Stem Cell Researchers Develop Promising Method to Treat Sickle Cell Disease
Stem cell researchers have shown that a novel stem cell gene therapy method could lead to a one-time, lasting treatment for sickle cell disease. The study outlines a method that corrects the mutated gene that causes sickle cell disease and shows, for the first time, that the gene correction method leads to the production of normal red blood cells. [Press release from UCLA discussing online publication in Blood] Press Release|Abstract
Role of Transgene Regulation in Ex Vivo Lentiviral Correction of Artemis Deficiency
Researchers compared the human Artemis promoter to the moderate-strength human PGK promoter and the strong human EF1α promoter to regulate expression of Artemis after ex vivo lentiviral transduction of hematopoietic stem cells in a murine model of severe combined immunodeficiency. [Hum Gene Ther] Abstract
Haploidentical BMT and Post-Transplant Cy for Severe Aplastic Anemia: A Multicenter Retrospective Study
The use of post-transplant cyclophosphamide (Cy) has been shown to be an effective strategy to prevent GvHD in recipients of haploidentical hematopoietic SCT, but the majority of reports have focused on patients with hematology malignancies. Researchers describe the outcome of 16 patients who underwent haploidentical transplantation using a reduced-intensity conditioning regimen with post-transplant Cy. [Bone Marrow Transplant] Abstract
Development of Hematopoietic Stem and Progenitor Cells from Human Pluripotent Stem Cells
The authors discuss the current status in the generation of multipotent hematopoietic stem and progenitor cells from human pluripotent stem cells via hemogenic endothelial cells. They also review the achievements in direct reprogramming from non-hematopoietic cells to hematopoietic stem and progenitor cells. [J Cell Biochem] Abstract
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INDUSTRY NEWS
Spectrum Pharmaceuticals Announces FDA’s Acceptance of NDA Filing for Captisol-Enabled™ (Propylene Glycol-Free) Melphalan
Spectrum Pharmaceuticals announced that its New Drug Application (NDA) for captisol-enabled melphalan, has been accepted by the U.S. Food and Drug Administration (FDA). Spectrum is seeking FDA approval for its use as a high-dose conditioning treatment prior to autologous hematopoietic (progenitor) stem cell transplantation in patients with multiple myeloma, an orphan drug designation. [Spectrum Pharmaceuticals, Inc.]
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