标题
Pluripotent stem cells in disease modelling and drug discovery
作者
关键词
-
出版物
NATURE REVIEWS MOLECULAR CELL BIOLOGY
Volume 17, Issue 3, Pages 170-182
出版商
Springer Nature
发表日期
2016-01-28
DOI
10.1038/nrm.2015.27
参考文献
相关参考文献
注意：仅列出部分参考文献，下载原文获取全部文献信息。- Antisense oligonucleotides in therapy for neurodegenerative disorders
- (2015) Melvin M. Evers et al. ADVANCED DRUG DELIVERY REVIEWS
- Induced pluripotent stem cell-derived neuronal cells from a sporadic Alzheimer’s disease donor as a model for investigating AD-associated gene regulatory networks
- (2015) Amir M Hossini et al. BMC GENOMICS
- Programming and Reprogramming Cellular Age in the Era of Induced Pluripotency
- (2015) Lorenz Studer et al. Cell Stem Cell
- Genome-wide RNA-Seq of Human Motor Neurons Implicates Selective ER Stress Activation in Spinal Muscular Atrophy
- (2015) Shi-Yan Ng et al. Cell Stem Cell
- Elucidating Molecular Phenotypes Caused by the SORL1 Alzheimer’s Disease Genetic Risk Factor Using Human Induced Pluripotent Stem Cells
- (2015) Jessica E. Young et al. Cell Stem Cell
- From Dish to Bedside: Lessons Learned While Translating Findings from a Stem Cell Model of Disease to a Clinical Trial
- (2015) John McNeish et al. Cell Stem Cell
- Modeling mouse and human development using organoid cultures
- (2015) M. Huch et al. DEVELOPMENT
- A novel human model of the neurodegenerative disease GM1 gangliosidosis using induced pluripotent stem cells demonstrates inflammasome activation
- (2015) Mi-Young Son et al. JOURNAL OF PATHOLOGY
- Characterization of bipolar disorder patient-specific induced pluripotent stem cells from a family reveals neurodevelopmental and mRNA expression abnormalities
- (2015) J M Madison et al. MOLECULAR PSYCHIATRY
- Targeted disruption of DNMT1, DNMT3A and DNMT3B in human embryonic stem cells
- (2015) Jing Liao et al. NATURE GENETICS
- Therapeutic genome editing: prospects and challenges
- (2015) David Benjamin Turitz Cox et al. NATURE MEDICINE
- Automated, high-throughput derivation, characterization and differentiation of induced pluripotent stem cells
- (2015) Daniel Paull et al. NATURE METHODS
- HPGCD Outperforms HPBCD as a Potential Treatment for Niemann-Pick Disease Type C During Disease Modeling with iPS Cells
- (2015) Minami Soga et al. STEM CELLS
- N-butylidenephthalide Attenuates Alzheimer's Disease-Like Cytopathy in Down Syndrome Induced Pluripotent Stem Cell-Derived Neurons
- (2015) Chia-Yu Chang et al. Scientific Reports
- Molecular Mechanisms Regulating the Defects in Fragile X Syndrome Neurons Derived from Human Pluripotent Stem Cells
- (2015) Tomer Halevy et al. Stem Cell Reports
- Modeling the Early Phenotype at the Neuromuscular Junction of Spinal Muscular Atrophy Using Patient-Derived iPSCs
- (2015) Michiko Yoshida et al. Stem Cell Reports
- Unveiling a common mechanism of apoptosis in β-cells and neurons in Friedreich's ataxia
- (2015) Mariana Igoillo-Esteve et al. HUMAN MOLECULAR GENETICS
- Epigenetic therapy for Friedreich ataxia
- (2014) Elisabetta Soragni et al. ANNALS OF NEUROLOGY
- Comparable Frequencies of Coding Mutations and Loss of Imprinting in Human Pluripotent Cells Derived by Nuclear Transfer and Defined Factors
- (2014) Bjarki Johannesson et al. Cell Stem Cell
- Pathways Disrupted in Human ALS Motor Neurons Identified through Genetic Correction of Mutant SOD1
- (2014) Evangelos Kiskinis et al. Cell Stem Cell
- Mutant astrocytes differentiated from Rett syndrome patients-specific iPSCs have adverse effects on wild-type neurons
- (2014) Emily Cunningham Williams et al. HUMAN MOLECULAR GENETICS
- Induced pluripotent stem cells as a model for telomeric abnormalities in ICF type I syndrome
- (2014) Shira Sagie et al. HUMAN MOLECULAR GENETICS
- Selective demethylation and altered gene expression are associated with ICF syndrome in human-induced pluripotent stem cells and mesenchymal stem cells
- (2014) Kevin Huang et al. HUMAN MOLECULAR GENETICS
- Reactivation of maternal SNORD116 cluster via SETDB1 knockdown in Prader-Willi syndrome iPSCs
- (2014) E. Cruvinel et al. HUMAN MOLECULAR GENETICS
- Validation of genome-wide association study (GWAS)-identified disease risk alleles with patient-specific stem cell lines
- (2014) Jin Yang et al. HUMAN MOLECULAR GENETICS
- Genome maintenance in pluripotent stem cells
- (2014) Uri Weissbein et al. JOURNAL OF CELL BIOLOGY
- Modeling non-syndromic autism and the impact of TRPC6 disruption in human neurons
- (2014) K Griesi-Oliveira et al. MOLECULAR PSYCHIATRY
- Human oocytes reprogram adult somatic nuclei of a type 1 diabetic to diploid pluripotent stem cells
- (2014) Mitsutoshi Yamada et al. NATURE
- Autophagy induction enhances TDP43 turnover and survival in neuronal ALS models
- (2014) Sami J Barmada et al. Nature Chemical Biology
- The noncoding RNA IPW regulates the imprinted DLK1-DIO3 locus in an induced pluripotent stem cell model of Prader-Willi syndrome
- (2014) Yonatan Stelzer et al. NATURE GENETICS
- A guide to genome engineering with programmable nucleases
- (2014) Hyongbum Kim et al. NATURE REVIEWS GENETICS
- Investigating human disease using stem cell models
- (2014) Jared L. Sterneckert et al. NATURE REVIEWS GENETICS
- The role of genomic imprinting in biology and disease: an expanding view
- (2014) Jo Peters NATURE REVIEWS GENETICS
- A calcium-dependent protease as a potential therapeutic target for Wolfram syndrome
- (2014) Simin Lu et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Valproate reverts zinc and potassium imbalance in schizophrenia-derived reprogrammed cells
- (2014) Bruna da Silveira Paulsen et al. SCHIZOPHRENIA RESEARCH
- Organogenesis in a dish: Modeling development and disease using organoid technologies
- (2014) M. A. Lancaster et al. SCIENCE
- Targeting ATM ameliorates mutant Huntingtin toxicity in cell and animal models of Huntington's disease
- (2014) X.-H. Lu et al. Science Translational Medicine
- Parkin Mutations Reduce the Complexity of Neuronal Processes in iPSC-Derived Human Neurons
- (2014) Yong Ren et al. STEM CELLS
- Pathological roles of the VEGF/SphK pathway in Niemann–Pick type C neurons
- (2014) Hyun Lee et al. Nature Communications
- Intrinsic Membrane Hyperexcitability of Amyotrophic Lateral Sclerosis Patient-Derived Motor Neurons
- (2014) Brian J. Wainger et al. Cell Reports
- Effect of Potent γ-Secretase Modulator in Human Neurons Derived From Multiple Presenilin 1–Induced Pluripotent Stem Cell Mutant Carriers
- (2014) Qing Liu et al. JAMA Neurology
- Gene expression analysis of human induced pluripotent stem cell-derived neurons carrying copy number variants of chromosome 15q11-q13.1
- (2014) Noelle D Germain et al. Molecular Autism
- Genetic and Chemical Correction of Cholesterol Accumulation and Impaired Autophagy in Hepatic and Neural Cells Derived from Niemann-Pick Type C Patient-Specific iPS Cells
- (2014) Dorothea Maetzel et al. Stem Cell Reports
- Detailed Analysis of the Genetic and Epigenetic Signatures of iPSC-Derived Mesodiencephalic Dopaminergic Neurons
- (2014) Reinhard Roessler et al. Stem Cell Reports
- International Coordination of Large-Scale Human Induced Pluripotent Stem Cell Initiatives: Wellcome Trust and ISSCR Workshops White Paper
- (2014) Filipa A.C. Soares et al. Stem Cell Reports
- Comparing ESC and iPSC—Based Models for Human Genetic Disorders
- (2014) Tomer Halevy et al. Journal of Clinical Medicine
- Isogenic Human iPSC Parkinson’s Model Shows Nitrosative Stress-Induced Dysfunction in MEF2-PGC1α Transcription
- (2013) Scott D. Ryan et al. CELL
- Human Embryonic Stem Cells Derived by Somatic Cell Nuclear Transfer
- (2013) Masahito Tachibana et al. CELL
- Functional Repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients
- (2013) Gerald Schwank et al. Cell Stem Cell
- Human iPSC-Based Modeling of Late-Onset Disease via Progerin-Induced Aging
- (2013) Justine D. Miller et al. Cell Stem Cell
- Human iPSC-Derived Oligodendrocyte Progenitor Cells Can Myelinate and Rescue a Mouse Model of Congenital Hypomyelination
- (2013) Su Wang et al. Cell Stem Cell
- A Small Molecule Screen in Stem-Cell-Derived Motor Neurons Identifies a Kinase Inhibitor as a Candidate Therapeutic for ALS
- (2013) Yin M. Yang et al. Cell Stem Cell
- Global Transcriptional and Translational Repression in Human-Embryonic-Stem-Cell-Derived Rett Syndrome Neurons
- (2013) Yun Li et al. Cell Stem Cell
- Modeling Alzheimer’s Disease with iPSCs Reveals Stress Phenotypes Associated with Intracellular Aβ and Differential Drug Responsiveness
- (2013) Takayuki Kondo et al. Cell Stem Cell
- Imprinted expression of UBE3A in non-neuronal cells from a Prader–Willi syndrome patient with an atypical deletion
- (2013) Kristen Martins-Taylor et al. HUMAN MOLECULAR GENETICS
- Human iPSC models of neuronal ceroid lipofuscinosis capture distinct effects of TPP1 and CLN3 mutations on the endocytic pathway
- (2013) Xenia Lojewski et al. HUMAN MOLECULAR GENETICS
- Enhanced Aggregation of Androgen Receptor in Induced Pluripotent Stem Cell-derived Neurons from Spinal and Bulbar Muscular Atrophy
- (2013) Yoshihiro Nihei et al. JOURNAL OF BIOLOGICAL CHEMISTRY
- Inhibition of mitochondrial fragmentation diminishes Huntington’s disease–associated neurodegeneration
- (2013) Xing Guo et al. JOURNAL OF CLINICAL INVESTIGATION
- A cellular model for sporadic ALS using patient-derived induced pluripotent stem cells
- (2013) Matthew F. Burkhardt et al. MOLECULAR AND CELLULAR NEUROSCIENCE
- Cerebral organoids model human brain development and microcephaly
- (2013) Madeline A. Lancaster et al. NATURE
- Targeted DNA demethylation and activation of endogenous genes using programmable TALE-TET1 fusion proteins
- (2013) Morgan L Maeder et al. NATURE BIOTECHNOLOGY
- Meta-analysis of 74,046 individuals identifies 11 new susceptibility loci for Alzheimer's disease
- (2013) Jean-Charles Lambert et al. NATURE GENETICS
- A functional CFTR assay using primary cystic fibrosis intestinal organoids
- (2013) Johanna F Dekkers et al. NATURE MEDICINE
- RNA Toxicity from the ALS/FTD C9ORF72 Expansion Is Mitigated by Antisense Intervention
- (2013) Christopher J. Donnelly et al. NEURON
- Identification and Rescue of -Synuclein Toxicity in Parkinson Patient-Derived Neurons
- (2013) C. Y. Chung et al. SCIENCE
- Targeting RNA Foci in iPSC-Derived Motor Neurons from ALS Patients with a C9ORF72 Repeat Expansion
- (2013) D. Sareen et al. Science Translational Medicine
- High throughput screening for inhibitors of REST in neural derivatives of human embryonic stem cells reveals a chemical compound that promotes expression of neuronal genes
- (2013) Jérémie Charbord et al. STEM CELLS
- Loss of Spastin Function Results in Disease-Specific Axonal Defects in Human Pluripotent Stem Cell-Based Models of Hereditary Spastic Paraplegia
- (2013) Kyle R. Denton et al. STEM CELLS
- SMRT compounds abrogate cellular phenotypes of ataxia telangiectasia in neural derivatives of patient-specific hiPSCs
- (2013) Peiyee Lee et al. Nature Communications
- Involvement of parental imprinting in the antisense regulation of onco-miR-372-373
- (2013) Yonatan Stelzer et al. Nature Communications
- Induced Pluripotent Stem Cell Modeling of Multisystemic, Hereditary Transthyretin Amyloidosis
- (2013) Amy Leung et al. Stem Cell Reports
- A TALEN Genome-Editing System for Generating Human Stem Cell-Based Disease Models
- (2012) Qiurong Ding et al. Cell Stem Cell
- Cardiomyocytes Derived From Pluripotent Stem Cells Recapitulate Electrophysiological Characteristics of an Overlap Syndrome of Cardiac Sodium Channel Disease
- (2012) Richard P. Davis et al. CIRCULATION
- Large-scale screening using familial dysautonomia induced pluripotent stem cells identifies compounds that rescue IKBKAP expression
- (2012) Gabsang Lee et al. NATURE BIOTECHNOLOGY
- Induced pluripotent stem cells: the new patient?
- (2012) Milena Bellin et al. NATURE REVIEWS MOLECULAR CELL BIOLOGY
- Derivation of autism spectrum disorder-specific induced pluripotent stem cells from peripheral blood mononuclear cells
- (2012) Brooke A. DeRosa et al. NEUROSCIENCE LETTERS
- Inhibition of Apoptosis Blocks Human Motor Neuron Cell Death in a Stem Cell Model of Spinal Muscular Atrophy
- (2012) Dhruv Sareen et al. PLoS One
- Modeling human cortical development in vitro using induced pluripotent stem cells
- (2012) J. Mariani et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Identification of a specific reprogramming-associated epigenetic signature in human induced pluripotent stem cells
- (2012) S. Ruiz et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Drug Screening for ALS Using Patient-Specific Induced Pluripotent Stem Cells
- (2012) N. Egawa et al. Science Translational Medicine
- Pharmacological Rescue of Mitochondrial Deficits in iPSC-Derived Neural Cells from Patients with Familial Parkinson's Disease
- (2012) O. Cooper et al. Science Translational Medicine
- Integration-Free Induced Pluripotent Stem Cells Model Genetic and Neural Developmental Features of Down Syndrome Etiology
- (2012) James A. Briggs et al. STEM CELLS
- Molecular analysis of FMR1 reactivation in fragile-X induced pluripotent stem cells and their neuronal derivatives
- (2012) Ori Bar-Nur et al. Journal of Molecular Cell Biology
- Induced pluripotent stem cell models from X-linked adrenoleukodystrophy patients
- (2011) Jiho Jang et al. ANNALS OF NEUROLOGY
- LRRK2 Mutant iPSC-Derived DA Neurons Demonstrate Increased Susceptibility to Oxidative Stress
- (2011) Ha Nam Nguyen et al. Cell Stem Cell
- Epigenetic Memory and Preferential Lineage-Specific Differentiation in Induced Pluripotent Stem Cells Derived from Human Pancreatic Islet Beta Cells
- (2011) Ori Bar-Nur et al. Cell Stem Cell
- Altered Oxygen Metabolism Associated to Neurogenesis of Induced Pluripotent Stem Cells Derived from a Schizophrenic Patient
- (2011) Bruna Da Silveira Paulsen et al. CELL TRANSPLANTATION
- Extrapolation From Mouse to Man
- (2011) Robert L. Hamlin et al. Circulation-Cardiovascular Imaging
- Drug evaluation in cardiomyocytes derived from human induced pluripotent stem cells carrying a long QT syndrome type 2 mutation
- (2011) Elena Matsa et al. EUROPEAN HEART JOURNAL
- Epidemiology and etiology of Parkinson’s disease: a review of the evidence
- (2011) Karin Wirdefeldt et al. EUROPEAN JOURNAL OF EPIDEMIOLOGY
- Modeling abnormal early development with induced pluripotent stem cells from aneuploid syndromes
- (2011) Wen Li et al. HUMAN MOLECULAR GENETICS
- Modeling familial Alzheimer's disease with induced pluripotent stem cells
- (2011) Takuya Yagi et al. HUMAN MOLECULAR GENETICS
- Modelling schizophrenia using human induced pluripotent stem cells
- (2011) Kristen J. Brennand et al. NATURE
- Self-formation of functional adenohypophysis in three-dimensional culture
- (2011) Hidetaka Suga et al. NATURE
- Hotspots of aberrant epigenomic reprogramming in human induced pluripotent stem cells
- (2011) Ryan Lister et al. NATURE
- Self-organizing optic-cup morphogenesis in three-dimensional culture
- (2011) Mototsugu Eiraku et al. NATURE
- Donor cell type can influence the epigenome and differentiation potential of human induced pluripotent stem cells
- (2011) Kitai Kim et al. NATURE BIOTECHNOLOGY
- Incomplete DNA methylation underlies a transcriptional memory of somatic cells in human iPS cells
- (2011) Yuki Ohi et al. NATURE CELL BIOLOGY
- Using iPSC-derived neurons to uncover cellular phenotypes associated with Timothy syndrome
- (2011) Sergiu P Paşca et al. NATURE MEDICINE
- Genomic imprinting: the emergence of an epigenetic paradigm
- (2011) Anne C. Ferguson-Smith NATURE REVIEWS GENETICS
- Global analysis of parental imprinting in human parthenogenetic induced pluripotent stem cells
- (2011) Yonatan Stelzer et al. NATURE STRUCTURAL & MOLECULAR BIOLOGY
- Parkinson's disease induced pluripotent stem cells with triplication of the α-synuclein locus
- (2011) Michael J. Devine et al. Nature Communications
- Knockdown of Fanconi anemia genes in human embryonic stem cells reveals early developmental defects in the hematopoietic lineage
- (2010) A. Tulpule et al. BLOOD
- A Model for Neural Development and Treatment of Rett Syndrome Using Human Induced Pluripotent Stem Cells
- (2010) Maria C.N. Marchetto et al. CELL
- Differential Modeling of Fragile X Syndrome by Human Embryonic Stem Cells and Induced Pluripotent Stem Cells
- (2010) Achia Urbach et al. Cell Stem Cell
- Induced Pluripotent Stem Cells Can Be Used to Model the Genomic Imprinting Disorder Prader-Willi Syndrome
- (2010) Jiayin Yang et al. JOURNAL OF BIOLOGICAL CHEMISTRY
- Modeling inherited metabolic disorders of the liver using human induced pluripotent stem cells
- (2010) S. Tamir Rashid et al. JOURNAL OF CLINICAL INVESTIGATION
- Induced pluripotent stem cell models of the genomic imprinting disorders Angelman and Prader–Willi syndromes
- (2010) Stormy J. Chamberlain et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Human Embryonic Stem Cells as Models for Aneuploid Chromosomal Syndromes
- (2010) Juan-Carlos Biancotti et al. STEM CELLS
- Generation of induced pluripotent stem cells from human blood
- (2009) Y.-H. Loh et al. BLOOD
- Modelling pathogenesis and treatment of familial dysautonomia using patient-specific iPSCs
- (2009) Gabsang Lee et al. NATURE
- Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells
- (2009) Ángel Raya et al. NATURE
- Studying Early Lethality of 45,XO (Turner's Syndrome) Embryos Using Human Embryonic Stem Cells
- (2009) Achia Urbach et al. PLoS One
- Cell Lines Derived from Human Parthenogenetic Embryos Can Display Aberrant Centriole Distribution and Altered Expression Levels of Mitotic Spindle Check-point Transcripts
- (2009) Tiziana A. L. Brevini et al. Stem Cell Reviews and Reports
- Clone- and Gene-Specific Aberrations of Parental Imprinting in Human Induced Pluripotent Stem Cells
- (2009) Marjorie Pick et al. STEM CELLS
- Disease-Specific Induced Pluripotent Stem Cells
- (2008) In-Hyun Park et al. CELL
- Human Embryonic Stem Cell-Derived Motor Neurons Are Sensitive to the Toxic Effect of Glial Cells Carrying an ALS-Causing Mutation
- (2008) Francesco Paolo Di Giorgio et al. Cell Stem Cell
- Induced Pluripotent Stem Cells Generated from Patients with ALS Can Be Differentiated into Motor Neurons
- (2008) J. T. Dimos et al. SCIENCE
Discover Peeref hubs
Discuss science. Find collaborators. Network.
Join a conversationAdd your recorded webinar
Do you already have a recorded webinar? Grow your audience and get more views by easily listing your recording on Peeref.
Upload Now