Gene therapy approaches to regenerating bone

Nadav Kimelman Bleich*, Ilan Kallai, Jay R. Lieberman, Edward M. Schwarz, Gadi Pelled, Dan Gazit

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

78 Scopus citations

Abstract

Bone formation and regeneration therapies continue to require optimization and improvement because many skeletal disorders remain undertreated. Clinical solutions to nonunion fractures and osteoporotic vertebral compression fractures, for example, remain suboptimal and better therapeutic approaches must be created. The widespread use of recombinant human bone morphogenetic proteins (rhBMPs) for spine fusion was recently questioned by a series of reports in a special issue of The Spine Journal, which elucidated the side effects and complications of direct rhBMP treatments. Gene therapy - both direct (in vivo) and cell-mediated (ex vivo) - has long been studied extensively to provide much needed improvements in bone regeneration. In this article, we review recent advances in gene therapy research whose aims are in vivo or ex vivo bone regeneration or formation. We examine appropriate vectors, safety issues, and rates of bone formation. The use of animal models and their relevance for translation of research results to the clinical setting are also discussed in order to provide the reader with a critical view. Finally, we elucidate the main challenges and hurdles faced by gene therapy aimed at bone regeneration as well as expected future trends in this field.

Original languageEnglish
Pages (from-to)1320-1330
Number of pages11
JournalAdvanced Drug Delivery Reviews
Volume64
Issue number12
DOIs
StatePublished - Sep 2012

Keywords

  • Bone regeneration
  • Gene therapy
  • Nonviral vectors
  • Tissue engineering
  • Viral vectors

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