4.7 Review

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

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。
Review Cell & Tissue Engineering

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

Elisa Giacomelli et al.

Summary: This article evaluates the status of using induced pluripotent stem cells (iPSCs) to model neurodegenerative diseases, focusing on amyotrophic lateral sclerosis (ALS) as an example. It discusses the methods and challenges associated with deriving and using disease-relevant neuronal and glial lineages.

CELL STEM CELL (2022)

Article Geriatrics & Gerontology

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

Ryoichi Nakamura et al.

Summary: Two recent genetic studies found that loss-of-function mutation of the C-terminal cargo-binding tail domain of the KIF5A gene cause amyotrophic lateral sclerosis (ALS). This study in Japanese patients with sporadic ALS identified a novel loss-of-function variant in KIF5A, highlighting its rare occurrence as a cause of ALS.

NEUROBIOLOGY OF AGING (2021)

Article Biology

The therapeutic potential of multiclonal tumoricidal T cells derived from tumor infiltrating lymphocyte-1derived iPS cells

Takeshi Ito et al.

Summary: Ito et al. generated and characterized multiclonal tumoricidal T cells derived from TIL-iPSC, showing improved functions and potential therapeutic use. The study also investigated the optimization of TIL preparation to enhance anti-cancer effects, suggesting that cell reprogramming could play a role in improving outcomes of TIL-based therapies.

COMMUNICATIONS BIOLOGY (2021)

Review Biochemistry & Molecular Biology

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

Meritxell Pons-Espinal et al.

Summary: Parkinson's disease is an incurable neurodegenerative disease characterized by movement impairments due to the loss of dopamine neurons. Recent advances in iPSC technology have allowed for the recreation of PD-related pathological phenotypes, shedding light on early disease mechanisms.

CELLULAR AND MOLECULAR LIFE SCIENCES (2021)

Article Clinical Neurology

Effect of Ezogabine on Cortical and Spinal Motor Neuron Excitability in Amyotrophic Lateral Sclerosis A Randomized Clinical Trial

Brian J. Wainger et al.

Summary: This study found that ezogabine can decrease cortical and spinal motor neuron excitability in patients with ALS, indicating the potential use of neurophysiological metrics as pharmacodynamic biomarkers in multi-site clinical trials.

JAMA NEUROLOGY (2021)

Article Biochemistry & Molecular Biology

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

Hayato Fukusumi et al.

Summary: This study found increased accumulation of alpha-synuclein in DA neurons derived from PARK4 patients, but the accumulation disappeared over time. An SNCA-specific antisense oligonucleotide could reduce alpha-synuclein levels during the accumulation stage.

FEBS OPEN BIO (2021)

Article Immunology

Gut microbiota-driven brain Aβ amyloidosis in mice requires microglia

Hemraj B. Dodiya et al.

Summary: Researchers characterized a mouse model of A beta amyloidosis with a perturbed microbiome and demonstrated the causality between gut microbiota, microglia, A beta, and neurodegeneration using fecal transplantation approaches and microglial depletion studies. They found that short-term high-dose antibiotic treatment in preweaned male mice led to reductions in A beta amyloidosis and degenerative changes, and fecal microbiota transplantation from donor mice restored these changes. Microglia play a critical role in driving gut microbiome-mediated alterations of cerebral A beta deposition.

JOURNAL OF EXPERIMENTAL MEDICINE (2021)

Article Clinical Neurology

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

Keiko Imamura et al.

Summary: This study developed an ALS prediction model using artificial intelligence and iPSC technology, achieving high accuracy in analyzing images of spinal motor neurons, which may support the diagnosis and potential treatment of ALS through future prospective research.

ANNALS OF NEUROLOGY (2021)

Editorial Material Cell & Tissue Engineering

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

Jun Takahashi

Summary: These two papers demonstrate the induction of dopaminergic neurons from human embryonic stem cells and lay the foundation for future clinical trials.

CELL STEM CELL (2021)

Article Cell & Tissue Engineering

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

Jinghua Piao et al.

Summary: The study successfully generated midbrain dopamine neurons from human embryonic stem cells and conducted extensive tests on animals to demonstrate the efficacy and safety of the cell product intended for potential human use, paving the way for future clinical trials.

CELL STEM CELL (2021)

Article Neurosciences

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

Pranav Preman et al.

Summary: Transplanting human induced pluripotent stem cell (hiPSC)-derived astrocyte progenitors into the mouse brain offers a promising approach to study human astrocyte pathophysiology in the context of Alzheimer's disease. The transplanted astrocytes develop and integrate functionally within the mouse host brain, maintaining human-specific characteristics and responses to amyloid plaques in Alzheimer's chimeric brains. This method provides valuable insights into the differences between human and rodent astrocytes and their role in neuroinflammatory and neurodegenerative processes.

MOLECULAR NEURODEGENERATION (2021)

Article Genetics & Heredity

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

Wouter van Rheenen et al.

Summary: A cross-ancestry genome-wide association meta-analysis of amyotrophic lateral sclerosis (ALS) identified 15 risk loci with distinct genetic architectures and neuron-specific biology. The study emphasized the urgent need for disease-modifying therapies for ALS. High cholesterol levels were suggested to have a causal role in the onset of ALS, among other environmental and lifestyle risk factors.

NATURE GENETICS (2021)

Article Biochemical Research Methods

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

Ziga Avsec et al.

Summary: Enformer leverages a new deep learning architecture to improve gene expression prediction accuracy based on DNA sequences, integrating information from long-range interactions in the genome and accurately predicting the impact of genetic variants on gene expression. Additionally, Enformer has learned to predict enhancer-promoter interactions directly from DNA sequences.

NATURE METHODS (2021)

News Item Biotechnology & Applied Microbiology

ALS antisense drug falters in phase III

Asher Mullard

Summary: The SOD1-antisense oligonucleotide tofersen, developed by Biogen and Ionis, failed a phase III trial, prompting concerns about the drug's future and the direction of ALS trials to come.

NATURE REVIEWS DRUG DISCOVERY (2021)

Review Pharmacology & Pharmacy

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

Chao Ma et al.

Summary: Organ-on-a-Chip technology has the potential to revolutionize drug development by mimicking the physiological environment and function of human organs on a chip. However, challenges remain in translating and commercializing this technology for pharmacological and medical applications.

TRENDS IN PHARMACOLOGICAL SCIENCES (2021)

Article Biology

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

Cynthia Tang et al.

Summary: Mutations in CLN3 lead to photoreceptor cell loss in CLN3 disease, affecting the phagocytosis of photoreceptor outer segments by retinal pigment epithelium (RPE) cells. Patient-derived cells show that CLN3 is essential for RPE cell structure and phagocytosis, suggesting potential for gene therapy targeting RPE cells in treating CLN3 disease.

COMMUNICATIONS BIOLOGY (2021)

Review Cell & Tissue Engineering

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

Ary Marsee et al.

Summary: HPB organoids are powerful tools for studying organ development, disease, and regeneration. By introducing a classification system and nomenclature, experts aim to standardize and validate these structures, while also addressing challenges to their clinical application.

CELL STEM CELL (2021)

Article Cell & Tissue Engineering

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

Jerome Mertens et al.

Summary: Our study identifies AD-related neuronal changes as age-dependent cellular programs that impair neuronal identity.

CELL STEM CELL (2021)

Review Medicine, General & Internal

Parkinson's disease

Bastiaan R. Bloem et al.

Summary: Parkinson's disease is a neurodegenerative condition with diverse causes and clinical presentations. Diagnosis is clinically based, and treatment goals vary from person to person, emphasizing the need for personalized management. Currently, there is no therapy to slow down or arrest the progression of Parkinson's disease.

LANCET (2021)

Article Multidisciplinary Sciences

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

Jonathan Li et al.

Summary: Studying iPSC-derived neurons sheds light on disease-related outcomes, revealing both novel and known dysregulated pathways. Variations in -omics may occur with different differentiation protocols, but consistent core dysregulated pathways help elucidate network-based signatures in neurodegenerative diseases.

ISCIENCE (2021)

News Item Multidisciplinary Sciences

GENETIC THERAPIES FOR HUNTINGTON'S DISEASE FAIL IN CLINICAL TRIALS

Diana Kwon

NATURE (2021)

News Item Biotechnology & Applied Microbiology

Double setback for ASO trials in Huntington disease

Katie Kingwell

NATURE REVIEWS DRUG DISCOVERY (2021)

Article Cell Biology

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

Hideyuki Nakashima et al.

Summary: This study reveals the mechanism of MeCP2/miR-199a axis in regulating neural stem cell differentiation, further confirming the importance of BMP signaling in RTT brain development, and proposing the MeCP2/miR-199a/Smad1 axis as a potential therapeutic target for RTT.

CELL REPORTS (2021)

Article Medicine, General & Internal

Alzheimer disease

David S. Knopman et al.

Summary: Alzheimer's disease is a neurodegenerative disorder characterized by cognitive impairments. The pathophysiology involves a complex interplay of loss of synaptic homeostasis and dysfunction in endosomal/lysosomal clearance pathways. Therapeutic options are still being sought to substantially alter the clinical course of individuals with Alzheimer's disease.

NATURE REVIEWS DISEASE PRIMERS (2021)

Review Cell Biology

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

Ewelina Latoszek et al.

Summary: The review highlights the essential role of SOCE in the physiology of neural stem cells and its dysregulation in HD pathology. Recent studies demonstrate the impact of elevated STIM2 expression on excessive Ca2+ entry in juvenile-onset HD-induced GABAergic cells. Progress in the in vitro differentiation of MSNs derived from various cell sources is discussed, along with the establishment of HD brain organoids for studying HD pathology and potential treatments. Stem cell therapy, particularly the transplantation of MSNs or their precursors, shows promise in restoring normal neural cell function in HD patients.

FRONTIERS IN CELL AND DEVELOPMENTAL BIOLOGY (2021)

Review Cell 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

Konstantinos Avgerinos et al.

Summary: Monoclonal antibodies targeting A beta have shown small clinical improvements and large biomarker improvements in Alzheimer's disease patients. However, they also increase the risk of adverse events such as ARIA.

AGEING RESEARCH REVIEWS (2021)

Review Neurosciences

Parkinson's Disease Genetics and Pathophysiology

Gabriel E. Vazquez-Velez et al.

Summary: Parkinson's disease is a common neurodegenerative disorder characterized by degeneration of the substantia nigra pars compacta and accumulation of alpha-synuclein in Lewy bodies. Genetic factors play an important role in PD pathophysiology, with dysfunction of endosomal, lysosomal, and mitochondrial pathways being associated with the disease. This knowledge could be utilized for developing disease-modifying therapeutics.

ANNUAL REVIEW OF NEUROSCIENCE, VOL 44, 2021 (2021)

Article Multidisciplinary Sciences

Highly accurate protein structure prediction with AlphaFold

John Jumper et al.

Summary: Proteins are essential for life, and accurate prediction of their structures is a crucial research problem. Current experimental methods are time-consuming, highlighting the need for accurate computational approaches to address the gap in structural coverage. Despite recent progress, existing methods fall short of atomic accuracy in protein structure prediction.

NATURE (2021)

Article Multidisciplinary Sciences

Highly accurate protein structure prediction for the human proteome

Kathryn Tunyasuvunakool et al.

Summary: Using the AlphaFold method, the structural coverage of the proteome has been significantly expanded, covering 98.5% of human proteins with 58% of residues having confident predictions and 36% having very high confidence. Introducing new metrics to interpret the dataset and identify disordered regions, this study aims to provide high-quality predictions for generating biological hypotheses.

NATURE (2021)

Article Biotechnology & Applied Microbiology

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

Tetsuya Nagata et al.

Summary: This study demonstrates that DNA/RNA heteroduplex oligonucleotides (HDOs) conjugated to cholesterol or alpha-tocopherol can regulate endogenous gene expression in the central nervous system when administered systemically, with limited side effects. The HDOs effectively distribute throughout the CNS and suppress the expression of target genes by up to 90%, showing potential for ASO-based therapies for neurological diseases.

NATURE BIOTECHNOLOGY (2021)

Review Neurosciences

Advancing models of neural development with biomaterials

Julien G. Roth et al.

Summary: Human pluripotent stem cell-derived in vitro models have the potential to study aspects of human brain development, but they currently have limitations in fully recapitulating certain features of human neural development. Engineered biomaterials have the ability to create more biomimetic neural microenvironments, but further refinement is required before widespread implementation.

NATURE REVIEWS NEUROSCIENCE (2021)

Article Multidisciplinary Sciences

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

Katharina Cosker et al.

Summary: The study revealed that microglia carrying the TREM2 R47H variant have impaired response to cell damage signals, potentially contributing to the progression of late onset Alzheimer's disease.

SCIENTIFIC REPORTS (2021)

Article Medicine, General & Internal

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

Takayuki Kondo et al.

Summary: AD is a common cause of dementia, with PSEN1 gene mutations being a frequent cause. Current treatments for PSEN1-AD are limited, but iPSC-based drug repurposing identified bromocriptine as a therapeutic candidate. A multicentre trial will investigate the safety and efficacy of bromocriptine in PSEN1-AD patients, with primary outcomes focusing on cognitive and psychological function. The study is conducted ethically and results will be disseminated at conferences and published in journals.

BMJ OPEN (2021)

Article Cell Biology

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

Xuan Huang et al.

Summary: Screening using patient-derived motor neurons has identified multiple effective targets for managing hyperexcitability in ALS, demonstrating the potential of human disease cell-based phenotypic screens in identifying clinically relevant targets for neurological disorders.

CELL REPORTS (2021)

Review Neurosciences

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

Mark Tomishima et al.

Summary: After years of preclinical development, cell and gene therapies have become important products in Parkinson's disease clinical trials, with proven efficacy in preclinical models, but now need further validation of functionality and safety in clinical trials.

JOURNAL OF PARKINSONS DISEASE (2021)

Article Cell & Tissue Engineering

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

Sayaka Deguchi et al.

Summary: Experimental cell models using human ES/iPS cell-derived somatic cells and organoids play a crucial role in understanding the pathophysiology of COVID-19 and developing therapeutic agents, by replicating SARS-CoV-2 infection and damage in various organs to evaluate antiviral efficacy and safety of potential treatments.

STEM CELLS TRANSLATIONAL MEDICINE (2021)

Article Cell & Tissue Engineering

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

Shio Mitsuzawa et al.

Summary: The study found that reduced expression of PHOX2B caused by TARDBP mutations may contribute to increased axonal vulnerability in amyotrophic lateral sclerosis (ALS). Knockdown of PHOX2B resulted in decreased neurite length in human MNs and induced abnormal spinal axons and impaired escape response in zebrafish. This suggests that loss of axonal resilience mediated by PHOX2B downregulation is an important ALS-related phenotype.

STEM CELL REPORTS (2021)

Article Cell & Tissue Engineering

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

Justyna Okarmus et al.

Summary: PARK2 knockout cells exhibit increased TCA cycle activity, disturbed mitochondrial ultrastructure, ATP depletion, and dysregulation of glycolysis and carnitine metabolism. These perturbations are associated with elevated oxidative stress and reduced anti-oxidative response. Key findings were validated using patient-specific iPSC-derived neurons.

STEM CELL REPORTS (2021)

Article Cell Biology

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

Enrique Armijo et al.

Summary: Alzheimer's disease is the most common type of dementia in elderly population, characterized by progressive memory loss and cerebral atrophy. Induced pluripotent stem cells (iPSCs) have been suggested as a promising therapy to benefit patients at advanced clinical and pathological stages of AD.
Review Clinical Neurology

The genetic architecture of Parkinson's disease

Cornelis Blauwendraat et al.

LANCET NEUROLOGY (2020)

Review Pharmacology & Pharmacy

Ropinirole, a New ALS Drug Candidate Developed Using iPSCs

Hideyuki Okano et al.

TRENDS IN PHARMACOLOGICAL SCIENCES (2020)

Review Neurosciences

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

Katrin Simmnacher et al.

FRONTIERS IN CELLULAR NEUROSCIENCE (2020)

Article Biochemistry & Molecular Biology

Visualizing and Modulating Mitophagy for Therapeutic Studies of Neurodegeneration

Hiroyuki Katayama et al.

Review Cell & Tissue Engineering

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

Arun Sharma et al.

CELL STEM CELL (2020)

Article Biochemistry & Molecular Biology

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

Razvan-Marius Brazdis et al.

HUMAN MOLECULAR GENETICS (2020)

Article Medicine, General & Internal

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

Jeffrey S. Schweitzer et al.

NEW ENGLAND JOURNAL OF MEDICINE (2020)

Review Neurosciences

Rising Stars: Astrocytes as a Therapeutic Target for ALS Disease

Michal Izrael et al.

FRONTIERS IN NEUROSCIENCE (2020)

Review Cell & Tissue Engineering

Pluripotent Stem Cell-Based Cell Therapy- Promise and Challenges

Shinya Yamanaka

CELL STEM CELL (2020)

Article Medicine, General & Internal

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

Timothy Miller et al.

NEW ENGLAND JOURNAL OF MEDICINE (2020)

Review Developmental Biology

New trends in cellular therapy

Hideyuki Okano et al.

DEVELOPMENT (2020)

Article Multidisciplinary Sciences

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

Kevin A. Guttenplan et al.

NATURE COMMUNICATIONS (2020)

Article Medicine, General & Internal

Trial of Sodium Phenylbutyrate-Taurursodiol for Amyotrophic Lateral Sclerosis

Sabrina Paganoni et al.

NEW ENGLAND JOURNAL OF MEDICINE (2020)

Editorial Material Multidisciplinary Sciences

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

Guoping Feng et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2020)

Article Engineering, Biomedical

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

Jun Takahashi

Regenerative Therapy (2020)

Article Neurosciences

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

Hideyuki Okano

Annual Review of Neuroscience (2020)

Review Clinical Neurology

The microglial component of amyotrophic lateral sclerosis

Benjamin E. Clarke et al.

Article Biochemistry & Molecular Biology

Generation of Functional Human 3D Cortico-Motor Assembloids

Jimena Andersen et al.

Review Neurosciences

The Neglected Genes of ALS: Cytoskeletal Dynamics Impact Synaptic Degeneration in ALS

Maria Jose Castellanos-Montiel et al.

FRONTIERS IN CELLULAR NEUROSCIENCE (2020)

Article Genetics & Heredity

Genetics of Amyotrophic Lateral Sclerosis

Jenna M. Gregory et al.

CURRENT GENETIC MEDICINE REPORTS (2020)

Review Biochemistry & Molecular Biology

NAD+metabolism: pathophysiologic mechanisms and therapeutic potential

Na Xie et al.

SIGNAL TRANSDUCTION AND TARGETED THERAPY (2020)

Article Biochemical Research Methods

Flow-enhanced vascularization and maturation of kidney organoids in vitro

Kimberly A. Homan et al.

NATURE METHODS (2019)

Article Medicine, General & Internal

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

C. V. M. Verschuur et al.

NEW ENGLAND JOURNAL OF MEDICINE (2019)

Article Biochemistry & Molecular Biology

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

Akira Mizuno et al.

EUROPEAN JOURNAL OF HUMAN GENETICS (2019)

Article Medicine, General & Internal

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

Tetsuya Akiyama et al.

EBIOMEDICINE (2019)

Review Biochemistry & Molecular Biology

Getting to the Cores of Autism

Lilia M. Iakoucheva et al.

Article Multidisciplinary Sciences

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

Hiroyuki Koike et al.

NATURE (2019)

Article Genetics & Heredity

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

Shigeki Hirabayashi et al.

NATURE GENETICS (2019)

Review Biochemistry & Molecular Biology

Alzheimer Disease: An Update on Pathobiology and Treatment Strategies

Justin M. Long et al.

Article Biochemistry & Molecular Biology

Mutations in CHCHD2 cause α-synuclein aggregation

Aya Ikeda et al.

HUMAN MOLECULAR GENETICS (2019)

Review Biochemistry & Molecular Biology

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

Elvira Immacolata Parrotta et al.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2019)

Article Medicine, Research & Experimental

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

Daniel M. Ramos et al.

JOURNAL OF CLINICAL INVESTIGATION (2019)

Review Cell Biology

Microglia in Alzheimer's disease

David V. Hansen et al.

JOURNAL OF CELL BIOLOGY (2018)

Article Neurosciences

Microglia-Mediated Synapse Loss in Alzheimer's Disease

Lawrence Rajendran et al.

JOURNAL OF NEUROSCIENCE (2018)

Review Neurosciences

The multi-dimensional roles of astrocytes in ALS

Koji Yamanaka et al.

NEUROSCIENCE RESEARCH (2018)

Article Cell & Tissue Engineering

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

Philip W. Brownjohn et al.

STEM CELL REPORTS (2018)

Article Biochemistry & Molecular Biology

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

Koki Fujimori et al.

NATURE MEDICINE (2018)

Review Clinical Neurology

Nusinersen for the treatment of spinal muscular atrophy

Claudia A. Chiriboga

EXPERT REVIEW OF NEUROTHERAPEUTICS (2017)

Article Medicine, Research & Experimental

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

Kyosuke Hino et al.

JOURNAL OF CLINICAL INVESTIGATION (2017)

Review Medicine, Research & Experimental

Microglia and C9orf72 in neuroinflammation and ALS and frontotemporal dementia

Deepti Lall et al.

JOURNAL OF CLINICAL INVESTIGATION (2017)

Review Medicine, General & Internal

Amyotrophic lateral sclerosis

Michael A. van Es et al.

LANCET (2017)

Article Multidisciplinary Sciences

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

Tetsuhiro Kikuchi et al.

NATURE (2017)

Editorial Material Neurosciences

Testing the Amyloid Hypothesis with a Humanized AD Mouse Model

Michelle K. Cahill et al.

NEURON (2017)

Article Medicine, General & Internal

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

R. S. Finkel et al.

NEW ENGLAND JOURNAL OF MEDICINE (2017)

Article Multidisciplinary Sciences

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

Mohini Jangi et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2017)

Article Cell Biology

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

Keiko Imamura et al.

SCIENCE TRANSLATIONAL MEDICINE (2017)

Review Clinical Neurology

Nusinersen for the treatment of spinal muscular atrophy

Claudia A. Chiriboga

EXPERT REVIEW OF NEUROTHERAPEUTICS (2017)

Article Cell & Tissue Engineering

Sporadic ALS Astrocytes Induce Neuronal Degeneration In Vivo

Kun Qian et al.

STEM CELL REPORTS (2017)

Article Medicine, General & Internal

Parkinson disease

Werner Poewe et al.

NATURE REVIEWS DISEASE PRIMERS (2017)

Review Neurosciences

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

Panchanan Maiti et al.

TRANSLATIONAL NEURODEGENERATION (2017)

Article Biochemistry & Molecular Biology

Efficient induction of dopaminergic neuron differentiation from induced pluripotent stem cells reveals impaired mitophagy in PARK2 neurons

Sadafumi Suzuki et al.

BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS (2017)

Article Clinical Neurology

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

Hazuki Watanabe et al.

JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY (2016)

Review Multidisciplinary Sciences

Decoding ALS: from genes to mechanism

J. Paul Taylor et al.

NATURE (2016)

Review Cell Biology

Pluripotent stem cells in disease modelling and drug discovery

Yishai Avior et al.

NATURE REVIEWS MOLECULAR CELL BIOLOGY (2016)

Review Neurosciences

ASTROCYTES IN PHYSIOLOGICAL AGING AND ALZHEIMER'S DISEASE

J. J. Rodriguez-Arellano et al.

NEUROSCIENCE (2016)

Article Multidisciplinary Sciences

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

Laura Ferraiuolo et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2016)

Article Biochemical Research Methods

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

Michelle Richner et al.

NATURE PROTOCOLS (2015)

Review Cell & Tissue Engineering

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

Kyle D. Fink et al.

REGENERATIVE MEDICINE (2015)

Article Biochemistry & Molecular Biology

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

Bethany L. Johnson-Kerner et al.

HUMAN MOLECULAR GENETICS (2015)

Article Cell & Tissue Engineering

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

Evangelos Kiskinis et al.

CELL STEM CELL (2014)

Review Biochemistry & Molecular Biology

Modeling human neurological disorders with induced pluripotent stem cells

Yoichi Imaizumi et al.

JOURNAL OF NEUROCHEMISTRY (2014)

Article Multidisciplinary Sciences

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

Se Hoon Choi et al.

NATURE (2014)

Article Multidisciplinary Sciences

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

Chul-Yong Park et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2014)

Article Cell & Tissue Engineering

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

Takayuki Kondo et al.

STEM CELL REPORTS (2014)

Article Cell & Tissue Engineering

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

Justine D. Miller et al.

CELL STEM CELL (2013)

Article Multidisciplinary Sciences

Cerebral organoids model human brain development and microcephaly

Madeline A. Lancaster et al.

NATURE (2013)

Review Clinical Neurology

100 years of Lewy pathology

Michel Goedert et al.

NATURE REVIEWS NEUROLOGY (2013)

Article Multidisciplinary Sciences

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

Andrea Serio et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2013)

Article Mathematical & Computational Biology

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

Eleftheria K. Pissadaki et al.

FRONTIERS IN COMPUTATIONAL NEUROSCIENCE (2013)

Article Cell & Tissue Engineering

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

Mahru C. An et al.

CELL STEM CELL (2012)

Article Multidisciplinary Sciences

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

Mason A. Israel et al.

NATURE (2012)

Review Clinical Neurology

Olfactory dysfunction in Parkinson disease

Richard L. Doty

NATURE REVIEWS NEUROLOGY (2012)

Article Geriatrics & Gerontology

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

Kin Mok et al.

NEUROBIOLOGY OF AGING (2012)

Article Multidisciplinary Sciences

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

Paul J. Barrett et al.

SCIENCE (2012)

Article Biochemistry & Molecular Biology

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

Aritoshi Iida et al.

HUMAN MOLECULAR GENETICS (2011)

Article Multidisciplinary Sciences

Modelling schizophrenia using human induced pluripotent stem cells

Kristen J. Brennand et al.

NATURE (2011)

Review Biotechnology & Applied Microbiology

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

Marica Grskovic et al.

NATURE REVIEWS DRUG DISCOVERY (2011)

Review Neurosciences

Linking lipids to Alzheimer's disease: cholesterol and beyond

Gilbert Di Paolo et al.

NATURE REVIEWS NEUROSCIENCE (2011)

Review Physiology

WHAT GENETICS TELLS US ABOUT THE CAUSES AND MECHANISMS OF PARKINSON'S DISEASE

Olga Corti et al.

PHYSIOLOGICAL REVIEWS (2011)

Article Multidisciplinary Sciences

Direct conversion of fibroblasts to functional neurons by defined factors

Thomas Vierbuchen et al.

NATURE (2010)

Article Multidisciplinary Sciences

Induced pluripotent stem cells from a spinal muscular atrophy patient

Allison D. Ebert et al.

NATURE (2009)

Article Multidisciplinary Sciences

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

Marius Wernig et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2008)

Article Biochemistry & Molecular Biology

Induction of pluripotent stem cells from adult human fibroblasts by defined factors

Kazutoshi Takahashi et al.

Article Clinical Neurology

Restoration of normal motor control in Parkinson's disease during REM sleep

Valerie Cochen De Cock et al.

Article Multidisciplinary Sciences

Onset and progression in inherited ALS determined by motor neurons and microglia

Severine Boillee et al.

SCIENCE (2006)

Article Multidisciplinary Sciences

Cap analysis gene expression for high-throughput analysis of transcriptional starting point and identification of promoter usage

T Shiraki et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2003)