone Regeneration [Internet]. Current Osteoporosis Reports 2018; Copy at


Purpose of Review The purpose of this review is to discuss the recent advances in gene therapy as a treatment for bone regeneration. While most fractures heal spontaneously, patients who present with fracture nonunion suffer from prolonged pain, disability, and often require additional operations to regain musculoskeletal function.

Recent Findings In the last few years, BMP gene delivery by means of electroporation and sonoporation resulted in repair of nonunion bone defects in mice, rats, and minipigs. Ex vivo transfection of porcine mesenchymal stem cells (MSCs) resulted in bone regeneration following implantation in vertebral defects of minipigs. Sustained release of VEGF gene from a collagenhydroxyapatite scaffold to the mandible of a human patient was shown to be safe and osteoinductive.

Summary In conclusion, gene therapy methods for bone regeneration are systematically becoming more efficient and show proof-of-concept in clinically relevant animal models. Yet, on the pathway to clinical use, more investigation is needed to determine the safety aspects of the various techniques in terms of biodistribution, toxicity, and tumorigenicity.

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