{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Virk MS"],"funding":["NIAMS NIH HHS"],"pagination":["960-8"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC3098640"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["19(5)"],"pubmed_abstract":["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."],"journal":["Molecular therapy : the journal of the American Society of Gene Therapy"],"pubmed_title":["\"Same day\" ex-vivo regional gene therapy: a novel strategy to enhance bone repair."],"pmcid":["PMC3098640"],"funding_grant_id":["R01 AR057076-01A1","R01 AR057076"],"pubmed_authors":["Adams DJ","Virk MS","Lieberman JR","Park SH","Drissi H","Sugiyama O","Gambhir SS"],"additional_accession":[]},"is_claimable":false,"name":"\"Same day\" ex-vivo regional gene therapy: a novel strategy to enhance bone repair.","description":"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.","dates":{"release":"2011-01-01T00:00:00Z","publication":"2011 May","modification":"2024-11-21T10:44:03.574Z","creation":"2019-03-27T00:41:49Z"},"accession":"S-EPMC3098640","cross_references":{"pubmed":["21343916"],"doi":["10.1038/mt.2011.2"]}}