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Showing papers in "Cell Stem Cell in 2013"


Journal ArticleDOI
TL;DR: The CRISPR/Cas9 genome editing system is used to correct the CFTR locus by homologous recombination in cultured intestinal stem cells of CF patients and the corrected allele is expressed and fully functional as measured in clonally expanded organoids.

1,226 citations


Journal ArticleDOI
TL;DR: Current insights into the ways in which MSCs sense and control inflammation are outlined, highlighting the central role of macrophage polarization and progress toward clinical application is discussed.

1,086 citations


Journal ArticleDOI
TL;DR: This work evaluated mechanisms controlling oxidative state in primary specimens derived from acute myelogenous leukemia (AML) patients and proposed a model wherein the unique physiology of ROS-low LSCs provides an opportunity for selective targeting via disruption of BCL-2-dependent oxidative phosphorylation.

957 citations


Journal ArticleDOI
TL;DR: A nongenetic method for mass-producing cardiomyocytes from mouse and human PSC derivatives that is based on the marked biochemical differences in glucose and lactate metabolism between cardiomers and noncardiomyocyte and could facilitate progress toward PSC-based cardiac regenerative therapy.

805 citations


Journal ArticleDOI
TL;DR: Molecular and functional evidence linking Sox proteins with stem cell biology, cellular reprogramming, and disease with an emphasis on Sox2 is reviewed.

768 citations


Journal ArticleDOI
TL;DR: Differential manifestation of ER stress and DHA responsiveness may help explain variable clinical results obtained with the use of DHA treatment and suggests that DHA may in fact be effective for a subset of patients.

666 citations


Journal ArticleDOI
TL;DR: In this paper, a pyruvate dehydrogenase kinase (Pdk)-dependent mechanism was found to regulate glycolytic metabolic status governed by Pdk, which acts as a cell cycle checkpoint that modifies HSC quiescence and function.

636 citations


Journal ArticleDOI
TL;DR: It is shown that mice with a dominant mutation in Crygc gene that causes cataracts could be rescued by coinjection into zygotes of Cas9 mRNA and a single-guide RNA targeting the mutant allele, and were fertile and able to transmit the corrected allele to their progeny.

633 citations


Journal ArticleDOI
TL;DR: It is suggested that progerin-induced aging can be used to reveal late-onset age-related disease features in hiPSC-based disease models.

597 citations


Journal ArticleDOI
TL;DR: Patients generating patient-specific induced pluripotent stem cell cardiomyocytes from a ten-member family cohort carrying a hereditary HCM missense mutation (Arg663His) in the MYH7 gene are generated to help elucidate the mechanisms underlying HCM development and identify novel therapies for the disease.

567 citations



Journal ArticleDOI
TL;DR: It is indicated that human glia differentially enhance both activity-dependent plasticity and learning in mice.

Journal ArticleDOI
TL;DR: Four SLAM family markers, CD150, CD48, CD229, and CD244, can distinguish HSCs and MPPs from restricted progenitors and subdivide them into a hierarchy of functionally distinct subpopulations with stepwise changes in cell-cycle status, self-renewal, and reconstituting potential.

Journal ArticleDOI
TL;DR: Roles for thrombopoietin, CCL3, and direct cell-cell interactions in driving OBC expansion, and for changes in TGF-β, Notch, and inflammatory signaling in OBC remodeling are identified.

Journal ArticleDOI
TL;DR: This study defines the signals sufficient for modeling human ventral forebrain development in vitro and lays the foundation for studying cortical interneuron involvement in human disease pathology.

Journal ArticleDOI
TL;DR: The relative efficacies of CRISPRs and TALENs targeting the same genomic sites in the same hPSC lines are compared with the use of the same delivery platform as previously described.

Journal ArticleDOI
TL;DR: The speed and efficiency of myelination by hiPSC OPCs was higher than that previously observed using fetal-tissue-derived OPCS, and no tumors from these grafts were noted as long as 9 months after transplant, suggesting the potential utility of hiPSCs-derivedOPCs in treating disorders of myelin loss.

Journal ArticleDOI
TL;DR: It is found that early-stage progenitors progress via a radial glial-like stem cell enriched in the human fetal brain, and MGE-derived cortical interneuron deficiencies are implicated in a broad range of neurodevelopmental and degenerative disorders, highlighting the importance of these results for modeling human neural development and disease.


Journal ArticleDOI
TL;DR: The Lin28 pathway and its regulation is discussed, its roles in stem cells, tissue development, and pathogenesis are outlined, and the ramifications for re-engineering mammalian physiology are examined.

Journal ArticleDOI
TL;DR: WGBS analysis during adaptation of 2i ESCs to serum suggests that deposition ofDNA methylation is largely random, while loss of DNA methylation during reversion to 2i occurs passively, initiating at TET1 binding sites.

Journal ArticleDOI
TL;DR: It is found that age-associated HSC decline, replicative limits, and DNA methylation are largely dependent on the proliferative history of HSCs, yet appear to be telomere-length independent.

Journal ArticleDOI
TL;DR: It is shown by whole-genome bisulphite sequencing that 2i induces rapid and genome-wide demethylation on a scale and pattern similar to that in migratory PGCs and early embryos and provides a framework for understanding how signaling pathways regulate reprogramming to an epigenetic ground state of pluripotency.

Journal ArticleDOI
TL;DR: A combination of three small-molecule inhibitors is used to sustain hESCs in a LIF signaling-dependent hESC state (3iL h ESCs) with elevated expression of NANOG and epiblast-enriched genes such as KLF4, DPPA3, and TBX3.

Journal ArticleDOI
TL;DR: It is suggested that achieving true cardiac regeneration in patients may ultimately require resolution of critical controversies in experimental cardiac regeneration.

Journal ArticleDOI
TL;DR: Recent discoveries about the dynamic patterning and structural self-formation of complex organ buds in 3D stem cell culture, including the generation of various neuroectodermal and endodermal tissues are covered.

Journal ArticleDOI
TL;DR: It is shown that the tumor suppressor microRNA miR-34a is a cell-fate determinant in early-stage dividing colon cancer stem cells (CCSCs) and a unique microRNA-regulated mechanism that converts noisy input into a toggle switch for robust cell- fate decisions in CCSCs.

Journal ArticleDOI
TL;DR: Data support the idea that differentiated cells generated from autologous iPSCs could be applied for cell replacement therapy without eliciting immune rejection, and find little evidence of an immune response to undifferentiated, syngeneic iPSCS.

Journal ArticleDOI
TL;DR: It is found that RAC1 is required for expansion of the LGR5 intestinal stem cell (ISC) signature, progenitor hyperproliferation, and transformation, and Mechanistically, Rac1-driven ROS and NF-κB signaling mediate these processes.

Journal ArticleDOI
TL;DR: These findings provide a molecular basis for how cells with neural stem cell lineage emerge at sites of brain injury and imply that the high levels of SHH known to enter the brain from extraneural sources after invasive injury can trigger this response.