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  1 / 92 MEDLINE  
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[PMID]:29189772
[Au] Autor:Ukai H; Kiyonari H; Ueda HR
[Ad] Endereço:Laboratory for Synthetic Biology, RIKEN Quantitative Biology Center, Osaka, Japan.
[Ti] Título:Production of knock-in mice in a single generation from embryonic stem cells.
[So] Source:Nat Protoc;12(12):2513-2530, 2017 Dec.
[Is] ISSN:1750-2799
[Cp] País de publicação:England
[La] Idioma:eng
[Ab] Resumo:The system-level identification and analysis of molecular networks in mammals can be accelerated by 'next-generation' genetics, defined as genetics that does not require crossing of multiple generations of animals in order to achieve the desired genetic makeup. We have established a highly efficient procedure for producing knock-in (KI) mice within a single generation, by optimizing the genome-editing protocol for KI embryonic stem (ES) cells and the protocol for the generation of fully ES-cell-derived mice (ES mice). Using this protocol, the production of chimeric mice is eliminated, and, therefore, there is no requirement for the crossing of chimeric mice to produce mice that carry the KI gene in all cells of the body. Our procedure thus shortens the time required to produce KI ES mice from about a year to ∼3 months. Various kinds of KI ES mice can be produced with a minimized amount of work, facilitating the elucidation of organism-level phenomena using a systems biology approach. In this report, we describe the basic technologies and protocols for this procedure, and discuss the current challenges for next-generation mammalian genetics in organism-level systems biology studies.
[Mh] Termos MeSH primário: Edição de Genes/métodos
Técnicas de Introdução de Genes/métodos
Camundongos/genética
Células-Tronco Embrionárias Murinas/metabolismo
[Mh] Termos MeSH secundário: Animais
Linhagem Celular
Quimera/genética
Feminino
Vetores Genéticos/genética
Recombinação Homóloga
Camundongos Endogâmicos C57BL
Camundongos Endogâmicos ICR
Camundongos Transgênicos
Células-Tronco Embrionárias Murinas/citologia
Nucleases dos Efetores Semelhantes a Ativadores de Transcrição/genética
[Pt] Tipo de publicação:JOURNAL ARTICLE
[Nm] Nome de substância:
EC 3.1.- (Transcription Activator-Like Effector Nucleases)
[Em] Mês de entrada:1712
[Cu] Atualização por classe:171219
[Lr] Data última revisão:
171219
[Sb] Subgrupo de revista:IM
[Da] Data de entrada para processamento:171201
[St] Status:MEDLINE
[do] DOI:10.1038/nprot.2017.110


  2 / 92 MEDLINE  
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[PMID]:29053747
[Au] Autor:Matsuzaki Y; Sakuma T; Yamamoto T; Saya H
[Ad] Endereço:Division of Gene Regulation, Institute for Advanced Medical Research, School of Medicine, Keio University, Tokyo, Japan.
[Ti] Título:Establishment of pten knockout medaka with transcription activator-like effector nucleases (TALENs) as a model of PTEN deficiency disease.
[So] Source:PLoS One;12(10):e0186878, 2017.
[Is] ISSN:1932-6203
[Cp] País de publicação:United States
[La] Idioma:eng
[Ab] Resumo:Phosphatase and tensin homolog (PTEN) is a lipid and protein phosphatase that antagonizes signaling by the phosphatidylinositol 3-kinase (PI3K)-AKT signaling pathway. The PTEN gene is a major tumor suppressor, with mutations of this gene occurring frequently in tumors of humans and mice. We have now developed mutant medaka deficient in PTEN with the use of transcription activator-like effector nuclease (TALEN) technology. Medaka possesses two pten genes, ptena and ptenb, similar to zebrafish. We established 16 ptena mutant lines and two ptenb mutant lines. Homozygous single pten mutants were found to be viable and fertile. In contrast, pten double-knockout (dko) embryos manifested severe abnormalities in vasculogenesis, eye size, and tail development at 72 hours post fertilization(hpf) and died before hatching. Immunoblot analysis revealed that the ratio of phosphorylated to total forms of AKT (pAKT/AKT) in pten dko embryos was four times that in wild-type embryos, indicative of up-regulation of signaling by the PI3K-AKT pathway. Treatment of pten dko embryos with the PI3K inhibitor LY294002 reduced the pAKT/AKT ratio by about one-half and partially rescued the defect in vasculogenesis. Additional inhibitors of the PI3K-AKT pathway, including rapamycin and N-α-tosyl-L-phenylalanyl chloromethyl ketone, also partially restored vasculogenesis in the dko embryos. Our model system thus allows pten dko embryos to be readily distinguished from wild-type embryos at an early stage of development and is suitable for the screening of drugs able to compensate for PTEN deficiency.
[Mh] Termos MeSH primário: Técnicas de Silenciamento de Genes
Oryzias/genética
PTEN Fosfo-Hidrolase/genética
Nucleases dos Efetores Semelhantes a Ativadores de Transcrição/metabolismo
[Mh] Termos MeSH secundário: Animais
Sequência de Bases
Oryzias/embriologia
[Pt] Tipo de publicação:JOURNAL ARTICLE
[Nm] Nome de substância:
EC 3.1.- (Transcription Activator-Like Effector Nucleases); EC 3.1.3.67 (PTEN Phosphohydrolase)
[Em] Mês de entrada:1711
[Cu] Atualização por classe:171113
[Lr] Data última revisão:
171113
[Sb] Subgrupo de revista:IM
[Da] Data de entrada para processamento:171021
[St] Status:MEDLINE
[do] DOI:10.1371/journal.pone.0186878


  3 / 92 MEDLINE  
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[PMID]:28926587
[Au] Autor:Weh E; Takeuchi H; Muheisen S; Haltiwanger RS; Semina EV
[Ad] Endereço:Department of Pediatrics and Children's Research Institute at the Medical College of Wisconsin, Milwaukee, Wisconsin, United States of America.
[Ti] Título:Functional characterization of zebrafish orthologs of the human Beta 3-Glucosyltransferase B3GLCT gene mutated in Peters Plus Syndrome.
[So] Source:PLoS One;12(9):e0184903, 2017.
[Is] ISSN:1932-6203
[Cp] País de publicação:United States
[La] Idioma:eng
[Ab] Resumo:Peters Plus Syndrome (PPS) is a rare autosomal recessive disease characterized by ocular defects, short stature, brachydactyly, characteristic facial features, developmental delay and other highly variable systemic defects. Classic PPS is caused by loss-of-function mutations in the B3GLCT gene encoding for a ß3-glucosyltransferase that catalyzes the attachment of glucose via a ß1-3 glycosidic linkage to O-linked fucose on thrombospondin type 1 repeats (TSRs). B3GLCT was shown to participate in a non-canonical ER quality control mechanism; however, the exact molecular processes affected in PPS are not well understood. Here we report the identification and characterization of two zebrafish orthologs of the human B3GLCT gene, b3glcta and b3glctb. The b3glcta and b3glctb genes encode for 496-aa and 493-aa proteins with 65% and 57% identity to human B3GLCT, respectively. Expression studies demonstrate that both orthologs are widely expressed with strong presence in embryonic tissues affected in PPS. In vitro glucosylation assays demonstrated that extracts from wildtype embryos contain active b3glct enzyme capable of transferring glucose from UDP-glucose to an O-fucosylated TSR, indicating functional conservation with human B3GLCT. To determine the developmental role of the zebrafish genes, single and double b3glct knockouts were generated using TALEN-induced genome editing. Extracts from double homozygous b3glct-/- embryos demonstrated complete loss of in vitro b3glct activity. Surprisingly, b3glct-/- homozygous fish developed normally. Transcriptome analyses of head and trunk tissues of b3glct-/- 24-hpf embryos identified 483 shared differentially regulated transcripts that may be involved in compensation for b3glct function in these embryos. The presented data show that both sequence and function of B3GLCT/b3glct genes is conserved in vertebrates. At the same time, complete b3glct deficiency in zebrafish appears to be inconsequential and possibly compensated for by a yet unknown mechanism.
[Mh] Termos MeSH primário: Glucosiltransferases/metabolismo
Proteínas de Peixe-Zebra/metabolismo
[Mh] Termos MeSH secundário: Sequência de Aminoácidos
Animais
Fenda Labial/genética
Fenda Labial/patologia
Córnea/anormalidades
Córnea/patologia
Embrião não Mamífero/metabolismo
Edição de Genes
Perfilação da Expressão Gênica
Técnicas de Inativação de Genes
Glucosiltransferases/deficiência
Glucosiltransferases/genética
Transtornos do Crescimento/genética
Transtornos do Crescimento/patologia
Seres Humanos
Hibridização In Situ
Deformidades Congênitas dos Membros/genética
Deformidades Congênitas dos Membros/patologia
Dados de Sequência Molecular
Mutação
Alinhamento de Sequência
Nucleases dos Efetores Semelhantes a Ativadores de Transcrição/genética
Peixe-Zebra
Proteínas de Peixe-Zebra/deficiência
Proteínas de Peixe-Zebra/genética
[Pt] Tipo de publicação:JOURNAL ARTICLE
[Nm] Nome de substância:
0 (Zebrafish Proteins); EC 2.4.1.- (1,3-alpha-D-glucan synthase); EC 2.4.1.- (Glucosyltransferases); EC 3.1.- (Transcription Activator-Like Effector Nucleases)
[Em] Mês de entrada:1710
[Cu] Atualização por classe:171025
[Lr] Data última revisão:
171025
[Sb] Subgrupo de revista:IM
[Da] Data de entrada para processamento:170920
[St] Status:MEDLINE
[do] DOI:10.1371/journal.pone.0184903


  4 / 92 MEDLINE  
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[PMID]:28846101
[Au] Autor:Jachowicz JW; Bing X; Pontabry J; Boskovic A; Rando OJ; Torres-Padilla ME
[Ad] Endereço:Institute of Epigenetics and Stem Cells, Helmholtz Zentrum München, München, Germany.
[Ti] Título:LINE-1 activation after fertilization regulates global chromatin accessibility in the early mouse embryo.
[So] Source:Nat Genet;49(10):1502-1510, 2017 Oct.
[Is] ISSN:1546-1718
[Cp] País de publicação:United States
[La] Idioma:eng
[Ab] Resumo:After fertilization, to initiate development, gametes are reprogramed to become totipotent. Approximately half of the mammalian genome consists of repetitive elements, including retrotransposons, some of which are transcribed after fertilization. Retrotransposon activation is generally assumed to be a side effect of the extensive chromatin remodeling underlying the epigenetic reprogramming of gametes. Here, we used a targeted epigenomic approach to address whether specific retrotransposon families play a direct role in chromatin organization and developmental progression. We demonstrate that premature silencing of LINE-1 elements decreases chromatin accessibility, whereas prolonged activation prevents the gradual chromatin compaction that occurs naturally in developmental progression. Preventing LINE-1 activation and interfering with its silencing decreases developmental rates independently of the coding nature of the LINE-1 transcript, thus suggesting that LINE-1 functions primarily at the chromatin level. Our data suggest that activation of LINE-1 regulates global chromatin accessibility at the beginning of development and indicate that retrotransposon activation is integral to the developmental program.
[Mh] Termos MeSH primário: Blástula/metabolismo
Montagem e Desmontagem da Cromatina/genética
Desenvolvimento Embrionário/genética
Regulação da Expressão Gênica no Desenvolvimento
Elementos Nucleotídeos Longos e Dispersos/fisiologia
Zigoto/metabolismo
[Mh] Termos MeSH secundário: Animais
Cruzamentos Genéticos
Técnicas de Cultura Embrionária
Feminino
Fertilização
Inativação Gênica
Seres Humanos
Masculino
Camundongos
Camundongos Endogâmicos C57BL
Camundongos Endogâmicos CBA
Técnicas Analíticas Microfluídicas
RNA Mensageiro/genética
Nucleases dos Efetores Semelhantes a Ativadores de Transcrição
Transcrição Genética
[Pt] Tipo de publicação:JOURNAL ARTICLE
[Nm] Nome de substância:
0 (RNA, Messenger); EC 3.1.- (Transcription Activator-Like Effector Nucleases)
[Em] Mês de entrada:1710
[Cu] Atualização por classe:171024
[Lr] Data última revisão:
171024
[Sb] Subgrupo de revista:IM
[Da] Data de entrada para processamento:170829
[St] Status:MEDLINE
[do] DOI:10.1038/ng.3945


  5 / 92 MEDLINE  
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[PMID]:28777806
[Au] Autor:Tokuda S; Hirai T; Furuse M
[Ad] Endereço:Department of Respiratory Medicine, Graduate School of Medicine, Kyoto University, Kyoto, Japan.
[Ti] Título:Claudin-4 knockout by TALEN-mediated gene targeting in MDCK cells: Claudin-4 is dispensable for the permeability properties of tight junctions in wild-type MDCK cells.
[So] Source:PLoS One;12(8):e0182521, 2017.
[Is] ISSN:1932-6203
[Cp] País de publicação:United States
[La] Idioma:eng
[Ab] Resumo:Epithelia act as a barrier between the internal and external environments, and the movement of substances via the paracellular pathway is regulated by tight junctions (TJs). Claudins are major determinants of TJ permeability. Claudin-4 was the first claudin whose involvement in the TJ permeability in cultured cells was directly demonstrated, but the permeability properties of individual claudins including claudin-4 are still incompletely clarified. In this study, we established claudin-4 knockout cells using transcription activator-like effector nucleases (TALENs), a recently developed method for genome editing, and investigated the permeability property of claudin-4 in MDCK II cells. We found that claudin-4 knockout has no apparent effect on the localization of other claudins and electrophysiological properties in MDCK II cells. Therefore we further established claudin-2 and claudin-4 double knockout clones and investigated the effects on TJs. Claudin-4 knockout in addition to claudin-2 knockout slightly increased the localization of other claudins at TJs but showed no obvious effects on the electrophysiological properties in MDCK II cells. These results indicate that claudin-4 is dispensable for the barrier property of TJs in wild-type as well as claudin-2 knockout MDCK II cells. Our results suggest the need for further knockout analysis to reveal the permeability properties of individual claudins.
[Mh] Termos MeSH primário: Permeabilidade da Membrana Celular
Claudina-4/antagonistas & inibidores
Técnicas de Inativação de Genes/métodos
Junções Íntimas/fisiologia
Nucleases dos Efetores Semelhantes a Ativadores de Transcrição/metabolismo
[Mh] Termos MeSH secundário: Animais
Claudina-4/genética
Claudina-4/metabolismo
Cães
Células Madin Darby de Rim Canino
[Pt] Tipo de publicação:JOURNAL ARTICLE
[Nm] Nome de substância:
0 (Claudin-4); EC 3.1.- (Transcription Activator-Like Effector Nucleases)
[Em] Mês de entrada:1710
[Cu] Atualização por classe:171010
[Lr] Data última revisão:
171010
[Sb] Subgrupo de revista:IM
[Da] Data de entrada para processamento:170805
[St] Status:MEDLINE
[do] DOI:10.1371/journal.pone.0182521


  6 / 92 MEDLINE  
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[PMID]:28750036
[Au] Autor:Wang X; Yamada S; LaRiviere WB; Ye H; Bakeberg JL; Irazabal MV; Chebib FT; van Deursen J; Harris PC; Sussman CR; Behfar A; Ward CJ; Torres VE
[Ad] Endereço:Division of Nephrology and Hypertension, Mayo Clinic, Rochester, Minnesota, United States of America.
[Ti] Título:Generation and phenotypic characterization of Pde1a mutant mice.
[So] Source:PLoS One;12(7):e0181087, 2017.
[Is] ISSN:1932-6203
[Cp] País de publicação:United States
[La] Idioma:eng
[Ab] Resumo:It has been proposed that a reduction in intracellular calcium causes an increase in intracellular cAMP and PKA activity through stimulation of calcium inhibitable adenylyl cyclase 6 and inhibition of phosphodiesterase 1 (PDE1), the main enzymes generating and degrading cAMP in the distal nephron and collecting duct, thus contributing to the development and progression of autosomal dominant polycystic kidney disease (ADPKD). In zebrafish pde1a depletion aggravates and overexpression ameliorates the cystic phenotype. To study the role of PDE1A in a mammalian system, we used a TALEN pair to Pde1a exon 7, targeting the histidine-aspartic acid dipeptide involved in ligating the active site Zn++ ion to generate two Pde1a null mouse lines. Pde1a mutants had a mild renal cystic disease and a urine concentrating defect (associated with upregulation of PDE4 activity and decreased protein kinase A dependent phosphorylation of aquaporin-2) on a wild-type genetic background and aggravated renal cystic disease on a Pkd2WS25/- background. Pde1a mutants additionally had lower aortic blood pressure and increased left ventricular (LV) ejection fraction, without a change in LV mass index, consistent with the high aortic and low cardiac expression of Pde1a in wild-type mice. These results support an important role of PDE1A in the renal pathogenesis of ADPKD and in the regulation of blood pressure.
[Mh] Termos MeSH primário: Nucleotídeo Cíclico Fosfodiesterase do Tipo 1/metabolismo
Rim Policístico Autossômico Dominante/metabolismo
Rim Policístico Autossômico Dominante/patologia
[Mh] Termos MeSH secundário: Animais
Pressão Sanguínea
Peso Corporal
Sistema Cardiovascular/metabolismo
Sistema Cardiovascular/patologia
Sistema Cardiovascular/fisiopatologia
AMP Cíclico/metabolismo
Desamino Arginina Vasopressina/metabolismo
Homozigoto
Rim/metabolismo
Camundongos
Camundongos Knockout
Camundongos Mutantes
Miocárdio/metabolismo
Miocárdio/patologia
Tamanho do Órgão
Fenótipo
Rim Policístico Autossômico Dominante/fisiopatologia
Antígeno Nuclear de Célula em Proliferação/metabolismo
Nucleases dos Efetores Semelhantes a Ativadores de Transcrição/metabolismo
[Pt] Tipo de publicação:JOURNAL ARTICLE
[Nm] Nome de substância:
0 (Proliferating Cell Nuclear Antigen); E0399OZS9N (Cyclic AMP); EC 3.1.- (Transcription Activator-Like Effector Nucleases); EC 3.1.4.17 (Cyclic Nucleotide Phosphodiesterases, Type 1); EC 3.1.4.17 (Pde1A protein, mouse); ENR1LLB0FP (Deamino Arginine Vasopressin)
[Em] Mês de entrada:1709
[Cu] Atualização por classe:170929
[Lr] Data última revisão:
170929
[Sb] Subgrupo de revista:IM
[Da] Data de entrada para processamento:170728
[St] Status:MEDLINE
[do] DOI:10.1371/journal.pone.0181087


  7 / 92 MEDLINE  
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[PMID]:28666380
[Au] Autor:Luo W; Galvan DL; Woodard LE; Dorset D; Levy S; Wilson MH
[Ad] Endereço:Department of Veterans Affairs, Nashville, TN 37212 USA and Department of Medicine, Department of Pharmacology, Vanderbilt University Medical Center, Nashville, TN 37232, USA.
[Ti] Título:Comparative analysis of chimeric ZFP-, TALE- and Cas9-piggyBac transposases for integration into a single locus in human cells.
[So] Source:Nucleic Acids Res;45(14):8411-8422, 2017 Aug 21.
[Is] ISSN:1362-4962
[Cp] País de publicação:England
[La] Idioma:eng
[Ab] Resumo:Integrating DNA delivery systems hold promise for many applications including treatment of diseases; however, targeted integration is needed for improved safety. The piggyBac (PB) transposon system is a highly active non-viral gene delivery system capable of integrating defined DNA segments into host chromosomes without requiring homologous recombination. We systematically compared four different engineered zinc finger proteins (ZFP), four transcription activator-like effector proteins (TALE), CRISPR associated protein 9 (SpCas9) and the catalytically inactive dSpCas9 protein fused to the amino-terminus of the transposase enzyme designed to target the hypoxanthine phosphoribosyltransferase (HPRT) gene located on human chromosome X. Chimeric transposases were evaluated for expression, transposition activity, chromatin immunoprecipitation at the target loci, and targeted knockout of the HPRT gene in human cells. One ZFP-PB and one TALE-PB chimera demonstrated notable HPRT gene targeting. In contrast, Cas9/dCas9-PB chimeras did not result in gene targeting. Instead, the HPRT locus appeared to be protected from transposon integration. Supplied separately, PB permitted highly efficient isolation of Cas9-mediated knockout of HPRT, with zero transposon integrations in HPRT by deep sequencing. In summary, these tools may allow isolation of 'targeted-only' cells, be utilized to protect a genomic locus from transposon integration, and enrich for Cas9-mutated cells.
[Mh] Termos MeSH primário: Técnicas de Inativação de Genes/métodos
Marcação de Genes/métodos
Técnicas de Transferência de Genes
Mutagênese Insercional/métodos
[Mh] Termos MeSH secundário: Proteínas de Bactérias/genética
Sistemas CRISPR-Cas/genética
Linhagem Celular Tumoral
Elementos de DNA Transponíveis/genética
Endonucleases/genética
Seres Humanos
Hipoxantina Fosforribosiltransferase/genética
Hipoxantina Fosforribosiltransferase/metabolismo
Proteínas Recombinantes de Fusão/genética
Reprodutibilidade dos Testes
Nucleases dos Efetores Semelhantes a Ativadores de Transcrição/genética
Efetores Semelhantes a Ativadores de Transcrição/genética
Transposases/genética
Dedos de Zinco/genética
[Pt] Tipo de publicação:COMPARATIVE STUDY; JOURNAL ARTICLE
[Nm] Nome de substância:
0 (Bacterial Proteins); 0 (DNA Transposable Elements); 0 (Recombinant Fusion Proteins); 0 (Transcription Activator-Like Effectors); EC 2.4.2.8 (Hypoxanthine Phosphoribosyltransferase); EC 2.7.7.- (Transposases); EC 3.1.- (Cas9 endonuclease Streptococcus pyogenes); EC 3.1.- (Endonucleases); EC 3.1.- (Transcription Activator-Like Effector Nucleases)
[Em] Mês de entrada:1710
[Cu] Atualização por classe:171023
[Lr] Data última revisão:
171023
[Sb] Subgrupo de revista:IM
[Da] Data de entrada para processamento:170702
[St] Status:MEDLINE
[do] DOI:10.1093/nar/gkx572


  8 / 92 MEDLINE  
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[PMID]:28525759
[Au] Autor:Chen Y; Yu J; Niu Y; Qin D; Liu H; Li G; Hu Y; Wang J; Lu Y; Kang Y; Jiang Y; Wu K; Li S; Wei J; He J; Wang J; Liu X; Luo Y; Si C; Bai R; Zhang K; Liu J; Huang S; Chen Z; Wang S; Chen X; Bao X; Zhang Q; Li F; Geng R; Liang A; Shen D; Jiang T; Hu X; Ma Y; Ji W; Sun YE
[Ad] Endereço:Yunnan Key Laboratory of Primate Biomedicine Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming 650500, China; Yunnan Provincial Academy of Science and Technology, Kunming 650051, China; Kunming Enovate Institute of Bioscience, Kunming 650000
[Ti] Título:Modeling Rett Syndrome Using TALEN-Edited MECP2 Mutant Cynomolgus Monkeys.
[So] Source:Cell;169(5):945-955.e10, 2017 May 18.
[Is] ISSN:1097-4172
[Cp] País de publicação:United States
[La] Idioma:eng
[Ab] Resumo:Gene-editing technologies have made it feasible to create nonhuman primate models for human genetic disorders. Here, we report detailed genotypes and phenotypes of TALEN-edited MECP2 mutant cynomolgus monkeys serving as a model for a neurodevelopmental disorder, Rett syndrome (RTT), which is caused by loss-of-function mutations in the human MECP2 gene. Male mutant monkeys were embryonic lethal, reiterating that RTT is a disease of females. Through a battery of behavioral analyses, including primate-unique eye-tracking tests, in combination with brain imaging via MRI, we found a series of physiological, behavioral, and structural abnormalities resembling clinical manifestations of RTT. Moreover, blood transcriptome profiling revealed that mutant monkeys resembled RTT patients in immune gene dysregulation. Taken together, the stark similarity in phenotype and/or endophenotype between monkeys and patients suggested that gene-edited RTT founder monkeys would be of value for disease mechanistic studies as well as development of potential therapeutic interventions for RTT.
[Mh] Termos MeSH primário: Proteína 2 de Ligação a Metil-CpG/genética
Síndrome de Rett/genética
[Mh] Termos MeSH secundário: Animais
Encéfalo/fisiologia
Cromossomos Humanos X
Ritmo Circadiano
Modelos Animais de Doenças
Eletrocardiografia
Feminino
Edição de Genes
Seres Humanos
Macaca fascicularis
Imagem por Ressonância Magnética
Masculino
Mutação
Dor
Síndrome de Rett/fisiopatologia
Sono
Nucleases dos Efetores Semelhantes a Ativadores de Transcrição/metabolismo
Transcriptoma
[Pt] Tipo de publicação:JOURNAL ARTICLE
[Nm] Nome de substância:
0 (Methyl-CpG-Binding Protein 2); EC 3.1.- (Transcription Activator-Like Effector Nucleases)
[Em] Mês de entrada:1707
[Cu] Atualização por classe:170804
[Lr] Data última revisão:
170804
[Sb] Subgrupo de revista:IM
[Da] Data de entrada para processamento:170520
[St] Status:MEDLINE


  9 / 92 MEDLINE  
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[PMID]:28489428
[Au] Autor:Cuculis L; Schroeder CM
[Ad] Endereço:Department of Chemistry, University of Illinois at Urbana-Champaign, Urbana, Illinois 61801; email: cms@illinois.edu.
[Ti] Título:A Single-Molecule View of Genome Editing Proteins: Biophysical Mechanisms for TALEs and CRISPR/Cas9.
[So] Source:Annu Rev Chem Biomol Eng;8:577-597, 2017 Jun 07.
[Is] ISSN:1947-5446
[Cp] País de publicação:United States
[La] Idioma:eng
[Ab] Resumo:Exciting new advances in genome engineering have unlocked the potential to radically alter the treatment of human disease. In this review, we discuss the application of single-molecule techniques to uncover the mechanisms behind two premier classes of genome editing proteins: transcription activator-like effector nucleases (TALENs) and the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated system (Cas). These technologies have facilitated a striking number of gene editing applications in a variety of organisms; however, we are only beginning to understand the molecular mechanisms governing the DNA editing properties of these systems. Here, we discuss the DNA search and recognition process for TALEs and Cas9 that have been revealed by recent single-molecule experiments.
[Mh] Termos MeSH primário: Sistemas CRISPR-Cas
Edição de Genes/métodos
Imagem Individual de Molécula/métodos
Nucleases dos Efetores Semelhantes a Ativadores de Transcrição/análise
[Mh] Termos MeSH secundário: Animais
Repetições Palindrômicas Curtas Agrupadas e Regularmente Espaçadas
DNA/análise
DNA/genética
DNA/metabolismo
Genoma
Seres Humanos
Imagem Óptica/métodos
Espectrometria de Fluorescência/métodos
Análise Espectral/métodos
Nucleases dos Efetores Semelhantes a Ativadores de Transcrição/metabolismo
[Pt] Tipo de publicação:JOURNAL ARTICLE; REVIEW
[Nm] Nome de substância:
9007-49-2 (DNA); EC 3.1.- (Transcription Activator-Like Effector Nucleases)
[Em] Mês de entrada:1707
[Cu] Atualização por classe:170727
[Lr] Data última revisão:
170727
[Sb] Subgrupo de revista:IM
[Da] Data de entrada para processamento:170511
[St] Status:MEDLINE
[do] DOI:10.1146/annurev-chembioeng-060816-101603


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[PMID]:28405721
[Au] Autor:Chandrasekaran AP; Song M; Ramakrishna S
[Ad] Endereço:Graduate School of Biomedical Science and Engineering, Hanyang University, Seoul, Republic of Korea.
[Ti] Título:Genome editing: a robust technology for human stem cells.
[So] Source:Cell Mol Life Sci;74(18):3335-3346, 2017 Sep.
[Is] ISSN:1420-9071
[Cp] País de publicação:Switzerland
[La] Idioma:eng
[Ab] Resumo:Human pluripotent stem cells comprise induced pluripotent and embryonic stem cells, which have tremendous potential for biological and therapeutic applications. The development of efficient technologies for the targeted genome alteration of stem cells in disease models is a prerequisite for utilizing stem cells to their full potential. Genome editing of stem cells is possible with the help of synthetic nucleases that facilitate site-specific modification of a gene of interest. Recent advances in genome editing techniques have improved the efficiency and speed of the development of stem cells for human disease models. Zinc finger nucleases, transcription activator-like effector nucleases, and clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated system are powerful tools for editing DNA at specific loci. Here, we discuss recent technological advances in genome editing with site-specific nucleases in human stem cells.
[Mh] Termos MeSH primário: Edição de Genes
Células-Tronco/metabolismo
[Mh] Termos MeSH secundário: Proteína da Polipose Adenomatosa do Colo/genética
Proteína da Polipose Adenomatosa do Colo/metabolismo
Sistemas CRISPR-Cas/genética
Endodesoxirribonucleases/metabolismo
Genoma Humano
Seres Humanos
Receptores CCR5/genética
Receptores CCR5/metabolismo
Células-Tronco/citologia
Nucleases dos Efetores Semelhantes a Ativadores de Transcrição/genética
Nucleases dos Efetores Semelhantes a Ativadores de Transcrição/metabolismo
Proteína Supressora de Tumor p53/genética
Proteína Supressora de Tumor p53/metabolismo
Proteínas ras/genética
Proteínas ras/metabolismo
[Pt] Tipo de publicação:JOURNAL ARTICLE; REVIEW
[Nm] Nome de substância:
0 (Adenomatous Polyposis Coli Protein); 0 (Receptors, CCR5); 0 (Tumor Suppressor Protein p53); EC 3.1.- (Endodeoxyribonucleases); EC 3.1.- (Transcription Activator-Like Effector Nucleases); EC 3.6.5.2 (ras Proteins)
[Em] Mês de entrada:1708
[Cu] Atualização por classe:170830
[Lr] Data última revisão:
170830
[Sb] Subgrupo de revista:IM
[Da] Data de entrada para processamento:170414
[St] Status:MEDLINE
[do] DOI:10.1007/s00018-017-2522-0



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