Chimerism and gene therapy - Lessons learned from non-conditioned murine bone marrow transplantation models.

Objective: Hematopoietic stem and progenitor cells (HSPCs) can be used as a vector for gene therapies. In order to predict the number of HSPCs cells necessary to achieve the target level of chimerism in an autologous setting, syngeneic male bone marrow (BM) cells were transplanted into 35 non-conditioned female BALB/c mice.

Method: The resulting chimerism was determined at 6-53 weeks using qPCR, cell subpopulation sorting, and colony-forming units (CFU) analysis.

Assessment of stability of CD34+ cell products enriched by immunoselection from peripheral blood mononuclear cells during refrigerated storage.

Durable engraftment of transplanted CD34+ cells largely depends on the quality of the cell product. Limited data are currently available about extended storage of immunoselected CD34+ cells. The aim of our study was to assess the stability of CD34+ cell product with the cells stored in high concentration (80×10 in 6mL) in small bags intended for cell implantation.

The effect of CD34 cell telomere length and hTERT expression on the outcome of autologous CD34 cell transplantation in patients with chronic heart failure.

Age-related telomere attrition in stem/progenitor cells may diminish their functional capacity and thereby impair the outcome of cell-based therapies. The aim of the present study was to investigate the effect of CD34 cell telomere length and hTERT expression on the clinical outcome of autologous CD34 cell transplantation. We studied 43 patients with cardiomyopathy.

DNA Methylation and Hydroxymethylation Profile of CD34-Enriched Cell Products Intended for Autologous CD34 Cell Transplantation.

Epigenetic dysregulation has been shown to limit functional capacity of aging hematopoietic stem cells, which may contribute to impaired outcome of hematopoietic stem cell-based therapies. The aim of our study was to gain better insight into the epigenetic profile of CD34-enriched cell products intended for autologous CD34 cell transplantation in patients with cardiomyopathy. We found global DNA methylation content significantly higher in immunoselected CD34 cells compared to leukocytes in leukapheresis products (2.