Gene therapy approaches to regenerating bone

Citation:

Kimelman Bleich N, Kallai I, Lieberman JR, Schwarz EM, Pelled G, Gazit D. Gene therapy approaches to regenerating bone [Internet]. Adv Drug Deliv Rev 2012;64(12):1320-30. Copy at http://www.tinyurl.com/y472ukfr

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.

Notes:

Kimelman Bleich, Nadav Kallai, Ilan Lieberman, Jay R Schwarz, Edward M Pelled, Gadi Gazit, Dan eng R01 DE019902/DE/NIDCR NIH HHS/ R21 DE017096/DE/NIDCR NIH HHS/ Research Support, N.I.H., Extramural Research Support, Non-U.S. Gov't Research Support, U.S. Gov't, Non-P.H.S. Review Netherlands 2012/03/21 06:00 Adv Drug Deliv Rev. 2012 Sep;64(12):1320-30. doi: 10.1016/j.addr.2012.03.007. Epub 2012 Mar 10.

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