Correction of Sickle Cell Disease in Adult Mice by Interference with Fetal Hemoglobin Silencing Here, scientists show that the repressor BCL11A is required in vivo for silencing of γ-globin expression in adult animals, yet dispensable for red cell production. [Science] Abstract | Press ReleaseTherapeutic siRNA Silencing in Inflammatory Monocytes in Mice Here researchers present an optimized lipid nanoparticle and a CCR2-silencing short interfering RNA (siRNA) that, when administered systemically in mice, show rapid blood clearance, accumulate in spleen and bone marrow, and localize to monocytes. [Nat Biotechnol] Abstract | Press Release CD140a Identifies a Population of Highly Myelinogenic, Migration-Competent and Efficiently Engrafting Human Oligodendrocyte Progenitor Cells To establish a more efficient means of isolating myelinogenic oligodendrocyte progenitor cells (OPCs), investigators sorted fetal human forebrain cells for CD140a, an epitope of platelet derived growth factor receptor α, which is differentially expressed by OPCs. [Nat Biotechnol] Abstract | Press Release
Effect of Donor-Recipient HLA Matching at HLA A, B, C, and DRB1 on Outcomes After Umbilical-Cord Blood Transplantation for Leukemia and Myelodysplastic Syndrome: A Retrospective Analysis Researchers used Cox regression to assess retrospectively the effect of donor-recipient HLA matching on outcomes of single umbilical-cord blood transplantations for leukemia and myelodysplastic syndrome. [Lancet Oncol] Abstract | Press Release Parabronchial Smooth Muscle Constitutes an Airway Epithelial Stem Cell Niche in the Mouse Lung After Injury Here, investigators report that the Wnt/fibroblast growth factor 10 embryonic signaling cascade is reactivated in mature parabronchial SMCs after naphthalene-induced injury to airway epithelium. [J Clin Invest] Abstract | Press Release Blimp1 Expression Predicts Embryonic Stem Cell Development In Vitro Here researchers show that inner cell mass-generated cells expressing Blimp1, a key transcriptional repressor of the somatic program during germ cell specification, emerge on day 2 of blastocyst culture. [Curr Biol] Abstract | Press Release Rapid and Efficient Reprogramming of Somatic Cells to Induced Pluripotent Stem Cells By Retinoic Acid Receptor Gamma and Liver Receptor Homolog 1 Here scientists report that enhancing retinoic acid (RA) signaling by expressing RA receptors (RARs) or by RA agonists profoundly promoted reprogramming, but inhibiting it using a RAR-α dominant-negative form completely blocked it. [Proc Natl Acad Sci USA] Abstract | Press Release Why Is Less Cationic Lipid Required to Prepare Lipoplexes from Plasmid DNA than Linear DNA in Gene Therapy? Herein for the first time, researchers experimentally show that for different types of cationic lipids, plasmid DNA, in contrast to linear DNA, is compacted with a large amount of its counterions, thus yielding a lower effective negative charge. [J Am Chem Soc] Abstract | Press Release Insulin Biosynthesis in Neuronal Progenitors Derived from Adult Hippocampus and the Olfactory Bulb In the present study, investigators demonstrated that insulin is produced not only in the mammalian pancreas but also in adult neuronal cells derived from the hippocampus and olfactory bulb. [EMBO Mol Med] Abstract | Press Release Gene Therapy of Endometriosis Introduced by Polymeric Micelles with Glycolipid-Like Structure To reduce the side effects and improve the lack of clinical treatment countermeasures in endometriosis chemotherapy, a polymeric micelle gene delivery system composed of lipid grafted chitosan micelles and the pigment epithelium derived factor was designed. [Biomaterials] Abstract Influence of Cationic Lipid Composition on Gene Silencing Properties of Lipid Nanoparticle Formulations of siRNA in Antigen-Presenting Cells Scientists examined the uptake, intracellular trafficking, and gene silencing potency in primary bone marrow macrophages and dendritic cells of siRNA formulated in lipid nanoparticles containing four different ionizable cationic lipids namely DLinDAP, DLinDMA, DLinK-DMA, and DLinKC2-DMA. [Mol Ther] Abstract | Press Release A New Methodological Sequence to Expand and Transdifferentiate Human Umbilical Cord Blood Derived CD133+ Cells into a Cardiomyocyte-Like Phenotype In this study researchers investigated optimizing a protocol for the expansion and subsequent differentiation of human umbilical cord blood derived CD133+ stem cells into a cardiomyocyte-like lineage. [Stem Cell Rev] Abstract | Press Release High-Resolution Imaging and Antitumor Effects of GFP+ Bone Marrow-Derived Cells Homing to Syngeneic Mouse Colon Tumors Investigators have established a mouse model for imaging bone marrow-derived cells from whole tumor to single-cell resolution, whereby the bone marrow of lethally irradiated host animals is reconstituted with EGFP-expressing bone marrow cells from matched TgActb(EGFP) donors. [Am J Pathol] Abstract | Press Release Reprogramming within Hours Following Nuclear Transfer into Mouse but Not Human Zygotes Using fertilized eggs, investigators found that when the zygotic genome was replaced with that of a somatic cell, development progressed normally throughout the cleavage stages, but then arrested before the morula stage. [Nat Commun] Abstract | Press Release
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