Recent Advances and Future of Gene Therapy for Bone Regeneration

Galina Shapiro, Raphael Lieber, Dan Gazit, Gadi Pelled*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

45 Scopus citations

Abstract

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 collagen-hydroxyapatite 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.

Original languageEnglish
Pages (from-to)504-511
Number of pages8
JournalCurrent Osteoporosis Reports
Volume16
Issue number4
DOIs
StatePublished - 1 Aug 2018

Bibliographical note

Publisher Copyright:
© 2018, Springer Science+Business Media, LLC, part of Springer Nature.

Keywords

  • Fracture
  • Gene therapy
  • Gene-activated matrix
  • Nonunion
  • Orthobiologics
  • Regenerative medicine

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