Combating neurodegenerative disease with chemical probes and model systems

A Quantitative Chaperone Interaction Network Reveals the Architecture of Cellular Protein Homeostasis Pathways

(2014) Mikko Taipale et al.   CELL

The Reprogramming of Tumor Stroma by HSF1 Is a Potent Enabler of Malignancy

(2014) Ruth Scherz-Shouval et al.   CELL

Potentiated Hsp104 Variants Antagonize Diverse Proteotoxic Misfolding Events

(2014) Meredith E. Jackrel et al.   CELL

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

(2014) Evangelos Kiskinis et al.   Cell Stem Cell

Sorting out the trash: the spatial nature of eukaryotic protein quality control

(2014) Emily M Sontag et al.   CURRENT OPINION IN CELL BIOLOGY

Caenorhabditis elegans as a model system for studying non-cell-autonomous mechanisms in protein-misfolding diseases

(2014) C. I. Nussbaum-Krammer et al.   Disease Models & Mechanisms

Mechanisms of protein-folding diseases at a glance

(2014) J. S. Valastyan et al.   Disease Models & Mechanisms

Aneuploidy: implications for protein homeostasis and disease

(2014) A. B. Oromendia et al.   Disease Models & Mechanisms

iPS cells: a game changer for future medicine

(2014) H. Inoue et al.   EMBO JOURNAL

Autophagy induction enhances TDP43 turnover and survival in neuronal ALS models

(2014) Sami J Barmada et al.   Nature Chemical Biology

Pharmacological chaperones stabilize retromer to limit APP processing

(2014) Vincent J Mecozzi et al.   Nature Chemical Biology

PERK mediates eIF2α phosphorylation responsible for BACE1 elevation, CREB dysfunction and neurodegeneration in a mouse model of Alzheimer's disease

(2014) Latha Devi et al.   NEUROBIOLOGY OF AGING

DNA Damage and Its Links to Neurodegeneration

(2014) Ram Madabhushi et al.   NEURON

Targeting the Intrinsically Disordered Structural Ensemble of α-Synuclein by Small Molecules as a Potential Therapeutic Strategy for Parkinson’s Disease

(2014) Gergely Tóth et al.   PLoS One

Neuropathologic Changes of Multiple System Atrophy and Diffuse Lewy Body Disease

(2014) Etty Ramirez et al.   SEMINARS IN NEUROLOGY

Genetic and Chemical Correction of Cholesterol Accumulation and Impaired Autophagy in Hepatic and Neural Cells Derived from Niemann-Pick Type C Patient-Specific iPS Cells

(2014) Dorothea Maetzel et al.   Stem Cell Reports

The therapeutics of Alzheimer's disease: Where we stand and where we are heading

(2013) Dennis J. Selkoe   ANNALS OF NEUROLOGY

Regulation of autophagy by mTOR-dependent and mTOR-independent pathways: autophagy dysfunction in neurodegenerative diseases and therapeutic application of autophagy enhancers

(2013) Sovan Sarkar   BIOCHEMICAL SOCIETY TRANSACTIONS

XBP-1 Is a Cell-Nonautonomous Regulator of Stress Resistance and Longevity

(2013) Rebecca C. Taylor et al.   CELL

Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity

(2013) F. Ann Ran et al.   CELL

Regulation of Organismal Proteostasis by Transcellular Chaperone Signaling

(2013) Patricija van Oosten-Hawle et al.   CELL

PolyQ Proteins Interfere with Nuclear Degradation of Cytosolic Proteins by Sequestering the Sis1p Chaperone

(2013) Sae-Hun Park et al.   CELL

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

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

Circadian clocks and neurodegenerative diseases: time to aggregate?

(2013) Michael H Hastings et al.   CURRENT OPINION IN NEUROBIOLOGY

Enhanced Aggregation of Androgen Receptor in Induced Pluripotent Stem Cell-derived Neurons from Spinal and Bulbar Muscular Atrophy

(2013) Yoshihiro Nihei et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

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

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

VX-809 corrects folding defects in cystic fibrosis transmembrane conductance regulator protein through action on membrane-spanning domain 1

(2013) Hong Yu Ren et al.   MOLECULAR BIOLOGY OF THE CELL

Therapeutic modulation of eIF2α phosphorylation rescues TDP-43 toxicity in amyotrophic lateral sclerosis disease models

(2013) Hyung-Jun Kim et al.   NATURE GENETICS

Physiologic brain activity causes DNA double-strand breaks in neurons, with exacerbation by amyloid-β

(2013) Elsa Suberbielle et al.   NATURE NEUROSCIENCE

Interaction of FUS and HDAC1 regulates DNA damage response and repair in neurons

(2013) Wen-Yuan Wang et al.   NATURE NEUROSCIENCE

Opportunities and challenges of pluripotent stem cell neurodegenerative disease models

(2013) Jackson Sandoe et al.   NATURE NEUROSCIENCE

Pharmacological reduction of ER stress protects against TDP-43 neuronal toxicity in vivo

(2013) Alexandra Vaccaro et al.   NEUROBIOLOGY OF DISEASE

Direct conversion of patient fibroblasts demonstrates non-cell autonomous toxicity of astrocytes to motor neurons in familial and sporadic ALS

(2013) K. Meyer et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Identification and Rescue of  -Synuclein Toxicity in Parkinson Patient-Derived Neurons

(2013) C. Y. Chung et al.   SCIENCE

Yeast Reveal a "Druggable" Rsp5/Nedd4 Network that Ameliorates  -Synuclein Toxicity in Neurons

(2013) D. F. Tardiff et al.   SCIENCE

Genetic Screens in Human Cells Using the CRISPR-Cas9 System

(2013) Tim Wang et al.   SCIENCE

Oral Treatment Targeting the Unfolded Protein Response Prevents Neurodegeneration and Clinical Disease in Prion-Infected Mice

(2013) J. A. Moreno et al.   Science Translational Medicine

A Functional Variomics Tool for Discovering Drug-Resistance Genes and Drug Targets

(2013) Zhiwei Huang et al.   Cell Reports

SMN Is Required for Sensory-Motor Circuit Function in Drosophila

(2012) Wendy L. Imlach et al.   CELL

A TALEN Genome-Editing System for Generating Human Stem Cell-Based Disease Models

(2012) Qiurong Ding et al.   Cell Stem Cell

Inhibition of tau aggregation in a novel Caenorhabditis elegans model of tauopathy mitigates proteotoxicity

(2012) Chronis Fatouros et al.   HUMAN MOLECULAR GENETICS

Chemical screen reveals small molecules suppressing fragile X premutation rCGG repeat-mediated neurodegeneration in Drosophila

(2012) A. Qurashi et al.   HUMAN MOLECULAR GENETICS

Kinase inhibitors arrest neurodegeneration in cell and C. elegans models of LRRK2 toxicity

(2012) C. Yao et al.   HUMAN MOLECULAR GENETICS

RNA-mediated toxicity in neurodegenerative disease

(2012) Veronique V. Belzil et al.   MOLECULAR AND CELLULAR NEUROSCIENCE

Activation of Hsp70 reduces neurotoxicity by promoting polyglutamine protein degradation

(2012) Adrienne M Wang et al.   Nature Chemical Biology

Methylene Blue Protects against TDP-43 and FUS Neuronal Toxicity in C. elegans and D. rerio

(2012) Alexandra Vaccaro et al.   PLoS One

Human Genetic Variation, Shared and Private

(2012) F. Casals et al.   SCIENCE

Pathological  -Synuclein Transmission Initiates Parkinson-like Neurodegeneration in Nontransgenic Mice

(2012) K. C. Luk et al.   SCIENCE

FoldEco: A Model for Proteostasis in E. coli

(2012) Evan T. Powers et al.   Cell Reports

The Cell-Non-Autonomous Nature of Electron Transport Chain-Mediated Longevity

(2011) Jenni Durieux et al.   CELL

Inhibitors of LRRK2 kinase attenuate neurodegeneration and Parkinson-like phenotypes in Caenorhabditis elegans and Drosophila Parkinson's disease models

(2011) Zhaohui Liu et al.   HUMAN MOLECULAR GENETICS

Protein localization in disease and therapy

(2011) M.-C. Hung et al.   JOURNAL OF CELL SCIENCE

Small-molecule proteostasis regulators for protein conformational diseases

(2011) Barbara Calamini et al.   Nature Chemical Biology

How were new medicines discovered?

(2011) David C. Swinney et al.   NATURE REVIEWS DRUG DISCOVERY

Proteome Half-Life Dynamics in Living Human Cells

(2011) E. Eden et al.   SCIENCE

Chemical and Biological Approaches for Adapting Proteostasis to Ameliorate Protein Misfolding and Aggregation Diseases-Progress and Prognosis

(2011) S. L. Lindquist et al.   Cold Spring Harbor Perspectives in Biology

Aging as an Event of Proteostasis Collapse

(2011) R. C. Taylor et al.   Cold Spring Harbor Perspectives in Biology

Ataxin-2 intermediate-length polyglutamine expansions are associated with increased risk for ALS

(2010) Andrew C. Elden et al.   NATURE

Inhibitors of protein disulfide isomerase suppress apoptosis induced by misfolded proteins

(2010) Benjamin G Hoffstrom et al.   Nature Chemical Biology

Prion-like transmission of protein aggregates in neurodegenerative diseases

(2010) Patrik Brundin et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

The secretases: enzymes with therapeutic potential in Alzheimer disease

(2010) Bart De Strooper et al.   Nature Reviews Neurology

Modelling neurodegeneration in Saccharomyces cerevisiae: why cook with baker's yeast?

(2010) Vikram Khurana et al.   NATURE REVIEWS NEUROSCIENCE

Large-scale in vivo femtosecond laser neurosurgery screen reveals small-molecule enhancer of regeneration

(2010) C. Samara et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Evolutionary Conservation of Vertebrate Blood-Brain Barrier Chemoprotective Mechanisms in Drosophila

(2009) F. Mayer et al.   JOURNAL OF NEUROSCIENCE

α-Synuclein is part of a diverse and highly conserved interaction network that includes PARK9 and manganese toxicity

(2009) Aaron D Gitler et al.   NATURE GENETICS

Collapse of proteostasis represents an early molecular event in Caenorhabditis elegans aging

(2009) A. Ben-Zvi et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Hsp90 Inhibition Suppresses Mutant EGFR-T790M Signaling and Overcomes Kinase Inhibitor Resistance

(2008) T. Shimamura et al.   CANCER RESEARCH

Mistranslation-Induced Protein Misfolding as a Dominant Constraint on Coding-Sequence Evolution

(2008) D. Allan Drummond et al.   CELL

Impaired ERAD and ER stress are early and specific events in polyglutamine toxicity

(2008) M. L. Duennwald et al.   GENES & DEVELOPMENT

Autophagy fights disease through cellular self-digestion

(2008) Noboru Mizushima et al.   NATURE

Identification of small molecules rescuing fragile X syndrome phenotypes in Drosophila

(2008) Shuang Chang et al.   Nature Chemical Biology

Automated on-chip rapid microscopy, phenotyping and sorting of C. elegans

(2008) Kwanghun Chung et al.   NATURE METHODS

Deregulation of sphingolipid metabolism in Alzheimer's disease

(2008) Xingxuan He et al.   NEUROBIOLOGY OF AGING

Genetic and Chemical Modifiers of a CUG Toxicity Model in Drosophila

(2008) Amparo Garcia-Lopez et al.   PLoS One

Adapting Proteostasis for Disease Intervention

(2008) William E. Balch et al.   SCIENCE