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Scoop.it!
Diamond-Blackfan anemia is a congenital erythroid hypoplasia. Twenty-five percent 
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BigField GEG Tech's insight:
Here the scientists optimised insulin production using lentiviral transduced canine mesenchymal stromal cells (MSCs) aiming to evaluate their ability for use as surrogate beta cells. Lentivirus vectors encoding the proinsulin gene under the control of SFFV, CMV, EF1α and SV40 promotors were generated and used to transduce primary cMSCs and a hepatocyte cell line. Their results suggest that optimised lentiviral transduction of the insulin gene into primary canine MSCs renders these cells capable of secreting insulin both short- and long-term, in sufficient quantities in vitro to support their potential use in insulin gene therapy.
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In the present study, the scientists assessed the efficacy of a clinically relevant promoter, the human elongation factor 1α short promoter, with or without the locus control region of the β-globin gene for treatment of RPS19-deficient Diamond-Blackfan anemia. The findings demonstrate that these vectors rescue the proliferation defect and improve erythroid development of transduced RPS19-deficient bone marrow cells.