Use of human pluripotent stem cell-derived cells for neurodegenerative disease modeling and drug screening platform

Generation of a human induced pluripotent stem cell–based model for tauopathies combining three microtubule-associated protein tau mutations which displays several phenotypes linked to neurodegeneration

(2018) Juan Antonio García-León et al.   Alzheimers & Dementia

Toward a New Concept of Alzheimer’s Disease Models: A Perspective from Neuroinflammation

(2018) Antonia Gutierrez et al.   JOURNAL OF ALZHEIMERS DISEASE

Multiple sclerosis

(2018) Alan J Thompson et al.   LANCET

Gain of toxic apolipoprotein E4 effects in human iPSC-derived neurons is ameliorated by a small-molecule structure corrector

(2018) Chengzhong Wang et al.   NATURE MEDICINE

CRISPR off-targets: a reassessment

(2018)   NATURE METHODS

Short-term retinoic acid treatment sustains pluripotency and suppresses differentiation of human induced pluripotent stem cells

(2018) Maria Teresa De Angelis et al.   Cell Death & Disease

Microglia in Alzheimer’s Disease: Activated, Dysfunctional or Degenerative

(2018) Victoria Navarro et al.   Frontiers in Aging Neuroscience

The Parkinson Pandemic—A Call to Action

(2018) E. Ray Dorsey et al.   JAMA Neurology

Functional Studies of Missense TREM2 Mutations in Human Stem Cell-Derived Microglia

(2018) Philip W. Brownjohn et al.   Stem Cell Reports

All-Optical Electrophysiology for High-Throughput Functional Characterization of a Human iPSC-Derived Motor Neuron Model of ALS

(2018) Evangelos Kiskinis et al.   Stem Cell Reports

SOX10 Single Transcription Factor-Based Fast and Efficient Generation of Oligodendrocytes from Human Pluripotent Stem Cells

(2018) Juan Antonio García-León et al.   Stem Cell Reports

In vivo CRISPR editing with no detectable genome-wide off-target mutations

(2018) Pinar Akcakaya et al.   NATURE

Accumulation of 8,9-unsaturated sterols drives oligodendrocyte formation and remyelination

(2018) Zita Hubler et al.   NATURE

Induction of myelinating oligodendrocytes in human cortical spheroids

(2018) Mayur Madhavan et al.   NATURE METHODS

Animal models of neurodegenerative diseases

(2018) Ted M. Dawson et al.   NATURE NEUROSCIENCE

Human Induced Pluripotent Stem Cell-Derived Microglia-Like Cells Harboring TREM2 Missense Mutations Show Specific Deficits in Phagocytosis

(2018) Pablo Garcia-Reitboeck et al.   Cell Reports

Modeling Parkinson’s Disease Using Patient-specific Induced Pluripotent Stem Cells

(2018) Hong Li et al.   Journal of Parkinsons Disease

CRISPR/Cas9 editing of APP C-terminus attenuates β-cleavage and promotes α-cleavage

(2018) Jichao Sun et al.   Nature Communications

APOE ε4/ε4 diminishes neurotrophic function of human iPSC-derived astrocytes

(2017) Jing Zhao et al.   HUMAN MOLECULAR GENETICS

Clemastine fumarate as a remyelinating therapy for multiple sclerosis (ReBUILD): a randomised, controlled, double-blind, crossover trial

(2017) Ari J Green et al.   LANCET

A simplified protocol for differentiation of electrophysiologically mature neuronal networks from human induced pluripotent stem cells

(2017) N Gunhanlar et al.   MOLECULAR PSYCHIATRY

CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo

(2017) Alex Mas Monteys et al.   MOLECULAR THERAPY

Human iPS cell-derived dopaminergic neurons function in a primate Parkinson’s disease model

(2017) Tetsuhiro Kikuchi et al.   NATURE

Differentiation of human and murine induced pluripotent stem cells to microglia-like cells

(2017) Hetal Pandya et al.   NATURE NEUROSCIENCE

Amyloid-β and tau complexity — towards improved biomarkers and targeted therapies

(2017) Juan Carlos Polanco et al.   Nature Reviews Neurology

iPSC-Derived Human Microglia-like Cells to Study Neurological Diseases

(2017) Edsel M. Abud et al.   NEURON

Rapid and efficient generation of oligodendrocytes from human induced pluripotent stem cells using transcription factors

(2017) Marc Ehrlich et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Aberrant iPSC-derived human astrocytes in Alzheimer's disease

(2017) V C Jones et al.   Cell Death & Disease

HDAC6 inhibition reverses axonal transport defects in motor neurons derived from FUS-ALS patients

(2017) Wenting Guo et al.   Nature Communications

iPSC-Based Compound Screening and In Vitro Trials Identify a Synergistic Anti-amyloid β Combination for Alzheimer’s Disease

(2017) Takayuki Kondo et al.   Cell Reports

Amyotrophic lateral sclerosis patient iPSC-derived astrocytes impair autophagy via non-cell autonomous mechanisms

(2017) Martin Madill et al.   Molecular Brain

Cerebral organoids reveal early cortical maldevelopment in schizophrenia—computational anatomy and genomics, role of FGFR1

(2017) E. K. Stachowiak et al.   Translational Psychiatry

An Efficient Platform for Astrocyte Differentiation from Human Induced Pluripotent Stem Cells

(2017) Julia TCW et al.   Stem Cell Reports

Directed Differentiation of Human Pluripotent Stem Cells to Microglia

(2017) Panagiotis Douvaras et al.   Stem Cell Reports

Reversal of Phenotypic Abnormalities by CRISPR/Cas9-Mediated Gene Correction in Huntington Disease Patient-Derived Induced Pluripotent Stem Cells

(2017) Xiaohong Xu et al.   Stem Cell Reports

Parkinson disease

(2017) Werner Poewe et al.   Nature Reviews Disease Primers

CRISPR/Cas9-Correctable mutation-related molecular and physiological phenotypes in iPSC-derived Alzheimer’s PSEN2 N141I neurons

(2017) Maitane Ortiz-Virumbrales et al.   Acta Neuropathologica Communications

Soluble phospho-tau from Alzheimer’s disease hippocampus drives microglial degeneration

(2016) Elisabeth Sanchez-Mejias et al.   ACTA NEUROPATHOLOGICA

High throughput physiological screening of iPSC-derived cardiomyocytes for drug development

(2016) Juan C. del Álamo et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR CELL RESEARCH

Development of a central nervous system axonal myelination assay for high throughput screening

(2016) Karen D. Lariosa-Willingham et al.   BMC NEUROSCIENCE

iPSC-based drug screening for Huntington׳s disease

(2016) Ningzhe Zhang et al.   BRAIN RESEARCH

Neural Subtype Specification from Human Pluripotent Stem Cells

(2016) Yunlong Tao et al.   Cell Stem Cell

Zika Virus Depletes Neural Progenitors in Human Cerebral Organoids through Activation of the Innate Immune Receptor TLR3

(2016) Jason Dang et al.   Cell Stem Cell

Induced Pluripotent Stem Cells Meet Genome Editing

(2016) Dirk Hockemeyer et al.   Cell Stem Cell

Defeating Alzheimer's disease and other dementias: a priority for European science and society

(2016) Bengt Winblad et al.   LANCET NEUROLOGY

Manifestation of Huntington’s disease pathology in human induced pluripotent stem cell-derived neurons

(2016) Evgeny D. Nekrasov et al.   Molecular Neurodegeneration

Decoding ALS: from genes to mechanism

(2016) J. Paul Taylor et al.   NATURE

Parkinson-associated risk variant in distal enhancer of α-synuclein modulates target gene expression

(2016) Frank Soldner et al.   NATURE

Efficient derivation of microglia-like cells from human pluripotent stem cells

(2016) Julien Muffat et al.   NATURE MEDICINE

Modeling ALS with motor neurons derived from human induced pluripotent stem cells

(2016) Samuel Sances et al.   NATURE NEUROSCIENCE

Prevalence, characteristics, and survival of frontotemporal lobar degeneration syndromes

(2016) Ian T.S. Coyle-Gilchrist et al.   NEUROLOGY

Purification and Characterization of Progenitor and Mature Human Astrocytes Reveals Transcriptional and Functional Differences with Mouse

(2016) Ye Zhang et al.   NEURON

Oligodendrocytes contribute to motor neuron death in ALS via SOD1-dependent mechanism

(2016) Laura Ferraiuolo et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Projected increase in amyotrophic lateral sclerosis from 2015 to 2040

(2016) Karissa C. Arthur et al.   Nature Communications

Stem Cell-Derived Oligodendroglial Cells for Therapy in Neurological Diseases

(2016) Juan A. García-León et al.   Current Stem Cell Research & Therapy

Self-Organizing 3D Human Neural Tissue Derived from Induced Pluripotent Stem Cells Recapitulate Alzheimer’s Disease Phenotypes

(2016) Waseem K. Raja et al.   PLoS One

Dopaminergic neurons differentiating from LRRK2 G2019S induced pluripotent stem cells show early neuritic branching defects

(2016) Laurence Borgs et al.   Scientific Reports

Early maturation and distinct tau pathology in induced pluripotent stem cell-derived neurons from patients withMAPTmutations

(2015) Mariangela Iovino et al.   BRAIN

FOXG1-Dependent Dysregulation of GABA/Glutamate Neuron Differentiation in Autism Spectrum Disorders

(2015) Jessica Mariani et al.   CELL

Human Embryonic Stem Cell-Derived Oligodendrocyte Progenitors Remyelinate the Brain and Rescue Behavioral Deficits following Radiation

(2015) Jinghua Piao et al.   Cell Stem Cell

In vitro myelin formation using embryonic stem cells

(2015) B. E. Kerman et al.   DEVELOPMENT

Drug-based modulation of endogenous stem cells promotes functional remyelination in vivo

(2015) Fadi J. Najm et al.   NATURE

Neuronal medium that supports basic synaptic functions and activity of human neurons in vitro

(2015) Cedric Bardy et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Clemastine rescues behavioral changes and enhances remyelination in the cuprizone mouse model of demyelination

(2015) Zhifang Li et al.   Neuroscience Bulletin

Off-target Effects in CRISPR/Cas9-mediated Genome Engineering

(2015) Xiao-Hui Zhang et al.   Molecular Therapy-Nucleic Acids

Efficient Recombinase-Mediated Cassette Exchange in hPSCs to Study the Hepatocyte Lineage Reveals AAVS1 Locus-Mediated Transgene Inhibition

(2015) Laura Ordovás et al.   Stem Cell Reports

Huntington disease

(2015) Gillian P. Bates et al.   Nature Reviews Disease Primers

Pathways Disrupted in Human ALS Motor Neurons Identified through Genetic Correction of Mutant SOD1

(2014) Evangelos Kiskinis et al.   Cell Stem Cell

Human oligodendrocytes in remyelination research

(2014) Marcin Czepiel et al.   GLIA

Induced Pluripotent Stem Cells for Disease Modeling and Drug Discovery in Neurodegenerative Diseases

(2014) Lei Cao et al.   MOLECULAR NEUROBIOLOGY

A three-dimensional human neural cell culture model of Alzheimer’s disease

(2014) Se Hoon Choi et al.   NATURE

Combinatorial analysis of developmental cues efficiently converts human pluripotent stem cells into multiple neuronal subtypes

(2014) Yves Maury et al.   NATURE BIOTECHNOLOGY

A comparison of non-integrating reprogramming methods

(2014) Thorsten M Schlaeger et al.   NATURE BIOTECHNOLOGY

Micropillar arrays as a high-throughput screening platform for therapeutics in multiple sclerosis

(2014) Feng Mei et al.   NATURE MEDICINE

The Productive Entry Pathway of HIV-1 in Macrophages Is Dependent on Endocytosis through Lipid Rafts Containing CD4

(2014) Bonnie van Wilgenburg et al.   PLoS One

Physiological Characterisation of Human iPS-Derived Dopaminergic Neurons

(2014) Elizabeth M. Hartfield et al.   PLoS One

Histone chaperone ASF1A is required for maintenance of pluripotency and cellular reprogramming

(2014) E. Gonzalez-Munoz et al.   SCIENCE

Direct induction of ramified microglia-like cells from human monocytes: Dynamic microglial dysfunction in Nasu-Hakola disease

(2014) Masahiro Ohgidani et al.   Scientific Reports

Intrinsic Membrane Hyperexcitability of Amyotrophic Lateral Sclerosis Patient-Derived Motor Neurons

(2014) Brian J. Wainger et al.   Cell Reports

iPSC-Derived Dopamine Neurons Reveal Differences between Monozygotic Twins Discordant for Parkinson’s Disease

(2014) Chris M. Woodard et al.   Cell Reports

Efficient Generation of Myelinating Oligodendrocytes from Primary Progressive Multiple Sclerosis Patients by Induced Pluripotent Stem Cells

(2014) Panagiotis Douvaras et al.   Stem Cell Reports

Human iPSC-Derived Oligodendrocyte Progenitor Cells Can Myelinate and Rescue a Mouse Model of Congenital Hypomyelination

(2013) Su Wang et al.   Cell Stem Cell

Accelerated High-Yield Generation of Limb-Innervating Motor Neurons from Human Stem Cells

(2013) M. W. Amoroso et al.   JOURNAL OF NEUROSCIENCE

A cellular model for sporadic ALS using patient-derived induced pluripotent stem cells

(2013) Matthew F. Burkhardt et al.   MOLECULAR AND CELLULAR NEUROSCIENCE

A regenerative approach to the treatment of multiple sclerosis

(2013) Vishal A. Deshmukh et al.   NATURE

Cerebral organoids model human brain development and microcephaly

(2013) Madeline A. Lancaster et al.   NATURE

State of play in amyotrophic lateral sclerosis genetics

(2013) Alan E Renton et al.   NATURE NEUROSCIENCE

Seamless genome editing in human pluripotent stem cells using custom endonuclease–based gene targeting and the piggyBac transposon

(2013) Kosuke Yusa   Nature Protocols

Efficient, Long Term Production of Monocyte-Derived Macrophages from Human Pluripotent Stem Cells under Partly-Defined and Fully-Defined Conditions

(2013) Bonnie van Wilgenburg et al.   PLoS One

Unique drug screening approach for prion diseases identifies tacrolimus and astemizole as antiprion agents

(2013) Y. E. Karapetyan et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Pluripotent Stem Cells Induced from Mouse Somatic Cells by Small-Molecule Compounds

(2013) P. Hou et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong 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

Improved Cell Therapy Protocols for Parkinson's Disease Based on Differentiation Efficiency and Safety of hESC-, hiPSC-, and Non-Human Primate iPSC-Derived Dopaminergic Neurons

(2013) Maria Sundberg et al.   STEM CELLS

Origin and differentiation of microglia

(2013) Florent Ginhoux et al.   Frontiers in Cellular Neuroscience

APP Processing in Human Pluripotent Stem Cell-Derived Neurons Is Resistant to NSAID-Based γ-Secretase Modulation

(2013) Jerome Mertens et al.   Stem Cell Reports

Induced Pluripotent Stem Cells from Patients with Huntington's Disease Show CAG-Repeat-Expansion-Associated Phenotypes

(2012) The HD iPSC Consortium   Cell Stem Cell

A novel in vitro human microglia model: Characterization of human monocyte-derived microglia

(2012) Samar Etemad et al.   JOURNAL OF NEUROSCIENCE METHODS

Probing sporadic and familial Alzheimer’s disease using induced pluripotent stem cells

(2012) Mason A. Israel et al.   NATURE

A culture system to study oligodendrocyte myelination processes using engineered nanofibers

(2012) Seonok Lee et al.   NATURE METHODS

Human cerebral cortex development from pluripotent stem cells to functional excitatory synapses

(2012) Yichen Shi et al.   NATURE NEUROSCIENCE

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

(2012) M. Jinek et al.   SCIENCE

Drug Screening for ALS Using Patient-Specific Induced Pluripotent Stem Cells

(2012) N. Egawa et al.   Science Translational Medicine

Prevention of β-amyloid induced toxicity in human iPS cell-derived neurons by inhibition of Cyclin-dependent kinases and associated cell cycle events

(2012) Xiaohong Xu et al.   Stem Cell Research

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

Modeling familial Alzheimer's disease with induced pluripotent stem cells

(2011) Takuya Yagi et al.   HUMAN MOLECULAR GENETICS

Induction of oligodendrocyte differentiation from adult human fibroblast-derived induced pluripotent stem cells

(2011) Shin-ichiro Ogawa et al.   IN VITRO CELLULAR & DEVELOPMENTAL BIOLOGY-ANIMAL

Genetic engineering of human pluripotent cells using TALE nucleases

(2011) Dirk Hockemeyer et al.   NATURE BIOTECHNOLOGY

Specification of transplantable astroglial subtypes from human pluripotent stem cells

(2011) Robert Krencik et al.   NATURE BIOTECHNOLOGY

Are the Prevalence and Incidence of Multiple Sclerosis Changing?

(2011) Julián Benito-León   NEUROEPIDEMIOLOGY

Anti-Aβ Drug Screening Platform Using Human iPS Cell-Derived Neurons for the Treatment of Alzheimer's Disease

(2011) Naoki Yahata et al.   PLoS One

Targeting DNA Double-Strand Breaks with TAL Effector Nucleases

(2010) M. Christian et al.   GENETICS

Molecular Mechanisms and Potential Therapeutical Targets in Huntington's Disease

(2010) Chiara Zuccato et al.   PHYSIOLOGICAL REVIEWS

Human neural stem cells ameliorate autoimmune encephalomyelitis in non-human primates

(2009) Stefano Pluchino et al.   ANNALS OF NEUROLOGY

Highly efficient neural conversion of human ES and iPS cells by dual inhibition of SMAD signaling

(2009) Stuart M Chambers et al.   NATURE BIOTECHNOLOGY

Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases

(2009) Dirk Hockemeyer et al.   NATURE BIOTECHNOLOGY

Differentiation of human oligodendrocytes from pluripotent stem cells

(2009) Bao-Yang Hu et al.   Nature Protocols

Differentiation of spinal motor neurons from pluripotent human stem cells

(2009) Bao-Yang Hu et al.   Nature Protocols

Neonatal Chimerization with Human Glial Progenitor Cells Can Both Remyelinate and Rescue the Otherwise Lethally Hypomyelinated Shiverer Mouse

(2008) Martha S. Windrem et al.   Cell Stem Cell

High Throughput Screening for Neurodegeneration and Complex Disease Phenotypes

(2008) Hemant Varma et al.   COMBINATORIAL CHEMISTRY & HIGH THROUGHPUT SCREENING

Homogeneous monocytes and macrophages from human embryonic stem cells following coculture-free differentiation in M-CSF and IL-3

(2008) Karl R. Karlsson et al.   EXPERIMENTAL HEMATOLOGY

Induced pluripotent stem cells from a spinal muscular atrophy patient

(2008) Allison D. Ebert et al.   NATURE

Induced Pluripotent Stem Cells Generated from Patients with ALS Can Be Differentiated into Motor Neurons

(2008) J. T. Dimos et al.   SCIENCE

FTY720 modulates human oligodendrocyte progenitor process extension and survival

(2007) Veronique E. Miron et al.   ANNALS OF NEUROLOGY