iPSC-based disease modeling and drug discovery in cardinal neurodegenerative disorders

Human stem cell models of neurodegeneration: From basic science of amyotrophic lateral sclerosis to clinical translation

(2022) Elisa Giacomelli et al.   Cell Stem Cell

Prediction Model of Amyotrophic Lateral Sclerosis by Deep Learning with Patient Induced Pluripotent Stem Cells

(2021) Keiko Imamura et al.   ANNALS OF NEUROLOGY

Clinical Trial for Parkinson’s Disease Gets a Green Light in the US

(2021) Jun Takahashi   Cell Stem Cell

Preclinical Efficacy and Safety of a Human Embryonic Stem Cell-Derived Midbrain Dopamine Progenitor Product, MSK-DA01

(2021) Jinghua Piao et al.   Cell Stem Cell

A human model of Batten disease shows role of CLN3 in phagocytosis at the photoreceptor–RPE interface

(2021) Cynthia Tang et al.   Communications Biology

Flexible and Accurate Substrate Processing with Distinct Presenilin/γ-Secretases in Human Cortical Neurons

(2021) Hirotaka Watanabe et al.   eNeuro

Building consensus on definition and nomenclature of hepatic, pancreatic, and biliary organoids

(2021) Ary Marsee et al.   Cell Stem Cell

Age-dependent instability of mature neuronal fate in induced neurons from Alzheimer’s patients

(2021) Jerome Mertens et al.   Cell Stem Cell

Parkinson's disease

(2021) Bastiaan R Bloem et al.   LANCET

Failure of genetic therapies for Huntington’s devastates community

(2021) Diana Kwon   NATURE

Double setback for ASO trials in Huntington disease

(2021) Katie Kingwell   NATURE REVIEWS DRUG DISCOVERY

MeCP2 controls neural stem cell fate specification through miR-199a-mediated inhibition of BMP-Smad signaling

(2021) Hideyuki Nakashima et al.   Cell Reports

Alzheimer disease

(2021) David S. Knopman et al.   Nature Reviews Disease Primers

Molecular Components of Store-Operated Calcium Channels in the Regulation of Neural Stem Cell Physiology, Neurogenesis, and the Pathology of Huntington’s Disease

(2021) Ewelina Latoszek et al.   Frontiers in Cell and Developmental Biology

Effects of monoclonal antibodies against amyloid-β on clinical and biomarker outcomes and adverse event risks: A systematic review and meta-analysis of phase III RCTs in Alzheimer’s disease

(2021) Konstantinos I. Avgerinos et al.   AGEING RESEARCH REVIEWS

Parkinson's Disease Genetics and Pathophysiology

(2021) Gabriel E. Vázquez-Vélez et al.   Annual Review of Neuroscience

Highly accurate protein structure prediction with AlphaFold

(2021) John Jumper et al.   NATURE

Highly accurate protein structure prediction for the human proteome

(2021) Kathryn Tunyasuvunakool et al.   NATURE

Cholesterol-functionalized DNA/RNA heteroduplexes cross the blood–brain barrier and knock down genes in the rodent CNS

(2021) Tetsuya Nagata et al.   NATURE BIOTECHNOLOGY

Advancing models of neural development with biomaterials

(2021) Julien G. Roth et al.   NATURE REVIEWS NEUROSCIENCE

Microglial signalling pathway deficits associated with the patient derived R47H TREM2 variants linked to AD indicate inability to activate inflammasome

(2021) Katharina Cosker et al.   Scientific Reports

Repurposing bromocriptine for Aβ metabolism in Alzheimer’s disease (REBRAnD) study: randomised placebo-controlled double-blind comparative trial and open-label extension trial to investigate the safety and efficacy of bromocriptine in Alzheimer’s disease with presenilin 1 (PSEN1) mutations

(2021) Takayuki Kondo et al.   BMJ Open

Human amyotrophic lateral sclerosis excitability phenotype screen: Target discovery and validation

(2021) Xuan Huang et al.   Cell Reports

Bringing Advanced Therapies for Parkinson’s Disease to the Clinic: The Scientist’s Perspective

(2021) Mark Tomishima et al.   Journal of Parkinsons Disease

SARS‐CoV ‐2 research using human pluripotent stem cells and organoids

(2021) Sayaka Deguchi et al.   Stem Cells Translational Medicine

Reduced PHOX2B stability causes axonal growth impairment in motor neurons with TARDBP mutations

(2021) Shio Mitsuzawa et al.   Stem Cell Reports

Identification of bioactive metabolites in human iPSC-derived dopaminergic neurons with PARK2 mutation: Altered mitochondrial and energy metabolism

(2021) Justyna Okarmus et al.   Stem Cell Reports

Induced Pluripotent Stem Cell-Derived Neural Precursors Improve Memory, Synaptic and Pathological Abnormalities in a Mouse Model of Alzheimer’s Disease

(2021) Enrique Armijo et al.   Cells

The therapeutic potential of multiclonal tumoricidal T cells derived from tumor infiltrating lymphocyte-derived iPS cells

(2021) Takeshi Ito et al.   Communications Biology

Gut microbiota–driven brain Aβ amyloidosis in mice requires microglia

(2021) Hemraj B. Dodiya et al.   JOURNAL OF EXPERIMENTAL MEDICINE

Human iPSC-derived astrocytes transplanted into the mouse brain undergo morphological changes in response to amyloid-β plaques

(2021) Pranav Preman et al.   Molecular Neurodegeneration

Common and rare variant association analyses in amyotrophic lateral sclerosis identify 15 risk loci with distinct genetic architectures and neuron-specific biology

(2021) Wouter van Rheenen et al.   NATURE GENETICS

Effective gene expression prediction from sequence by integrating long-range interactions

(2021) Žiga Avsec et al.   NATURE METHODS

ALS antisense drug falters in phase III

(2021) Asher Mullard   NATURE REVIEWS DRUG DISCOVERY

An integrated multi-omic analysis of iPSC-derived motor neurons from C9ORF72 ALS patients

(2021) Jonathan Li et al.   iScience

Non‐clinical efficacy, safety, and stable clinical cell processing of iPSC‐derived anti‐GPC3 CAR‐expressing NK/ILC cells

(2020) Tatsuki Ueda et al.   CANCER SCIENCE

iPSC modeling of young-onset Parkinson’s disease reveals a molecular signature of disease and novel therapeutic candidates

(2020) A. H. Laperle et al.   NATURE MEDICINE

Ropinirole, a New ALS Drug Candidate Developed Using iPSCs

(2020) Hideyuki Okano et al.   TRENDS IN PHARMACOLOGICAL SCIENCES

Modeling Cell-Cell Interactions in Parkinson’s Disease Using Human Stem Cell-Based Models

(2020) Katrin Simmnacher et al.   Frontiers in Cellular Neuroscience

miRNA-Based Rapid Differentiation of Purified Neurons from hPSCs Advancestowards Quick Screening for Neuronal Disease Phenotypes In Vitro

(2020) Mitsuru Ishikawa et al.   Cells

Variants in saposin D domain of prosaposin gene linked to Parkinson’s disease

(2020) Yutaka Oji et al.   BRAIN

Visualizing and Modulating Mitophagy for Therapeutic Studies of Neurodegeneration

(2020) Hiroyuki Katayama et al.   CELL

Multi-lineage Human iPSC-Derived Platforms for Disease Modeling and Drug Discovery

(2020) Arun Sharma et al.   Cell Stem Cell

Demonstration of brain region-specific neuronal vulnerability in human iPSC-based model of familial Parkinson’s disease

(2020) Razvan-Marius Brazdis et al.   HUMAN MOLECULAR GENETICS

A phase I/IIa double blind single institute trial of low dose sirolimus for Pendred syndrome/DFNB4

(2020) Masato Fujioka et al.   MEDICINE

Personalized iPSC-Derived Dopamine Progenitor Cells for Parkinson’s Disease

(2020) Jeffrey S. Schweitzer et al.   NEW ENGLAND JOURNAL OF MEDICINE

Human Glial Progenitor Cells Effectively Remyelinate the Demyelinated Adult Brain

(2020) Martha S. Windrem et al.   Cell Reports

Identifying Therapeutic Agents for Amelioration of Mitochondrial Clearance Disorder in Neurons of Familial Parkinson Disease

(2020) Akihiro Yamaguchi et al.   Stem Cell Reports

Anti-human TREM2 induces microglia proliferation and reduces pathology in an Alzheimer’s disease model

(2020) Shoutang Wang et al.   JOURNAL OF EXPERIMENTAL MEDICINE

Genetic and functional analysis of KIF5A variants in Japanese patients with sporadic amyotrophic lateral sclerosis.

(2020) Ryoichi Nakamura et al.   NEUROBIOLOGY OF AGING

Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS

(2020) Timothy Miller et al.   NEW ENGLAND JOURNAL OF MEDICINE

Pre-clinical study of induced pluripotent stem cell-derived dopaminergic progenitor cells for Parkinson’s disease

(2020) Daisuke Doi et al.   Nature Communications

Knockout of reactive astrocyte activating factors slows disease progression in an ALS mouse model

(2020) Kevin A. Guttenplan et al.   Nature Communications

Rising Stars: Astrocytes as a Therapeutic Target for ALS Disease

(2020) Michal Izrael et al.   Frontiers in Neuroscience

Pluripotent Stem Cell-Based Cell Therapy—Promise and Challenges

(2020) Shinya Yamanaka   Cell Stem Cell

New trends in cellular therapy

(2020) Hideyuki Okano et al.   DEVELOPMENT

Modifying macrophages at the periphery has the capacity to change microglial reactivity and to extend ALS survival

(2020) Aude Chiot et al.   NATURE NEUROSCIENCE

Trial of Sodium Phenylbutyrate–Taurursodiol for Amyotrophic Lateral Sclerosis

(2020) Sabrina Paganoni et al.   NEW ENGLAND JOURNAL OF MEDICINE

Opportunities and limitations of genetically modified nonhuman primate models for neuroscience research

(2020) Guoping Feng et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

iPS cell-based therapy for Parkinson's disease: A Kyoto trial

(2020) Jun Takahashi   Regenerative Therapy

A multi-ethnic meta-analysis identifies novel genes, including ACSL5, associated with amyotrophic lateral sclerosis

(2020) Ryoichi Nakamura et al.   Communications Biology

Current Status of and Perspectives on the Application of Marmosets in Neurobiology

(2020) Hideyuki Okano   Annual Review of Neuroscience

The microglial component of amyotrophic lateral sclerosis

(2020) Benjamin E Clarke et al.   BRAIN

Identification and in vitro characterization of C05-01, a PBB3 derivative with improved affinity for alpha-synuclein

(2020) Patricia Miranda-Azpiazu et al.   BRAIN RESEARCH

Generation of Functional Human 3D Cortico-Motor Assembloids

(2020) Jimena Andersen et al.   CELL

Dissecting the non-neuronal cell contribution to Parkinson’s disease pathogenesis using induced pluripotent stem cells

(2020) Meritxell Pons-Espinal et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

Effect of Ezogabine on Cortical and Spinal Motor Neuron Excitability in Amyotrophic Lateral Sclerosis

(2020) Brian J. Wainger et al.   JAMA Neurology

Alpha‐synuclein dynamics in induced pluripotent stem cell‐derived dopaminergic neurons from a Parkinson’s disease patient ( PARK4 ) with SNCA triplication

(2020) Hayato Fukusumi et al.   FEBS Open Bio

Organ-on-a-Chip: A New Paradigm for Drug Development

(2020) Chao Ma et al.   TRENDS IN PHARMACOLOGICAL SCIENCES

Stress-induced precocious aging in PD-patient iPSC-derived NSCs may underlie the pathophysiology of Parkinson’s disease

(2019) Liang Zhu et al.   Cell Death & Disease

Randomized Delayed-Start Trial of Levodopa in Parkinson’s Disease

(2019) Constant V.M. Verschuur et al.   NEW ENGLAND JOURNAL OF MEDICINE

Flow-enhanced vascularization and maturation of kidney organoids in vitro

(2019) Kimberly A. Homan et al.   NATURE METHODS

Cholesterol Metabolism Is a Druggable Axis that Independently Regulates Tau and Amyloid-β in iPSC-Derived Alzheimer’s Disease Neurons

(2019) Rik van der Kant et al.   Cell Stem Cell

Cell-specific overexpression of COMT in dopaminergic neurons of Parkinson’s disease

(2019) Naoko Kuzumaki et al.   BRAIN

Biological characterization of expression quantitative trait loci (eQTLs) showing tissue-specific opposite directional effects

(2019) Akira Mizuno et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

Development of a Chimeric Model to Study and Manipulate Human Microglia In Vivo

(2019) Jonathan Hasselmann et al.   NEURON

Aberrant axon branching via Fos-B dysregulation in FUS-ALS motor neurons

(2019) Tetsuya Akiyama et al.   EBioMedicine

Getting to the Cores of Autism

(2019) Lilia M. Iakoucheva et al.   CELL

The genetic architecture of Parkinson's disease

(2019) Cornelis Blauwendraat et al.   LANCET NEUROLOGY

Modelling human hepato-biliary-pancreatic organogenesis from the foregut–midgut boundary

(2019) Hiroyuki Koike et al.   NATURE

NET-CAGE characterizes the dynamics and topology of human transcribed cis-regulatory elements

(2019) Shigeki Hirabayashi et al.   NATURE GENETICS

Fragmented mitochondria released from microglia trigger A1 astrocytic response and propagate inflammatory neurodegeneration

(2019) Amit U. Joshi et al.   NATURE NEUROSCIENCE

Alzheimer Disease: An Update on Pathobiology and Treatment Strategies

(2019) Justin M. Long et al.   CELL

Mutations in CHCHD2 cause α-synuclein aggregation

(2019) Aya Ikeda et al.   HUMAN MOLECULAR GENETICS

Stem Cells: The Game Changers of Human Cardiac Disease Modelling and Regenerative Medicine

(2019) Elvira Immacolata Parrotta et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment

(2019) Daniel M. Ramos et al.   JOURNAL OF CLINICAL INVESTIGATION

A combinational treatment of carotenoids decreases Aβ secretion in human neurons via β-secretase inhibition

(2019) Misato Sho et al.   NEUROSCIENCE RESEARCH

Site-specific phosphorylation and caspase cleavage of GFAP are new markers of Alexander disease severity

(2019) Rachel A Battaglia et al.   eLife

Stem Cell Modeling of Neuroferritinopathy Reveals Iron as a Determinant of Senescence and Ferroptosis during Neuronal Aging

(2019) Anna Cozzi et al.   Stem Cell Reports

Mutant C9orf72 human iPSC‐derived astrocytes cause non‐cell autonomous motor neuron pathophysiology

(2019) Chen Zhao et al.   GLIA

Induced pluripotent stem cell–based Drug Repurposing for Amyotrophic lateral sclerosis Medicine (iDReAM) study: protocol for a phase I dose escalation study of bosutinib for amyotrophic lateral sclerosis patients

(2019) Keiko Imamura et al.   BMJ Open

Human iPSC-derived astrocytes from ALS patients with mutated C9ORF72 show increased oxidative stress and neurotoxicity

(2019) Anastasya Birger et al.   EBioMedicine

Ropinirole hydrochloride remedy for amyotrophic lateral sclerosis – Protocol for a randomized, double-blind, placebo-controlled, single-center, and open-label continuation phase I/IIa clinical trial (ROPALS trial)

(2019) Satoru Morimoto et al.   Regenerative Therapy

Microglia-Mediated Synapse Loss in Alzheimer's Disease

(2018) Lawrence Rajendran et al.   JOURNAL OF NEUROSCIENCE

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

Striatal neurons directly converted from Huntington’s disease patient fibroblasts recapitulate age-associated disease phenotypes

(2018) Matheus B. Victor et al.   NATURE NEUROSCIENCE

APOE4 Causes Widespread Molecular and Cellular Alterations Associated with Alzheimer’s Disease Phenotypes in Human iPSC-Derived Brain Cell Types

(2018) Yuan-Ta Lin et al.   NEURON

The multi-dimensional roles of astrocytes in ALS

(2018) Koji Yamanaka et al.   NEUROSCIENCE RESEARCH

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

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

GFAP Mutations in Astrocytes Impair Oligodendrocyte Progenitor Proliferation and Myelination in an hiPSC Model of Alexander Disease

(2018) Li Li et al.   Cell Stem Cell

Modeling sporadic ALS in iPSC-derived motor neurons identifies a potential therapeutic agent

(2018) Koki Fujimori et al.   NATURE MEDICINE

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

Human ESC-Derived Chimeric Mouse Models of Huntington’s Disease Reveal Cell-Intrinsic Defects in Glial Progenitor Cell Differentiation

(2018) Mikhail Osipovitch et al.   Cell Stem Cell

Efficacy and safety of edaravone in treatment of amyotrophic lateral sclerosis—a systematic review and meta-analysis

(2018) Linting Luo et al.   NEUROLOGICAL SCIENCES

T-type Calcium Channels Determine the Vulnerability of Dopaminergic Neurons to Mitochondrial Stress in Familial Parkinson Disease

(2018) Yoshikuni Tabata et al.   Stem Cell Reports

Nusinersen for the treatment of spinal muscular atrophy

(2017) Claudia A. Chiriboga   Expert Review of Neurotherapeutics

The TREM2-APOE Pathway Drives the Transcriptional Phenotype of Dysfunctional Microglia in Neurodegenerative Diseases

(2017) Susanne Krasemann et al.   IMMUNITY

Microglia in Alzheimer’s disease

(2017) David V. Hansen et al.   JOURNAL OF CELL BIOLOGY

Activin-A enhances mTOR signaling to promote aberrant chondrogenesis in fibrodysplasia ossificans progressiva

(2017) Kyosuke Hino et al.   JOURNAL OF CLINICAL INVESTIGATION

Microglia and C9orf72 in neuroinflammation and ALS and frontotemporal dementia

(2017) Deepti Lall et al.   JOURNAL OF CLINICAL INVESTIGATION

Amyotrophic lateral sclerosis

(2017) Michael A van Es et al.   LANCET

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

(2017) Tetsuhiro Kikuchi et al.   NATURE

Testing the Amyloid Hypothesis with a Humanized AD Mouse Model

(2017) Michelle K. Cahill et al.   NEURON

Hallmarks of Alzheimer’s Disease in Stem-Cell-Derived Human Neurons Transplanted into Mouse Brain

(2017) Ira Espuny-Camacho et al.   NEURON

Amyotrophic Lateral Sclerosis

(2017) Robert H. Brown et al.   NEW ENGLAND JOURNAL OF MEDICINE

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

(2017) Richard S. Finkel et al.   NEW ENGLAND JOURNAL OF MEDICINE

SMN deficiency in severe models of spinal muscular atrophy causes widespread intron retention and DNA damage

(2017) Mohini Jangi et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

The Src/c-Abl pathway is a potential therapeutic target in amyotrophic lateral sclerosis

(2017) Keiko Imamura et al.   Science Translational Medicine

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

Nusinersen for the treatment of spinal muscular atrophy

(2017) Claudia A. Chiriboga   Expert Review of Neurotherapeutics

Sporadic ALS Astrocytes Induce Neuronal Degeneration In Vivo

(2017) Kun Qian et al.   Stem Cell Reports

Parkinson disease

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

Current understanding of the molecular mechanisms in Parkinson's disease: Targets for potential treatments

(2017) Panchanan Maiti et al.   Translational Neurodegeneration

A rapid functional decline type of amyotrophic lateral sclerosis is linked to low expression ofTTN

(2016) Hazuki Watanabe et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

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

Pluripotent stem cells in disease modelling and drug discovery

(2016) Yishai Avior et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Astrocytes in physiological aging and Alzheimer’s disease

(2016) J.J. Rodríguez-Arellano et al.   NEUROSCIENCE

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

Generating Late-Onset Human iPSC-Based Disease Models by Inducing Neuronal Age-Related Phenotypes through Telomerase Manipulation

(2016) Elsa Vera et al.   Cell Reports

Successful Function of Autologous iPSC-Derived Dopamine Neurons following Transplantation in a Non-Human Primate Model of Parkinson’s Disease

(2015) Penelope J. Hallett et al.   Cell Stem Cell

Directly Reprogrammed Human Neurons Retain Aging-Associated Transcriptomic Signatures and Reveal Age-Related Nucleocytoplasmic Defects

(2015) Jerome Mertens et al.   Cell Stem Cell

I2020T mutant LRRK2 iPSC-derived neurons in the Sagamihara family exhibit increased Tau phosphorylation through the AKT/GSK-3β signaling pathway

(2015) Etsuro Ohta et al.   HUMAN MOLECULAR GENETICS

MicroRNA-based conversion of human fibroblasts into striatal medium spiny neurons

(2015) Michelle Richner et al.   Nature Protocols

Developing stem cell therapies for juvenile and adult-onset Huntington's disease

(2015) Kyle D Fink et al.   Regenerative Medicine

Neuroinflammation and Depression: Microglia Activation, Extracellular Microvesicles and microRNA Dysregulation

(2015) Dora Brites et al.   Frontiers in Cellular Neuroscience

Controlling the Regional Identity of hPSC-Derived Neurons to Uncover Neuronal Subtype Specificity of Neurological Disease Phenotypes

(2015) Kent Imaizumi et al.   Stem Cell Reports

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

(2014) Evangelos Kiskinis et al.   Cell Stem Cell

Intermediate filament protein accumulation in motor neurons derived from giant axonal neuropathy iPSCs rescued by restoration of gigaxonin

(2014) Bethany L. Johnson-Kerner et al.   HUMAN MOLECULAR GENETICS

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

(2014) Se Hoon Choi et al.   NATURE

Targeted inversion and reversion of the blood coagulation factor 8 gene in human iPS cells using TALENs

(2014) C.-Y. Park et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

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

iPS cell technologies: significance and applications to CNS regeneration and disease

(2014) Hideyuki Okano et al.   Molecular Brain

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

(2014) Panagiotis Douvaras et al.   Stem Cell Reports

Focal Transplantation of Human iPSC-Derived Glial-Rich Neural Progenitors Improves Lifespan of ALS Mice

(2014) Takayuki Kondo et al.   Stem Cell Reports

Involvement of ER Stress in Dysmyelination of Pelizaeus-Merzbacher Disease with PLP1 Missense Mutations Shown by iPSC-Derived Oligodendrocytes

(2014) Yuko Numasawa-Kuroiwa et al.   Stem Cell Reports

Isogenic Human iPSC Parkinson’s Model Shows Nitrosative Stress-Induced Dysfunction in MEF2-PGC1α Transcription

(2013) Scott D. Ryan et al.   CELL

Human iPSC-Based Modeling of Late-Onset Disease via Progerin-Induced Aging

(2013) Justine D. Miller et al.   Cell Stem Cell

Genetic Correction of a LRRK2 Mutation in Human iPSCs Links Parkinsonian Neurodegeneration to ERK-Dependent Changes in Gene Expression

(2013) Peter Reinhardt 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

Modeling human neurological disorders with induced pluripotent stem cells

(2013) Yoichi Imaizumi et al.   JOURNAL OF NEUROCHEMISTRY

An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study

(2013) Timothy M Miller et al.   LANCET NEUROLOGY

Changes in cognition and behaviour in amyotrophic lateral sclerosis: nature of impairment and implications for assessment

(2013) Laura H Goldstein et al.   LANCET NEUROLOGY

Cerebral organoids model human brain development and microcephaly

(2013) Madeline A. Lancaster et al.   NATURE

RNA Toxicity from the ALS/FTD C9ORF72 Expansion Is Mitigated by Antisense Intervention

(2013) Christopher J. Donnelly et al.   NEURON

Astrocyte pathology and the absence of non-cell autonomy in an induced pluripotent stem cell model of TDP-43 proteinopathy

(2013) A. Serio et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

The energy cost of action potential propagation in dopamine neurons: clues to susceptibility in Parkinson's disease

(2013) Eleftheria K. Pissadaki et al.   Frontiers in Computational Neuroscience

The Presenilin-1 ΔE9 Mutation Results in Reduced γ-Secretase Activity, but Not Total Loss of PS1 Function, in Isogenic Human Stem Cells

(2013) Grace Woodruff et al.   Cell Reports

Neural Progenitors Derived From Human Induced Pluripotent Stem Cells Survive and Differentiate Upon Transplantation Into a Rat Model of Amyotrophic Lateral Sclerosis

(2013) Iuliana Ristea Popescu et al.   Stem Cells Translational Medicine

Genetic Correction of Huntington's Disease Phenotypes in Induced Pluripotent Stem Cells

(2012) Mahru C. An et al.   Cell Stem Cell

Frequency of the C9orf72 hexanucleotide repeat expansion in patients with amyotrophic lateral sclerosis and frontotemporal dementia: a cross-sectional study

(2012) Elisa Majounie et al.   LANCET NEUROLOGY

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

(2012) Mason A. Israel et al.   NATURE

Olfactory dysfunction in Parkinson disease

(2012) Richard L. Doty   Nature Reviews Neurology

100 years of Lewy pathology

(2012) Michel Goedert et al.   Nature Reviews Neurology

The Amyloid Precursor Protein Has a Flexible Transmembrane Domain and Binds Cholesterol

(2012) P. J. Barrett et al.   SCIENCE

Mitochondrial dysfunction associated with increased oxidative stress and α-synuclein accumulation in PARK2 iPSC-derived neurons and postmortem brain tissue

(2012) Yoichi Imaizumi et al.   Molecular Brain

A functional variant in ZNF512B is associated with susceptibility to amyotrophic lateral sclerosis in Japanese

(2011) Aritoshi Iida 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

Induced pluripotent stem cells — opportunities for disease modelling and drug discovery

(2011) Marica Grskovic et al.   NATURE REVIEWS DRUG DISCOVERY

Linking lipids to Alzheimer's disease: cholesterol and beyond

(2011) Gilbert Di Paolo et al.   NATURE REVIEWS NEUROSCIENCE

The chromosome 9 ALS and FTD locus is probably derived from a single founder

(2011) Kin Mok et al.   NEUROBIOLOGY OF AGING

Expanded GGGGCC Hexanucleotide Repeat in Noncoding Region of C9ORF72 Causes Chromosome 9p-Linked FTD and ALS

(2011) Mariely DeJesus-Hernandez et al.   NEURON

What Genetics Tells us About the Causes and Mechanisms of Parkinson's Disease

(2011) Olga Corti et al.   PHYSIOLOGICAL REVIEWS

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

Direct conversion of fibroblasts to functional neurons by defined factors

(2010) Thomas Vierbuchen et al.   NATURE

Self-Organized Formation of Polarized Cortical Tissues from ESCs and Its Active Manipulation by Extrinsic Signals

(2008) Mototsugu Eiraku et al.   Cell Stem Cell

Induced pluripotent stem cells from a spinal muscular atrophy patient

(2008) Allison D. Ebert et al.   NATURE

Neurons derived from reprogrammed fibroblasts functionally integrate into the fetal brain and improve symptoms of rats with Parkinson's disease

(2008) M. Wernig et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA