Dendritic cell functional improvement in a preclinical model of lentiviral-mediated gene therapy for Wiskott-Aldrich syndrome.

Wiskott-Aldrich syndrome (WAS) is a rare X-linked primary immunodeficiency caused by the defective expression of the WAS protein (WASP) in hematopoietic cells. It has been shown that dendritic cells (DCs) are functionally impaired in WAS patients and was(-/-) mice. We have previously demonstrated the efficacy and safety of a murine model of WAS gene therapy (GT), using stem cells transduced with a lentiviral vector (LV). The aim of this study was to investigate whether GT can correct DC defects in was(-/-) mice. As DCs expressing WASP were detected in the secondary lymphoid organs of the treated mice, we tested the in vitro and in vivo function of bone marrow-derived DCs (BMDCs). The BMDCs showed efficient in vitro uptake of latex beads and Salmonella typhimurium. When BMDCs from the treated mice (GT BMDCs) and the was(-/-) mice were injected into wild-type hosts, we found a higher number of cells that had migrated to the draining lymph nodes compared with mice injected with was(-/-) BMDCs. Finally, we found that ovalbumin (OVA)-pulsed GT BMDCs or vaccination of GT mice with anti-DEC205 OVA fusion protein can efficiently induce antigen-specific T-cell activation in vivo. These findings show that WAS GT significantly improves DC function, thus adding new evidence of the preclinical efficacy of LV-mediated WAS GT.

Root Resorption Diagnostic: Role of Digital Panoramic Radiography.

INTRODUCTION: Root resorption is a pathological process characterized by loss of dental root substance, caused by bacterial infections, traumatic injuries or chemical irritation. Root resorption might be accidentally observed on digital panoramic radiography. OBJECTIVE: The objective of the study was to identify characteristic radiological aspects for the different types of root resorption that could be observed on digital panoramic radiography, to make an easier diagnostic of root resorption. MATERIAL AND METHOD: The retrospective study used the X-ray base from the Oral Rehabilitation and Dental Prosthetics Clinic of UMF Craiova to identify the most representative images for different types of root resorption. Digital panoramic radiographies were analysed by two investigators, of which the most suggestive images were selected and described. RESULTS: Digital panoramic radiographies and dental charts of 240 patients were analyzed. 113 cases of root resorption were identified. External inflammatory root resorption (EIRR) was present in 27.07% of studied cases, external cervical root resorption (ECRR) was identified in 10.83% of all studied cases, external replacement root resorption (ERRR) was diagnosed in 7.08% of studied cases and internal root resorption (IRR) was the most rare type of root resorption, with only 2.08% from all studied cases. 16 cases were selected to describe the radiologic features of different types of root resorption, featuring the most interesting images of root resorption evident on digital panoramic radiographies. DISCUSSION: Comparative analyses have been made between our results and the results of other specific studies, with both similar and different values. The radiological features which lead to the diagnostic of each type of RR were highlighted, assessing the causes that caused the lesions, as well as the treatment recommendations. CONCLUSIONS: Digital panoramic radiography is a useful tool to identify root resorption, since it has become the most common radiological investigation for diagnostic in dentistry. Description of radiological aspects of different types of root resorption on panoramic digital radiography allows faster diagnosis. Still, the CBCT may be recommended in some cases to confirm the diagnosis.

Cell therapy of primary myopathies.

Mesoangioblasts are multipotent progenitors of mesodermal tissues. In vitro mesoangioblasts differentiate into many mesoderm cell types, such as smooth, cardiac and striated muscle, bone and endothelium. After transplantation mesoangioblasts colonize mostly mesoderm tissues and differentiate into many cell types of the mesoderm. When delivered through the arterial circulation, mesoangioblasts significantly restore skeletal muscle structure and function in a mouse model of muscular dystrophy. Their ability to extensively self-renew in vitro, while retaining multipotency, qualifies mesoangioblasts as a novel class of stem cells. Phenotype, properties and possible origin of mesoangioblasts are addressed in the first part of this paper. In the second part we will focus on the cell therapy approach for the treatment of Muscular Dystrophy and we will describe why mesangioblasts appear to be promising candidates for this strategy.