ESC & iPSC News Volume 10.10 | Mar 18 2015

Lymphoid Regeneration from Gene-Corrected SCID-X1 Subject-Derived iPSCs
X-linked severe combined immunodeficiency (SCID-X1) pathogenesis is due to mutations in the gene encoding the interleukin-2 receptor gamma chain, leading to a lack of functional lymphocytes. Researchers utilized induced pluripotent stem cell (iPSC) technology and genome editing mediated by TALENs to generate isogenic subject-specific mutant and gene-corrected iPSC lines. [Cell Stem Cell]
Abstract | Graphical Abstract | Press Release | Video

Smg6/Est1 Licenses Embryonic Stem Cell Differentiation via Nonsense-Mediated mRNA Decay
Scientists generated knockout mice lacking Smg6/Est1, a key nuclease in nonsense-mediated mRNA decay (NMD) and a telomerase cofactor. While the complete loss of Smg6 causes mouse lethality at the blastocyst stage, inducible deletion of Smg6 is compatible with embryonic stem cell proliferation despite the absence of telomere maintenance and functional NMD. [EMBO J] Full Article | Graphical Abstract

Cellular Reprogramming Allows Generation of Autologous Hematopoietic Progenitors from AML Patients that Are Devoid of Patient-Specific Genomic Aberrations
Skin fibroblasts were obtained from acute myeloid leukemia (AML) patients whose leukemic cells possessed a distinct, leukemia-associated aberration, and used to create AML patient-specific induced pluripotent stem cells. [Stem Cells] Abstract

New Protocol to Optimize iPS Cells for Genome Analysis of Fibrodysplasia Ossificans Progressiva
Investigators identified MMP1 and PAI1 as genes responsible for accelerating the chondrogenesis of fibrodysplasia ossificans progressiva-induced mesenchymal stromal cells. [Stem Cells] Abstract

Nr0b1 Is a Negative Regulator of Zscan4c in Mouse Embryonic Stem Cells
Scientists generated inducible knockout embryonic stem cells for nuclear receptor subfamily 0, group B, member 1 (Nr0b1) using the Cre-loxP system and analyzed its precise function. [Sci Rep] Full Article

Dual Optical Recordings for Action Potentials and Calcium Handling in Induced Pluripotent Stem Cell Models of Cardiac Arrhythmias Using Genetically Encoded Fluorescent Indicators
Investigators optimized a protocol for reprogramming of human fibroblasts and keratinocytes into pluripotency using single lipofection and the episomal vectors in a 24-well plate format. [Stem Cells Transl Med] Abstract

Pluripotent Cell Models of Fanconi Anemia Identify the Early Pathological Defect in Human Hemoangiogenic Progenitors
Scientists established induced pluripotent stem cells (iPSCs) from fibroblasts of six patients with Fanconi anemia (FA) and FANCA mutations. An improved reprogramming method yielded iPSC-like colonies from all patients, and iPSC clones were propagated from two patients. Quantitative evaluation of the differentiation ability demonstrated that the differentiation propensity toward the hematopoietic and endothelial lineages is already defective in early hemoangiogenic progenitors. [Stem Cells Transl Med] Abstract

Resveratrol Ameliorates the Maturation Process of β-Cell-Like Cells Obtained from an Optimized Differentiation Protocol of Human Embryonic Stem Cells
Investigators optimized an efficient differentiation protocol that mimics in vivo pancreatic organogenesis and investigated whether resveratrol may improve the final maturation step to obtain functional insulin-secreting cells. [PLoS One] Full Article

Inducible and Reversible Lentiviral and Recombination Mediated Cassette Exchange (RMCE) Systems for Controlling Gene Expression
Researchers developed broadly applicable platforms for reliable, tightly controlled and reversible Dox-inducible systems for lentiviral mediated generation of cell lines or FLP RMCE into the collagen 1a1 locus in mouse embryonic stem cells. [PLoS One] Full Article

Differentiation of Pluripotent Stem Cells into Endothelial Cells
Progress in differentiating endothelial cells from embryonic stem cell (ESC) and induced pluripotent stem cell (iPSC) has been made. The recent report of formation of endothelial colony forming cells from human ESC and iPSC provides a protocol that can generate clinically relevant numbers of cells for human cell therapy. [Curr Opin Hematol] Abstract

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WuXi PharmaTech to Build New Cell Therapy Manufacturing Facility for CAR T Cells and Other Cancer Immunotherapies
WuXi PharmaTech (Cayman) Inc. announced that it will soon begin construction of a new, 145,000-square-foot cGMP facility in Philadelphia for the manufacture of cell therapy products. [WuXi PharmaTech (Cayman) Inc.] Press Release

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Don’t Edit Embryos, Researchers Warn
Scientists should refrain from studies that alter the genome of human embryos, sperm, or egg cells, researchers warn in a commentary published in Nature. In it, they sound the alarm about new genome-editing techniques known as CRISPR and zinc-finger nucleases that make it much easier for scientists to delete, add, or change specific genes. [ScienceInsider] Editorial | Commentary

Stemming the Media Hype on Stem Cell Therapies
A new study by University of Alberta law researchers reveals sometimes overly optimistic news coverage of clinical translation of stem cell therapies — and as spokespeople, scientists need to be mindful of harnessing public expectations. [ScienceInsider] Press Release | Abstract

Reprieve for Australian Research Facilities
The Australian government has bowed to pressure from the scientific community to fund key national research infrastructure that had been under threat of closure. [Nature News] Editorial

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NEW Postdoctoral Position – Induced Pluripotent Stem Cells to Study Metabolism and Gene Expression Regulating Neural Tube Development (Joslin Diabetes Center)

Scientist – Reprogramming and Pluripotent Stem Cell Biology (STEMCELL Technologies Inc.)

Scientist – Pluripotent Stem Cell Biology Endoderm Lineages (STEMCELL Technologies Inc.)

Postdoctoral Associate – RNA Modification in Stem Cell Biology and Cancer Development (Sanford-Burnham Medical Research Institute)

Postdoctoral Positions – Interface of Physics and Stem Cell Biology (University of Copenhagen)

Postdoctoral Research Associate – Stem Cell Engineering (Rensselaer Polytechnic Institute)

Research Associate – Stem Cells and Regenerative Medicine (King’s College London)

Principal Investigators – Cell & Developmental Biology (University of Dundee)

Postdoctoral Position – Human Induced Pluripotent Stem Cells (hiPSCs) and the Peripheral Nervous System (Inserm)

Senior Process Development Engineer (Sangamo BioSciences, Inc.)

Director – Vector Production (Sangamo BioSciences, Inc.)

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