Identification of Therapeutic Targets for Amyotrophic Lateral Sclerosis Using PandaOmics – An AI-Enabled Biological Target Discovery Platform

Answer ALS, a large-scale resource for sporadic and familial ALS combining clinical and multi-omics data from induced pluripotent cell lines

(2022) Emily G. Baxi et al.   NATURE NEUROSCIENCE

Hallmarks of aging-based dual-purpose disease and age-associated targets predicted using PandaOmics AI-powered discovery engine

(2022) Frank W. Pun et al.   Aging-US

Treating Duchenne Muscular Dystrophy: The Promise of Stem Cells, Artificial Intelligence, and Multi-Omics

(2022) Carlos D. Vera et al.   Frontiers in Cardiovascular Medicine

Global transcriptome profile of the developmental principles of in vitro iPSC-to-motor neuron differentiation

(2021) Emilia Solomon et al.   BMC Molecular and Cell Biology

Multi-OMICS study of a CHCHD10 variant causing ALS demonstrates metabolic rewiring and activation of endoplasmic reticulum and mitochondrial unfolded protein responses

(2021) Isabella R Straub et al.   HUMAN MOLECULAR GENETICS

Fibroblast Growth Factor Signalling in the Diseased Nervous System

(2021) Lars Klimaschewski et al.   MOLECULAR NEUROBIOLOGY

Emerging genetic complexity and rare genetic variants in neurodegenerative brain diseases

(2021) Federica Perrone et al.   Genome Medicine

Nuclear accumulation of CHMP7 initiates nuclear pore complex injury and subsequent TDP-43 dysfunction in sporadic and familial ALS

(2021) Alyssa N. Coyne et al.   Science Translational Medicine

RNA Is a Double-Edged Sword in ALS Pathogenesis

(2021) Benjamin L. Zaepfel et al.   Frontiers in Cellular Neuroscience

TDP-43 regulates GAD1 mRNA splicing and GABA signaling in Drosophila CNS

(2021) Giulia Romano et al.   Scientific Reports

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

(2021) Jonathan Li et al.   iScience

Going the Extra (Synaptic) Mile: Excitotoxicity as the Road Toward Neurodegenerative Diseases

(2020) Adam Armada-Moreira et al.   Frontiers in Cellular Neuroscience

The Advent of Generative Chemistry

(2020) Quentin Vanhaelen et al.   ACS Medicinal Chemistry Letters

Omics-based exploration and functional validation of neurotrophic factors and histamine as therapeutic targets in ALS

(2020) Cinzia Volonté et al.   AGEING RESEARCH REVIEWS

The role of DNA damage response in amyotrophic lateral sclerosis

(2020) Yu Sun et al.   Essays in Biochemistry

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

(2020) Ryoichi Nakamura et al.   Communications Biology

Common Factors in Neurodegeneration: A Meta-Study Revealing Shared Patterns on a Multi-Omics Scale

(2020) Nicolas Ruffini et al.   Cells

Voltage-Gated Potassium Channels as Regulators of Cell Death

(2020) Magdalena Bachmann et al.   Frontiers in Cell and Developmental Biology

ALS blood expression profiling identifies new biomarkers, patient subgroups, and evidence for neutrophilia and hypoxia

(2019) William R. Swindell et al.   Journal of Translational Medicine

Splicing repression is a major function of TDP-43 in motor neurons

(2019) Aneesh Donde et al.   ACTA NEUROPATHOLOGICA

Epidemiology of amyotrophic lateral sclerosis

(2019) Elisa Longinetti et al.   CURRENT OPINION IN NEUROLOGY

Modeling Alzheimer’s disease with iPSC-derived brain cells

(2019) Jay Penney et al.   MOLECULAR PSYCHIATRY

Ageing as a risk factor for neurodegenerative disease

(2019) Yujun Hou et al.   Nature Reviews Neurology

Fighting the Fire: Mechanisms of Inflammatory Gene Regulation by the Glucocorticoid Receptor

(2019) Laura Escoter-Torres et al.   Frontiers in Immunology

Long-term effects of the glucocorticoid receptor modulator CORT113176 in murine motoneuron degeneration

(2019) Maria Meyer et al.   BRAIN RESEARCH

The reactome pathway knowledgebase

(2019) Bijay Jassal et al.   NUCLEIC ACIDS RESEARCH

IRE1 promotes neurodegeneration through autophagy-dependent neuron death in the Drosophila model of Parkinson’s disease

(2019) Cheng Yan et al.   Cell Death & Disease

Postmortem Cortex Samples Identify Distinct Molecular Subtypes of ALS: Retrotransposon Activation, Oxidative Stress, and Activated Glia

(2019) Oliver H. Tam et al.   Cell Reports

Decoding the relationship between ageing and amyotrophic lateral sclerosis: a cellular perspective

(2019) Virenkumar A Pandya et al.   BRAIN

ALS Genetics, Mechanisms, and Therapeutics: Where Are We Now?

(2019) Rita Mejzini et al.   Frontiers in Neuroscience

Transcriptome–pathology correlation identifies interplay between TDP-43 and the expression of its kinase CK1E in sporadic ALS

(2018) Florian Krach et al.   ACTA NEUROPATHOLOGICA

Amyotrophic lateral sclerosis (ALS) and Alzheimer’s disease (AD) are characterised by differential activation of ER stress pathways: focus on UPR target genes

(2018) L. Montibeller et al.   CELL STRESS & CHAPERONES

How is edaravone effective against acute ischemic stroke and amyotrophic lateral sclerosis?

(2018) Kazutoshi Watanabe et al.   JOURNAL OF CLINICAL BIOCHEMISTRY AND NUTRITION

Impaired Cu–Zn Superoxide Dismutase (SOD1) and Calcineurin (Cn) Interaction in ALS: A Presumed Consequence for TDP-43 and Zinc Aggregation in Tg SOD1G93A Rodent Spinal Cord Tissue

(2018) Jolene M. Kim et al.   NEUROCHEMICAL RESEARCH

Genome-wide Analyses Identify KIF5A as a Novel ALS Gene

(2018) Aude Nicolas et al.   NEURON

The Selective Glucocorticoid Receptor Modulator Cort 113176 Reduces Neurodegeneration and Neuroinflammation in Wobbler Mice Spinal Cord

(2018) Maria Meyer et al.   NEUROSCIENCE

An open label study of a novel immunosuppression intervention for the treatment of amyotrophic lateral sclerosis

(2018) Christina N. Fournier et al.   Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration

Prevalence of Amyotrophic Lateral Sclerosis — United States, 2014

(2018) Paul Mehta et al.   MMWR-MORBIDITY AND MORTALITY WEEKLY REPORT

Artificial intelligence for aging and longevity research: Recent advances and perspectives

(2018) Alex Zhavoronkov et al.   AGEING RESEARCH REVIEWS

Trehalose induces autophagy via lysosomal-mediated TFEB activation in models of motoneuron degeneration

(2018) Paola Rusmini et al.   Autophagy

Neuro-Immune Hemostasis: Homeostasis and Diseases in the Central Nervous System

(2018) Ciro De Luca et al.   Frontiers in Cellular Neuroscience

Prevalence of Amyotrophic Lateral Sclerosis — United States, 2015

(2018) Paul Mehta et al.   MMWR-MORBIDITY AND MORTALITY WEEKLY REPORT

Increased cytoplasmic TDP-43 reduces global protein synthesis by interacting with RACK1 on polyribosomes

(2017) Arianna Russo et al.   HUMAN MOLECULAR GENETICS

Genetic epidemiology of amyotrophic lateral sclerosis: a systematic review and meta-analysis

(2017) Zhang-Yu Zou et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Safety and efficacy of edaravone in well defined patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trial

(2017) Koji Abe et al.   LANCET NEUROLOGY

Copy Number Variations in Amyotrophic Lateral Sclerosis: Piecing the Mosaic Tiles Together through a Systems Biology Approach

(2017) Giovanna Morello et al.   MOLECULAR NEUROBIOLOGY

The evolving genetic risk for sporadic ALS

(2017) Summer B. Gibson et al.   NEUROLOGY

Use of deep neural network ensembles to identify embryonic-fetal transition markers: repression of COX7A1 in embryonic and cancer cells

(2017) Michael D. West et al.   Oncotarget

Post-hoc analysis of MCI186-17, the extension study to MCI186-16, the confirmatory double-blind, parallel-group, placebo-controlled study of edaravone in amyotrophic lateral sclerosis

(2017) Fumihiro Takahashi et al.   Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration

Current Advances and Limitations in Modeling ALS/FTD in a Dish Using Induced Pluripotent Stem Cells

(2017) Wenting Guo et al.   Frontiers in Neuroscience

Deep Learning Applications for Predicting Pharmacological Properties of Drugs and Drug Repurposing Using Transcriptomic Data

(2016) Alexander Aliper et al.   MOLECULAR PHARMACEUTICS

Genome-wide association analyses identify new risk variants and the genetic architecture of amyotrophic lateral sclerosis

(2016) Wouter van Rheenen et al.   NATURE GENETICS

Increased oxidative stress in patients with amyotrophic lateral sclerosis and the effect of edaravone administration

(2016) Midori Nagase et al.   REDOX REPORT

4-Aminopyridine Induced Activity Rescues Hypoexcitable Motor Neurons from Amyotrophic Lateral Sclerosis Patient-Derived Induced Pluripotent Stem Cells

(2016) Maximilian Naujock et al.   STEM CELLS

In silico Pathway Activation Network Decomposition Analysis (iPANDA) as a method for biomarker development

(2016) Ivan V. Ozerov et al.   Nature Communications

Unraveling gene expression profiles in peripheral motor nerve from amyotrophic lateral sclerosis patients: insights into pathogenesis

(2016) Nilo Riva et al.   Scientific Reports

Clinical Spectrum of Amyotrophic Lateral Sclerosis (ALS)

(2016) Leslie I. Grad et al.   Cold Spring Harbor Perspectives in Medicine

Pathway activation profiling reveals new insights into Age-related Macular Degeneration and provides avenues for therapeutic interventions

(2016) Evgeny Makarev et al.   Aging-US

Clinical and genetic basis of familial amyotrophic lateral sclerosis

(2015) Paulo Victor Sgobbi de Souza et al.   ARQUIVOS DE NEURO-PSIQUIATRIA

The ASK1-specific inhibitors K811 and K812 prolong survival in a mouse model of amyotrophic lateral sclerosis

(2015) Takao Fujisawa et al.   HUMAN MOLECULAR GENETICS

PPARβ/δ Agonist Provides Neuroprotection by Suppression of IRE1α–Caspase-12-Mediated Endoplasmic Reticulum Stress Pathway in the Rotenone Rat Model of Parkinson’s Disease

(2015) Qiang Tong et al.   MOLECULAR NEUROBIOLOGY

The C9orf72 repeat expansion disrupts nucleocytoplasmic transport

(2015) Ke Zhang et al.   NATURE

Distinct brain transcriptome profiles in C9orf72-associated and sporadic ALS

(2015) Mercedes Prudencio et al.   NATURE NEUROSCIENCE

Edaravone leads to proteome changes indicative of neuronal cell protection in response to oxidative stress

(2015) Mohammad-Saeid Jami et al.   NEUROCHEMISTRY INTERNATIONAL

Amelioration of toxicity in neuronal models of amyotrophic lateral sclerosis by hUPF1

(2015) Sami J. Barmada et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Activation of the endoplasmic reticulum stress response in skeletal muscle of G93A*SOD1 amyotrophic lateral sclerosis mice

(2015) Dapeng Chen et al.   Frontiers in Cellular Neuroscience

Gene expression parallels synaptic excitability and plasticity changes in Alzheimer’s disease

(2015) Carlos A. Saura et al.   Frontiers in Cellular Neuroscience

Generation and expansion of highly pure motor neuron progenitors from human pluripotent stem cells

(2015) Zhong-Wei Du et al.   Nature Communications

C9ORF72 GGGGCC Expanded Repeats Produce Splicing Dysregulation which Correlates with Disease Severity in Amyotrophic Lateral Sclerosis

(2015) Johnathan Cooper-Knock et al.   PLoS One

P2Y receptors in Alzheimer's disease

(2014) Laurie Erb et al.   BIOLOGY OF THE CELL

CIIA prevents SOD1(G93A)-induced cytotoxicity by blocking ASK1-mediated signaling

(2014) Jae Keun Lee et al.   Frontiers in Cellular Neuroscience

Knocking down metabotropic glutamate receptor 1 improves survival and disease progression in the SOD1G93A mouse model of amyotrophic lateral sclerosis

(2013) Marco Milanese et al.   NEUROBIOLOGY OF DISEASE

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

(2013) Christopher J. Donnelly et al.   NEURON

Expanded GGGGCC repeat RNA associated with amyotrophic lateral sclerosis and frontotemporal dementia causes neurodegeneration

(2013) Z. Xu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Hexanucleotide Repeats in ALS/FTD Form Length-Dependent RNA Foci, Sequester RNA Binding Proteins, and Are Neurotoxic

(2013) Youn-Bok Lee et al.   Cell Reports

Decreased motor cortex  -aminobutyric acid in amyotrophic lateral sclerosis

(2012) B. R. Foerster et al.   NEUROLOGY

Apoptosis Signal-Regulating Kinase 1 Mediates MPTP Toxicity and Regulates Glial Activation

(2012) Kang-Woo Lee et al.   PLoS One

Microarray analysis of peripheral blood lymphocytes from ALS patients and the SAFE detection of the KEGG ALS pathway

(2011) Jean-Luc C Mougeot et al.   BMC Medical Genomics

Phosphatase and tensin homologue/protein kinase B pathway linked to motor neuron survival in human superoxide dismutase 1-related amyotrophic lateral sclerosis

(2011) Janine Kirby et al.   BRAIN

Signaling Events in Axons and/or Dendrites Render Motor Neurons Vulnerable to Mutant Superoxide Dismutase Toxicity

(2011) G.-B. Jeong et al.   JOURNAL OF NEUROSCIENCE

Peroxiredoxin-2 Protects against 6-Hydroxydopamine-Induced Dopaminergic Neurodegeneration via Attenuation of the Apoptosis Signal-Regulating Kinase (ASK1) Signaling Cascade

(2011) X. Hu et al.   JOURNAL OF NEUROSCIENCE

Characterizing the RNA targets and position-dependent splicing regulation by TDP-43

(2011) James R Tollervey et al.   NATURE NEUROSCIENCE

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

(2011) Mariely DeJesus-Hernandez et al.   NEURON

A Hexanucleotide Repeat Expansion in C9ORF72 Is the Cause of Chromosome 9p21-Linked ALS-FTD

(2011) Alan E. Renton et al.   NEURON

Dextromethorphan Plus Ultra Low-Dose Quinidine Reduces Pseudobulbar Affect

(2010) Erik P. Pioro et al.   ANNALS OF NEUROLOGY

A Review of the Neural Mechanisms of Action and Clinical Efficiency of Riluzole in Treating Amyotrophic Lateral Sclerosis: What have we Learned in the Last Decade?

(2010) Mark C. Bellingham   CNS Neuroscience & Therapeutics

Gene expression profiling in peripheral blood mononuclear cells from patients with sporadic amyotrophic lateral sclerosis (sALS)

(2010) Rongzhen Zhang et al.   JOURNAL OF NEUROIMMUNOLOGY

Mutations in CHMP2B in Lower Motor Neuron Predominant Amyotrophic Lateral Sclerosis (ALS)

(2010) Laura E. Cox et al.   PLoS One

Enrichment Map: A Network-Based Method for Gene-Set Enrichment Visualization and Interpretation

(2010) Daniele Merico et al.   PLoS One

Prognostic factors in ALS: A critical review

(2009) Adriano Chiò et al.   Amyotrophic Lateral Sclerosis

ALS-linked mutant SOD1 induces ER stress- and ASK1-dependent motor neuron death by targeting Derlin-1

(2008) H. Nishitoh et al.   GENES & DEVELOPMENT