Advances in Patient-Specific Induced Pluripotent Stem Cells Shed Light on Drug Discovery for Amyotrophic Lateral Sclerosis

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

(2017) Keiko Imamura et al.   Science Translational Medicine

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

(2017) Wenting Guo et al.   Nature Communications

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

(2017) Martin Madill et al.   Molecular Brain

The Effect of Fetal Bovine Serum (FBS) on Efficacy of Cellular Reprogramming for Induced Pluripotent Stem Cell (iPSC) Generation

(2016) Daekee Kwon et al.   CELL TRANSPLANTATION

Translational Cell Therapies in Regenerative Medicine and Cancers

(2016) Shinn-Zong Lin   CELL TRANSPLANTATION

Complement activation at the motor end-plates in amyotrophic lateral sclerosis

(2016) Nawal Bahia El Idrissi et al.   Journal of Neuroinflammation

Poly(GR) in C9ORF72 -Related ALS/FTD Compromises Mitochondrial Function and Increases Oxidative Stress and DNA Damage in iPSC-Derived Motor Neurons

(2016) Rodrigo Lopez-Gonzalez et al.   NEURON

Autophagic down-regulation in motor neurons remarkably prolongs the survival of ALS mice

(2016) Kuo-Wei Hsueh et al.   NEUROPHARMACOLOGY

FDA Approval Summary: Olaparib Monotherapy in Patients with Deleterious Germline BRCA-Mutated Advanced Ovarian Cancer Treated with Three or More Lines of Chemotherapy

(2015) G. Kim et al.   CLINICAL CANCER RESEARCH

The C9orf72 repeat expansion disrupts nucleocytoplasmic transport

(2015) Ke Zhang et al.   NATURE

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

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

Aggregation-prone c9FTD/ALS poly(GA) RAN-translated proteins cause neurotoxicity by inducing ER stress

(2014) Yong-Jie Zhang et al.   ACTA NEUROPATHOLOGICA

Modeling ALS with iPSCs Reveals that Mutant SOD1 Misregulates Neurofilament Balance in Motor Neurons

(2014) Hong Chen et al.   Cell Stem Cell

Clinical trials in amyotrophic lateral sclerosis: why so many negative trials and how can trials be improved?

(2014) Hiroshi Mitsumoto et al.   LANCET NEUROLOGY

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

(2014) Yves Maury et al.   NATURE BIOTECHNOLOGY

Discovery of a Biomarker and Lead Small Molecules to Target r(GGGGCC)-Associated Defects in c9FTD/ALS

(2014) Zhaoming Su et al.   NEURON

PTEN regulates AMPA receptor-mediated cell viability in iPS-derived motor neurons

(2014) D-J Yang et al.   Cell Death & Disease

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

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

Multicentre quality control evaluation of different biomarker candidates for amyotrophic lateral sclerosis

(2014) Stefan Lehnert et al.   Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration

Reduced C9orf72 gene expression in c9FTD/ALS is caused by histone trimethylation, an epigenetic event detectable in blood

(2013) Veronique V. Belzil et al.   ACTA NEUROPATHOLOGICA

Modeling key pathological features of frontotemporal dementia with C9ORF72 repeat expansion in iPSC-derived human neurons

(2013) Sandra Almeida et al.   ACTA NEUROPATHOLOGICA

A Small Molecule Screen in Stem-Cell-Derived Motor Neurons Identifies a Kinase Inhibitor as a Candidate Therapeutic for ALS

(2013) Yin M. Yang et al.   Cell Stem Cell

Transplantation of Human Adipose Tissue-Derived Stem Cells Delays Clinical Onset and Prolongs Life Span in ALS Mouse Model

(2013) Kwang S. Kim et al.   CELL TRANSPLANTATION

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

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

Synergistic Effects of GDNF and VEGF on Lifespan and Disease Progression in a Familial ALS Rat Model

(2013) Dan Krakora et al.   MOLECULAR THERAPY

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

(2013) Christopher J. Donnelly et al.   NEURON

Inhibiting PTEN Protects Hippocampal Neurons against Stretch Injury by Decreasing Membrane Translocation of AMPA Receptor GluR2 Subunit

(2013) Yuan Liu et al.   PLoS One

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 C9orf72 GGGGCC Repeat Is Translated into Aggregating Dipeptide-Repeat Proteins in FTLD/ALS

(2013) K. Mori 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

TDP-43 plasma levels are higher in amyotrophic lateral sclerosis

(2012) Esther Verstraete et al.   Amyotrophic Lateral Sclerosis

Feasibility, Safety, and Preliminary Proof of Principles of Autologous Neural Stem Cell Treatment Combined with T-Cell Vaccination for ALS Patients

(2012) G. A. Moviglia et al.   CELL TRANSPLANTATION

iPSC technology to study human aging and aging-related disorders

(2012) Guang-Hui Liu et al.   CURRENT OPINION IN CELL BIOLOGY

Modulating inflammatory monocytes with a unique microRNA gene signature ameliorates murine ALS

(2012) Oleg Butovsky et al.   JOURNAL OF CLINICAL INVESTIGATION

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

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

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

Human Neural Crest Stem Cells Derived from Human ESCs and Induced Pluripotent Stem Cells: Induction, Maintenance, and Differentiation into Functional Schwann Cells

(2012) Qiuyue Liu et al.   Stem Cells Translational Medicine

Strategies for the Development of Cell Lines for Ex Vivo Gene Therapy in the Central Nervous System

(2011) Jana Mejía-Toiber et al.   CELL TRANSPLANTATION

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

Elevated CSF TDP-43 levels in amyotrophic lateral sclerosis: Specificity, sensitivity, and a possible prognostic value

(2010) Yu-Ichi Noto et al.   Amyotrophic Lateral Sclerosis

Current hypotheses for the underlying biology of amyotrophic lateral sclerosis

(2010) Jeffrey D. Rothstein   ANNALS OF NEUROLOGY

SOD1, ANG, VAPB, TARDBP, and FUS mutations in familial amyotrophic lateral sclerosis: genotype-phenotype correlations

(2010) S. Millecamps et al.   JOURNAL OF MEDICAL GENETICS

Cytoplasmic Mislocalization of TDP-43 Is Toxic to Neurons and Enhanced by a Mutation Associated with Familial Amyotrophic Lateral Sclerosis

(2010) S. J. Barmada et al.   JOURNAL OF NEUROSCIENCE

Mutations of optineurin in amyotrophic lateral sclerosis

(2010) Hirofumi Maruyama et al.   NATURE

From transcriptome analysis to therapeutic anti-CD40L treatment in the SOD1 model of amyotrophic lateral sclerosis

(2010) John M Lincecum et al.   NATURE GENETICS

Neural differentiation of human induced pluripotent stem cells follows developmental principles but with variable potency

(2010) B.-Y. Hu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Exposure to chemicals and metals and risk of amyotrophic lateral sclerosis: A systematic review

(2009) Nadia A. Sutedja et al.   Amyotrophic Lateral Sclerosis

Autologous Bone-Marrow Mononuclear Cell Transplantation after Acute Myocardial Infarction: Comparison of Two Delivery Techniques

(2009) Suzana A. Silva et al.   CELL TRANSPLANTATION

Human embryonic stem cell-derived motor neurons expressing SOD1 mutants exhibit typical signs of motor neuron degeneration linked to ALS

(2009) S. Karumbayaram et al.   Disease Models & Mechanisms

Mutations of FUS gene in sporadic amyotrophic lateral sclerosis

(2009) L. Corrado et al.   JOURNAL OF MEDICAL GENETICS

Immunoreactivity of the phosphorylated axonal neurofilament H subunit (pNF-H) in blood of ALS model rodents and ALS patients: evaluation of blood pNF-H as a potential ALS biomarker

(2009) Kevin Boylan et al.   JOURNAL OF NEUROCHEMISTRY

Progressive aggregation despite chaperone associations of a mutant SOD1-YFP in transgenic mice that develop ALS

(2009) J. Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

TDP-43 mutant transgenic mice develop features of ALS and frontotemporal lobar degeneration

(2009) I. Wegorzewska et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Regulation of Neural Specification from Human Embryonic Stem Cells by BMP and FGF

(2009) Timothy M. LaVaute et al.   STEM CELLS

Directed Differentiation of Human-Induced Pluripotent Stem Cells Generates Active Motor Neurons

(2009) Saravanan Karumbayaram et al.   STEM CELLS

The Novel Antioxidant Edaravone: From Bench to Bedside

(2008) Toshiaki Watanabe et al.   Cardiovascular Therapeutics

Human Embryonic Stem Cell-Derived Motor Neurons Are Sensitive to the Toxic Effect of Glial Cells Carrying an ALS-Causing Mutation

(2008) Francesco Paolo Di Giorgio et al.   Cell Stem Cell

Riluzole blocks persistent Na+and Ca2+currents and modulates release of glutamate via presynaptic NMDA receptors on neonatal rat hypoglossal motoneuronsin vitro

(2008) Nerijus Lamanauskas et al.   EUROPEAN JOURNAL OF NEUROSCIENCE

Treatment with edaravone, initiated at symptom onset, slows motor decline and decreases SOD1 deposition in ALS mice

(2008) Hidefumi Ito et al.   EXPERIMENTAL NEUROLOGY

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

(2008) J. T. Dimos et al.   SCIENCE

Directed Differentiation of Ventral Spinal Progenitors and Motor Neurons from Human Embryonic Stem Cells by Small Molecules

(2008) Xue-Jun Li et al.   STEM CELLS