Sanjiv Sam Gambhir, MD, PhD

Publication Details

  • "Same Day" Ex-vivo Regional Gene Therapy: A Novel Strategy to Enhance Bone Repair MOLECULAR THERAPY Virk, M. S., Sugiyama, O., Park, S. H., Gambhir, S. S., Adams, D. J., Drissi, H., Lieberman, J. R. 2011; 19 (5): 960-968


    Ex-vivo regional gene therapy with bone marrow cells (BMCs) overexpressing bone morphogenetic protein-2 (BMP-2) has demonstrated efficacy in healing critical sized bone defects in preclinical studies. The purpose of this preclinical study was to compare the osteoinductive potential of a novel "same day" ex-vivo regional gene therapy versus a traditional two-step approach, which involves culture expansion of the donor cells before implantation. In the "same day" strategy buffy coat cells were harvested from the rat bone marrow, transduced with a lentiviral vector-expressing BMP-2 for 1 hour and implanted into a rat femoral defect in the same sitting. There was no significant difference (P = 0.22) with respect to the radiographic healing rates between the femoral defects treated with the "same day" strategy (13/13; 100%) versus the traditional two-step approach (11/14; 78%). However, the femoral defects treated with the "same day" strategy induced earlier radiographic bone healing (P = 0.004) and higher bone volume (BV) [micro-computed tomography (micro-CT); P < 0.001]. The "same day" strategy represents a significant advance in the field of ex-vivo regional gene therapy because it offers a solution to limitations associated with the culture expansion process required in the traditional ex vivo approach. This strategy should be cost-effective when adapted for human use.

    View details for DOI 10.1038/mt.2011.2

    View details for Web of Science ID 000290146200022

    View details for PubMedID 21343916

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