Abstract：
:Combination of stem cell-based approaches with gene-editing technologies represents an attractive strategy for studying human disease and developing therapies. However, gene-editing methodologies described to date for human cells suffer from technical limitations including limited target gene size, low targeting efficiency at transcriptionally inactive loci, and off-target genetic effects that could hamper broad clinical application. To address these limitations, and as a proof of principle, we focused on homologous recombination-based gene correction of multiple mutations on lamin A (LMNA), which are associated with various degenerative diseases. We show that helper-dependent adenoviral vectors (HDAdVs) provide a highly efficient and safe method for correcting mutations in large genomic regions in human induced pluripotent stem cells and can also be effective in adult human mesenchymal stem cells. This type of approach could be used to generate genotype-matched cell lines for disease modeling and drug discovery and potentially also in therapeutics.
journal_name
Cell Stem Celljournal_title
Cell stem cellauthors
Liu GH,Suzuki K,Qu J,Sancho-Martinez I,Yi F,Li M,Kumar S,Nivet E,Kim J,Soligalla RD,Dubova I,Goebl A,Plongthongkum N,Fung HL,Zhang K,Loring JF,Laurent LC,Izpisua Belmonte JCdoi
10.1016/j.stem.2011.04.019subject
Has Abstractpub_date
2011-06-03 00:00:00pages
688-94issue
6eissn
1934-5909issn
1875-9777pii
S1934-5909(11)00220-7journal_volume
8pub_type
杂志文章相关文献
Cell Stem Cell文献大全abstract：:In this issue of Cell Stem Cell, Palii et al. reveal that TAL1 is a master regulator of adhesion and migration networks in human endothelial progenitors and that ex vivo treatment with the histone deacetylase inhibitor TSA enables their faster vascularization after ischemic injury. ...
journal_title：Cell stem cell
pub_type： 评论,杂志文章
doi：10.1016/j.stem.2014.04.018
更新日期：2014-05-01 00:00:00
abstract：:Accessibility of human oocytes for research poses a serious ethical challenge to society. This fact categorically holds true when pursuing some of the most promising areas of research, such as somatic cell nuclear transfer and embryonic stem cell studies. One approach to overcoming this limitation is to use an oocyte ...
journal_title：Cell stem cell
pub_type： 杂志文章,评审
doi：10.1016/j.stem.2007.10.009
更新日期：2007-11-01 00:00:00
abstract：:A survey of U.S. stem cell scientists shows that uncertainty following the legal challenge to the Obama Administration's hESC research policy has negative scientific and economic impacts and affects a range of stem cell scientists, not just those working with hESCs. The international implications of these results are ...
journal_title：Cell stem cell
pub_type： 杂志文章
doi：10.1016/j.stem.2011.01.002
更新日期：2011-02-04 00:00:00
abstract：:Neural precursor cells (NPCs) reside in the subventricular zone in association with blood vessels and ependymal cells. In this issue of Cell Stem Cell, Kokovay et al. (2010) show that SDF1 directs the association of NPCs with this niche and regulates their lineage progression in a stage-specific manner. ...
journal_title：Cell stem cell
pub_type： 评论,杂志文章
doi：10.1016/j.stem.2010.07.001
更新日期：2010-08-06 00:00:00
abstract：:As a response against serious infection, there is often a need to produce more leukocytes to defend the organism against the infectious agent. In this issue of Cell Stem Cell, Goodell and colleagues (Feng et al., 2008) find a link between regulators of host defense and hematopoietic stem cells. ...
journal_title：Cell stem cell
pub_type： 评论,社论
doi：10.1016/j.stem.2007.12.002
更新日期：2008-01-10 00:00:00
abstract：:Structural and transcriptional changes during early brain maturation follow fixed developmental programs defined by genetics. However, whether this is true for functional network activity remains unknown, primarily due to experimental inaccessibility of the initial stages of the living human brain. Here, we developed ...
journal_title：Cell stem cell
pub_type： 杂志文章
doi：10.1016/j.stem.2019.08.002
更新日期：2019-10-03 00:00:00
abstract：:Human induced pluripotent stem cells (HiPSCs) appear to be highly similar to human embryonic stem cells (HESCs). Using two genetic lineage-tracing systems, we demonstrate the generation of iPSC lines from human pancreatic islet beta cells. These reprogrammed cells acquired markers of pluripotent cells and differentiat...
journal_title：Cell stem cell
pub_type： 杂志文章
doi：10.1016/j.stem.2011.06.007
更新日期：2011-07-08 00:00:00
abstract：:Despite widespread interest in using human induced pluripotent stem cells (hiPSCs) in neurological disease modeling, a suitable model system to study human neuronal connectivity is lacking. Here, we report a comprehensive and efficient differentiation paradigm for hiPSCs that generate multiple CA3 pyramidal neuron sub...
journal_title：Cell stem cell
pub_type： 杂志文章
doi：10.1016/j.stem.2018.04.009
更新日期：2018-05-03 00:00:00
abstract：:Two groups recently reported the in vitro differentiation of human embryonic stem cells into insulin-secreting cells, achieving an elusive goal for regenerative medicine. Herein we provide a perspective regarding these developments, compare phenotypes of the insulin-containing cells to human β cells, and discuss impli...
journal_title：Cell stem cell
pub_type： 杂志文章
doi：10.1016/j.stem.2014.10.012
更新日期：2014-11-06 00:00:00
abstract：:Regulation of hematopoietic stem cell (HSC) dormancy by specific cell types in the hematopoietic niche remains poorly understood. Yamazaki et al. (2011) now report that nerve-associated nonmyelinating Schwann cells activate TGF-β to maintain dormant HSCs, suggesting that glia are novel players in the bone marrow niche...
journal_title：Cell stem cell
pub_type： 评论,杂志文章
doi：10.1016/j.stem.2011.11.016
更新日期：2011-12-02 00:00:00
abstract：:Stem-cell-derived tissues could transform disease research and therapy, yet most methods generate functionally immature products. We investigate how human pluripotent stem cells (hPSCs) differentiate into pancreatic islets in vitro by profiling DNA methylation, chromatin accessibility, and histone modification changes...
journal_title：Cell stem cell
pub_type： 杂志文章
doi：10.1016/j.stem.2019.11.011
更新日期：2020-01-02 00:00:00
abstract：:Leukemia stem cells (LSCs) are resistant to current therapies used to treat chronic myeloid leukemia (CML). Abraham et al. (2016) have identified a molecular network critical for CML LSC survival and propose that simultaneously targeting two of their major transcriptional regulators, p53 and c-Myc, may facilitate thei...
journal_title：Cell stem cell
pub_type： 评论,杂志文章
doi：10.1016/j.stem.2016.06.005
更新日期：2016-07-07 00:00:00
abstract：:The mechanisms that regulate the establishment of adult stem cell pools during normal and perturbed mammalian development are still largely unknown. Here, we asked whether a maternal cytokine surge, which occurs during human maternal infections and has been implicated in cognitive disorders, might have long-lasting co...
journal_title：Cell stem cell
pub_type： 杂志文章
doi：10.1016/j.stem.2013.10.002
更新日期：2013-11-07 00:00:00
abstract：:Two recent papers in Cell Stem Cell by Soliman et al. (2020) and Scott et al. (2019) explore the contributions of mesenchymal progenitor cells to fibrosis in heart and skeletal muscle. They find tissue-dependent roles for a subpopulation of mesenchymal progenitors expressing Hic1 with varied differentiation capacities...
journal_title：Cell stem cell
pub_type： 评论,杂志文章
doi：10.1016/j.stem.2019.12.010
更新日期：2020-02-06 00:00:00
abstract：:N6-methyladenosine (m6A), the most abundant internal modification in mRNA, has been implicated in tumorigenesis. As an m6A demethylase, ALKBH5 has been shown to promote the development of breast cancer and brain tumors. However, in acute myeloid leukemia (AML), ALKBH5 was reported to be frequently deleted, implying a ...
journal_title：Cell stem cell
pub_type： 杂志文章
doi：10.1016/j.stem.2020.04.009
更新日期：2020-07-02 00:00:00
abstract：:Cell types more advanced in development than embryonic stem cells, such as EpiSCs, fail to contribute to chimeras when injected into pre-implantation-stage blastocysts, apparently because the injected cells undergo apoptosis. Here we show that transient promotion of cell survival through expression of the anti-apoptot...
journal_title：Cell stem cell
pub_type： 杂志文章
doi：10.1016/j.stem.2016.10.013
更新日期：2016-11-03 00:00:00
abstract：:In homeostasis of adult vertebrate tissues, stem cells are thought to self-renew by infrequent and asymmetric divisions that generate another stem cell daughter and a progenitor daughter cell committed to differentiate. This model is based largely on in vivo invertebrate or in vitro mammal studies. Here, we examine th...
journal_title：Cell stem cell
pub_type： 杂志文章
doi：10.1016/j.stem.2009.06.004
更新日期：2009-09-04 00:00:00
abstract：:Fanconi anemia (FA) is an inherited DNA repair disorder characterized by progressive bone marrow failure (BMF) from hematopoietic stem and progenitor cell (HSPC) attrition. A greater understanding of the pathogenesis of BMF could improve the therapeutic options for FA patients. Using a genome-wide shRNA screen in huma...
journal_title：Cell stem cell
pub_type： 杂志文章
doi：10.1016/j.stem.2016.03.002
更新日期：2016-05-05 00:00:00
abstract：:Cell-autonomous Wnt signaling has well-characterized functions in controlling stem cell activity, including in the prostate. While niche cells secrete Wnt ligands, the effects of Wnt signaling in niche cells per se are less understood. Here, we show that stromal cells in the proximal prostatic duct near the urethra, a...
journal_title：Cell stem cell
pub_type： 杂志文章
doi：10.1016/j.stem.2019.03.010
更新日期：2019-05-02 00:00:00
abstract：:Large cutaneous and non-healing ulcers exhibit impaired epithelialization, which represents a major therapeutic challenge. Recently in Nature, Kurita et al. (2018) reported a surprising potential source of epithelial cells. The authors demonstrate that wound-resident mesenchymal cells can be reprogrammed to generate e...
journal_title：Cell stem cell
pub_type： 评论,杂志文章
doi：10.1016/j.stem.2018.10.014
更新日期：2018-11-01 00:00:00
abstract：:Cell cycle regulators play critical roles in the balance between hematopoietic stem cell (HSC) dormancy and proliferation. In this study, we report that cell cycle entry proceeded normally in HSCs null for cyclin-dependent kinase (CDK) inhibitor p57 due to compensatory upregulation of p27. HSCs null for both p57 and p...
journal_title：Cell stem cell
pub_type： 杂志文章
doi：10.1016/j.stem.2011.07.003
更新日期：2011-09-02 00:00:00
abstract：:Understanding the mechanisms underlying neuronal fate determination will provide important insights into brain development and regenerative approaches to neurological diseases. Now in Cell Stem Cell, Masserdotti et al. (2015) use neuronal conversion of astrocytes to dissect transcriptional mechanisms of fate determina...
journal_title：Cell stem cell
pub_type： 评论,杂志文章
doi：10.1016/j.stem.2015.06.011
更新日期：2015-07-02 00:00:00
abstract：:Minority subpopulations within embryonic stem cell cultures display an expanded developmental potential similar to that of early embryo blastomeres or the early inner cell mass. The ability to isolate and culture totipotent cells capable of giving rise to the entire conceptus would enhance our capacity to study early ...
journal_title：Cell stem cell
pub_type： 杂志文章,评审
doi：10.1016/j.stem.2017.12.011
更新日期：2018-01-04 00:00:00
abstract：:In injured tissues, regeneration is often associated with cell fate plasticity, in that cells deviate from their normal lineage paths. It is becoming increasingly clear that this plasticity often creates alternative strategies to restore damaged or lost cells. Alternatively, cell fate plasticity is also part and parce...
journal_title：Cell stem cell
pub_type： 杂志文章,评审
doi：10.1016/j.stem.2018.05.009
更新日期：2018-09-06 00:00:00
abstract：:Epithelial-to-mesenchymal transition (EMT) is a developmental process important for cell fate determination. Fibroblasts, a product of EMT, can be reset into induced pluripotent stem cells (iPSCs) via exogenous transcription factors but the underlying mechanism is unclear. Here we show that the generation of iPSCs fro...
journal_title：Cell stem cell
pub_type： 杂志文章
doi：10.1016/j.stem.2010.04.014
更新日期：2010-07-02 00:00:00
abstract：:Clonal tracking of hematopoietic stem and progenitor cells (HSPCs) has proven valuable for studying their behavior in murine recipients. Now in Cell Stem Cell, Kim et al. (2014) and Wu et al. (2014) extend these analyses to nonhuman primates, providing insights into dynamics of HSPC expansion and lineage commitment fo...
journal_title：Cell stem cell
pub_type： 评论,杂志文章
doi：10.1016/j.stem.2014.03.006
更新日期：2014-04-03 00:00:00
abstract：:In this issue of Cell Stem Cell, Csaszar et al. (2012) develop a culture method that overcomes current limitations in ex vivo hematopoietic stem/progenitor cell expansion by continuously diluting inhibitory signaling factors and maintaining stem cell density. This approach enhances the generation of precursors with po...
journal_title：Cell stem cell
pub_type： 评论,杂志文章
doi：10.1016/j.stem.2012.01.012
更新日期：2012-02-03 00:00:00
abstract：:In response to muscle injury, satellite cells activate the p38α/β MAPK pathway to exit quiescence, then proliferate, repair skeletal muscle, and self-renew, replenishing the quiescent satellite cell pool. Although satellite cells are capable of asymmetric division, the mechanisms regulating satellite cell self-renewal...
journal_title：Cell stem cell
pub_type： 杂志文章
doi：10.1016/j.stem.2012.05.025
更新日期：2012-10-05 00:00:00
abstract：:The California Institute for Regenerative Medicine (CIRM) Alpha Stem Cell Clinic (ASCC) Network was launched in 2015 to address a compelling unmet medical need for rigorous, FDA-regulated, stem cell-related clinical trials for patients with challenging, incurable diseases. Here, we describe our multi-center experience...
journal_title：Cell stem cell
pub_type： 杂志文章
doi：10.1016/j.stem.2018.05.007
更新日期：2018-06-01 00:00:00
abstract：:Mouse embryonic stem cells (mESCs) cultured under serum/LIF conditions exhibit heterogeneous expression of pluripotency-associated factors that can be overcome by two inhibitors (2i) of the MEK and GSK3 pathways. Several studies have shown that the "ground state" induced by 2i is characterized by global hypomethylatio...
journal_title：Cell stem cell
pub_type： 杂志文章
doi：10.1016/j.stem.2015.07.005
更新日期：2015-10-01 00:00:00
