Human Pluripotent Stem-Cell-Derived Models as a Missing Link in Drug Discovery and Development

Clinical Trial in a Dish

(2021) Chi Keung Lam et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

Generation of systemic lupus erythematosus patient-derived induced pluripotent stem cells from blood

(2021) Weilong Li et al.   STEM CELLS AND DEVELOPMENT

A logical network-based drug-screening platform for Alzheimer’s disease representing pathological features of human brain organoids

(2021) Jong-Chan Park et al.   Nature Communications

Establishment of an in vitro safety assessment model for lipid-lowering drugs using same-origin human pluripotent stem cell-derived cardiomyocytes and endothelial cells

(2021) Xuan Ni et al.   ACTA PHARMACOLOGICA SINICA

A human stem cell-derived test system for agents modifying neuronal N-methyl-d-aspartate-type glutamate receptor Ca2+-signalling

(2021) Stefanie Klima et al.   ARCHIVES OF TOXICOLOGY

Characterization of mitochondrial health from human peripheral blood mononuclear cells to cerebral organoids derived from induced pluripotent stem cells

(2021) Angela Duong et al.   Scientific Reports

Addressing variability in iPSC-derived models of human disease: guidelines to promote reproducibility

(2020) Viola Volpato et al.   Disease Models & Mechanisms

Transcriptome of iPSC-derived neuronal cells reveals a module of co-expressed genes consistently associated with autism spectrum disorder

(2020) K. Griesi-Oliveira et al.   MOLECULAR PSYCHIATRY

Dissecting transcriptomic signatures of neuronal differentiation and maturation using iPSCs

(2020) Emily E. Burke et al.   Nature Communications

Single-Cell Transcriptomic Comparison of Human Fetal Retina, hPSC-Derived Retinal Organoids, and Long-Term Retinal Cultures

(2020) Akshayalakshmi Sridhar et al.   Cell Reports

The Tumor-Suppressive Human Circular RNA CircITCH Sponges miR-330-5p to Ameliorate Doxorubicin-Induced Cardiotoxicity Through Upregulating SIRT6, Survivin, and SERCA2a

(2020) Dong Han et al.   CIRCULATION RESEARCH

RNA-based CRISPR-Mediated Loss-of-Function Mutagenesis in Human Pluripotent Stem Cells

(2020) Alan W. Leung et al.   JOURNAL OF MOLECULAR BIOLOGY

Clinical trial in a dish using iPSCs shows lovastatin improves endothelial dysfunction and cellular cross-talk in LMNA cardiomyopathy

(2020) Nazish Sayed et al.   Science Translational Medicine

Modeling and Rescue of RP2 Retinitis Pigmentosa Using iPSC-Derived Retinal Organoids

(2020) Amelia Lane et al.   Stem Cell Reports

Human Pluripotent Stem Cell-Derived Neural Cells as a Relevant Platform for Drug Screening in Alzheimer’s Disease

(2020) Juan Antonio Garcia-Leon et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Complement modulation reverses pathology in Y402H ‐retinal pigment epithelium cell model of age‐related macular degeneration by restoring lysosomal function

(2020) Edvinas Cerniauskas et al.   Stem Cells Translational Medicine

Generation of human liver organoids from pluripotent stem cell-derived hepatic endoderms

(2020) Kasem Kulkeaw et al.   PeerJ

Humanized neurofibroma model from induced pluripotent stem cells delineates tumor pathogenesis and developmental origins

(2020) Juan Mo et al.   JOURNAL OF CLINICAL INVESTIGATION

Network-based screen in iPSC-derived cells reveals therapeutic candidate for heart valve disease

(2020) Christina V. Theodoris et al.   SCIENCE

Human Motor Neurons With SOD1-G93A Mutation Generated From CRISPR/Cas9 Gene-Edited iPSCs Develop Pathological Features of Amyotrophic Lateral Sclerosis

(2020) Byung Woo Kim et al.   Frontiers in Cellular Neuroscience

Causes and Patterns of Dementia: An Update in the Era of Redefining Alzheimer's Disease

(2019) Bryan D. James et al.   Annual Review of Public Health

Induced pluripotent stem cells in disease modelling and drug discovery

(2019) R. Grant Rowe et al.   NATURE REVIEWS GENETICS

Genetic meta-analysis of diagnosed Alzheimer’s disease identifies new risk loci and implicates Aβ, tau, immunity and lipid processing

(2019) Brian W. Kunkle et al.   NATURE GENETICS

Human pluripotent stem cell–derived models and drug screening in CNS precision medicine

(2019) M. Catarina Silva et al.   Annals of the New York Academy of Sciences

Les organoïdes rénaux

(2019) Clara Steichen et al.   M S-MEDECINE SCIENCES

Oxidative Stress: A Key Modulator in Neurodegenerative Diseases

(2019) Anju Singh et al.   MOLECULES

Current Challenges of iPSC-Based Disease Modeling and Therapeutic Implications

(2019) Michael Xavier Doss et al.   Cells

Clinical trial in a dish: personalized stem cell derived cardiomyocyte assay compared to clinical trial results for two QT prolonging drugs

(2019) Ksenia Blinova et al.   CTS-Clinical and Translational Science

Use of human pluripotent stem cell-derived cells for neurodegenerative disease modeling and drug screening platform

(2019) Juan Antonio Garcia-Leon et al.   Future Medicinal Chemistry

Organoids are promising tools for species‐specific in vitro toxicological studies

(2019) Justyna Augustyniak et al.   JOURNAL OF APPLIED TOXICOLOGY

Generation of expandable human pluripotent stem cell-derived hepatocyte-like liver organoids

(2019) Seon Ju Mun et al.   JOURNAL OF HEPATOLOGY

Novel paradigms in systemic lupus erythematosus

(2019) Thomas Dörner et al.   LANCET

Individual brain organoids reproducibly form cell diversity of the human cerebral cortex

(2019) Silvia Velasco et al.   NATURE

Modeling of Fibrotic Lung Disease Using 3D Organoids Derived from Human Pluripotent Stem Cells

(2019) Alexandros Strikoudis et al.   Cell Reports

Enhanced structural maturation of human induced pluripotent stem cell-derived cardiomyocytes under a controlled microenvironment in a microfluidic system

(2019) Tomasz Jan Kolanowski et al.   Acta Biomaterialia

Human ESC-derived expandable hepatic organoids enable therapeutic liver repopulation and pathophysiological modeling of alcoholic liver injury

(2019) Shuyong Wang et al.   CELL RESEARCH

Use of Zebrafish in Drug Discovery Toxicology

(2019) Steven Cassar et al.   CHEMICAL RESEARCH IN TOXICOLOGY

Use of Family History and Genetic Testing to Determine Risk of Colorectal Cancer

(2019) Fay Kastrinos et al.   GASTROENTEROLOGY

Population-based toxicity screening in human induced pluripotent stem cell-derived cardiomyocytes

(2019) Sarah D. Burnett et al.   TOXICOLOGY AND APPLIED PHARMACOLOGY

Robust, Long-Term Culture of Endoderm-Derived Hepatic Organoids for Disease Modeling

(2019) Soheil Akbari et al.   Stem Cell Reports

Patient and Disease–Specific Induced Pluripotent Stem Cells for Discovery of Personalized Cardiovascular Drugs and Therapeutics

(2019) David T. Paik et al.   PHARMACOLOGICAL REVIEWS

Drug Discovery Platform Targeting M. tuberculosis with Human Embryonic Stem Cell-Derived Macrophages

(2019) Hyo-Won Han et al.   Stem Cell Reports

Generation of a human induced pluripotent stem cell–based model for tauopathies combining three microtubule-associated protein tau mutations which displays several phenotypes linked to neurodegeneration

(2018) Juan Antonio García-León et al.   Alzheimers & Dementia

Bioengineering an electro-mechanically functional miniature ventricular heart chamber from human pluripotent stem cells

(2018) Ronald A. Li et al.   BIOMATERIALS

Next generation organoids for biomedical research and applications

(2018) Yan-Ru Lou et al.   BIOTECHNOLOGY ADVANCES

High-throughput screen for compounds that modulate neurite growth of human induced pluripotent stem cell-derived neurons

(2018) Sean P. Sherman et al.   Disease Models & Mechanisms

Inflammation in CNS neurodegenerative diseases

(2018) Jodie Stephenson et al.   IMMUNOLOGY

Induced Pluripotent Stem Cell-Derived Hepatocytes and Precision Medicine in Human Liver Disease

(2018) Sakil Kulkarni et al.   JOURNAL OF PEDIATRIC GASTROENTEROLOGY AND NUTRITION

Transplantation of Human Embryonic Stem Cell–Derived Cardiovascular Progenitors for Severe Ischemic Left Ventricular Dysfunction

(2018) Philippe Menasché et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

How human embryonic stem cells sparked a revolution

(2018) David Cyranoski   NATURE

Precision pharmacology for Alzheimer’s disease

(2018) Harald Hampel et al.   PHARMACOLOGICAL RESEARCH

Neurodegenerative Erkrankungen

(2018) Wolfgang Reith   RADIOLOGE

A big data approach to the concordance of the toxicity of pharmaceuticals in animals and humans

(2018) Matthew Clark et al.   REGULATORY TOXICOLOGY AND PHARMACOLOGY

Concise Review: Human Induced Pluripotent Stem Cell Models of Retinitis Pigmentosa

(2018) Ana Artero Castro et al.   STEM CELLS

Open Science Meets Stem Cells: A New Drug Discovery Approach for Neurodegenerative Disorders

(2018) Chanshuai Han et al.   Frontiers in Neuroscience

Structural Immaturity of Human iPSC-Derived Cardiomyocytes: In Silico Investigation of Effects on Function and Disease Modeling

(2018) Jussi T. Koivumäki et al.   Frontiers in Physiology

Engineered Kidney Tubules for Modeling Patient-Specific Diseases and Drug Discovery

(2018) Valentina Benedetti et al.   EBioMedicine

Clinical Trials in a Dish: A Perspective on the Coming Revolution in Drug Development

(2018) Bernard Fermini et al.   SLAS Discovery

High-Content Assay Multiplexing for Muscle Toxicity Screening in Human-Induced Pluripotent Stem Cell-Derived Skeletal Myoblasts

(2018) William D. Klaren et al.   ASSAY AND DRUG DEVELOPMENT TECHNOLOGIES

Animal models of neurodegenerative diseases

(2018) Ted M. Dawson et al.   NATURE NEUROSCIENCE

Kidney organoids—a new tool for kidney therapeutic development

(2018) Joseph V. Bonventre   KIDNEY INTERNATIONAL

Global, regional, and national burden of Alzheimer's disease and other dementias, 1990–2016: a systematic analysis for the Global Burden of Disease Study 2016

(2018) Emma Nichols et al.   LANCET NEUROLOGY

Revealing the brain's molecular architecture

(2018)   SCIENCE

What is precision medicine?

(2017) Inke R. König et al.   EUROPEAN RESPIRATORY JOURNAL

The SH-SY5Y cell line in Parkinson’s disease research: a systematic review

(2017) Helena Xicoy et al.   Molecular Neurodegeneration

Amyloid-β and tau complexity — towards improved biomarkers and targeted therapies

(2017) Juan Carlos Polanco et al.   Nature Reviews Neurology

The use of brain organoids to investigate neural development and disease

(2017) Elizabeth Di Lullo et al.   NATURE REVIEWS NEUROSCIENCE

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

(2017) Keiko Imamura et al.   Science Translational Medicine

High-throughput screening of tyrosine kinase inhibitor cardiotoxicity with human induced pluripotent stem cells

(2017) Arun Sharma et al.   Science Translational Medicine

Cell Expansion During Directed Differentiation of Stem Cells Toward the Hepatic Lineage

(2017) Ravali Raju et al.   STEM CELLS AND DEVELOPMENT

In vitro cardiotoxicity assessment of environmental chemicals using an organotypic human induced pluripotent stem cell-derived model

(2017) Oksana Sirenko et al.   TOXICOLOGY AND APPLIED PHARMACOLOGY

Pathology of Neurodegenerative Diseases

(2017) Brittany N. Dugger et al.   Cold Spring Harbor Perspectives in Biology

Modeling susceptibility to drug-induced long QT with a panel of subject-specific induced pluripotent stem cells

(2017) Francesca Stillitano et al.   eLife

An Automated Platform for Assessment of Congenital and Drug-Induced Arrhythmia with hiPSC-Derived Cardiomyocytes

(2017) Wesley L. McKeithan et al.   Frontiers in Physiology

Recapitulation of Clinical Individual Susceptibility to Drug-Induced QT Prolongation in Healthy Subjects Using iPSC-Derived Cardiomyocytes

(2017) Tadahiro Shinozawa et al.   Stem Cell Reports

Directed differentiation of human pluripotent stem cells to blood-brain barrier endothelial cells

(2017) Tongcheng Qian et al.   Science Advances

A new era of disease modeling and drug discovery using induced pluripotent stem cells

(2016) Wonhee Suh   ARCHIVES OF PHARMACAL RESEARCH

Effect of Oxidative Damage on the Stability and Dimerization of Superoxide Dismutase 1

(2016) Drazen Petrov et al.   BIOPHYSICAL JOURNAL

Post-marketing withdrawal of 462 medicinal products because of adverse drug reactions: a systematic review of the world literature

(2016) Igho J. Onakpoya et al.   BMC Medicine

Cell sources for in vitro human liver cell culture models

(2016) Katrin Zeilinger et al.   EXPERIMENTAL BIOLOGY AND MEDICINE

Next-generation phenotypic screening

(2016) Scott J Warchal et al.   Future Medicinal Chemistry

TheC. elegansmodel in toxicity testing

(2016) Piper Reid Hunt   JOURNAL OF APPLIED TOXICOLOGY

New insights into the immunopathogenesis of systemic lupus erythematosus

(2016) George C. Tsokos et al.   Nature Reviews Rheumatology

From the Cover: High-Throughput Imaging of Cardiac Microtissues for the Assessment of Cardiac Contraction during Drug Discovery

(2016) Amy Pointon et al.   TOXICOLOGICAL SCIENCES

Limitations in Translating Animal Studies to Humans in Cardiovascular Disease

(2016) Sean Lal et al.   Journal of Cardiovascular Translational Research

Are Stem Cell-Based Therapies for Parkinson’s Disease Ready for the Clinic in 2016?

(2016) Roger A. Barker et al.   Journal of Parkinsons Disease

Systemic lupus erythematosus

(2016) Arvind Kaul et al.   Nature Reviews Disease Primers

Fyn Kinase regulates GluN2B subunit-dominant NMDA receptors in human induced pluripotent stem cell-derived neurons

(2016) Wen-Bo Zhang et al.   Scientific Reports

Biallelic Mutations in the Autophagy Regulator DRAM2 Cause Retinal Dystrophy with Early Macular Involvement

(2015) Mohammed E. El-Asrag et al.   AMERICAN JOURNAL OF HUMAN GENETICS

High-Content Assay Multiplexing for Toxicity Screening in Induced Pluripotent Stem Cell-Derived Cardiomyocytes and Hepatocytes

(2015) Fabian Alexander Grimm et al.   ASSAY AND DRUG DEVELOPMENT TECHNOLOGIES

Alzheimer’s Disease Risk Genes and Mechanisms of Disease Pathogenesis

(2015) Celeste M. Karch et al.   BIOLOGICAL PSYCHIATRY

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

From Dish to Bedside: Lessons Learned While Translating Findings from a Stem Cell Model of Disease to a Clinical Trial

(2015) John McNeish et al.   Cell Stem Cell

Human Induced Pluripotent Stem Cell-Derived Cardiomyocytes: Insights Into Molecular, Cellular, and Functional Phenotypes

(2015) I. Karakikes et al.   CIRCULATION RESEARCH

Gene expression of OCT4, SOX2, KLF4 and MYC (OSKM) induced pluripotent stem cells: identification for potential mechanisms

(2015) Yanning Cai et al.   Diagnostic Pathology

A New Mouse Model of Limb-Girdle Muscular Dystrophy Type 2I Homozygous for the Common L276I Mutation Mimicking the Mild Phenotype in Humans

(2015) Thomas O. Krag et al.   JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY

Mitigating risk in academic preclinical drug discovery

(2015) Jayme L. Dahlin et al.   NATURE REVIEWS DRUG DISCOVERY

Precision Medicine — Personalized, Problematic, and Promising

(2015) J. Larry Jameson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Miniature Swine Breeds in Toxicology and Drug Safety Assessments

(2015) Alain Stricker-Krongrad et al.   TOXICOLOGIC PATHOLOGY

Prediction of drug-induced nephrotoxicity and injury mechanisms with human induced pluripotent stem cell-derived cells and machine learning methods

(2015) Karthikeyan Kandasamy et al.   Scientific Reports

Generation of Self-Renewing Hepatoblasts From Human Embryonic Stem Cells by Chemical Approaches

(2015) Muzi Zhang et al.   Stem Cells Translational Medicine

Dysregulated autophagy in the RPE is associated with increased susceptibility to oxidative stress and AMD

(2014) Sayak K Mitter et al.   Autophagy

Lamin A/C mutations in dilated cardiomyopathy

(2014) Frédérique Tesson et al.   Cardiology Journal

Ranibizumab and pegaptanib for the treatment of age-related macular degeneration: a systematic review and economic evaluation

(2014) J Colquitt et al.   HEALTH TECHNOLOGY ASSESSMENT

Induction of early neural precursors and derivation of tripotent neural stem cells from human pluripotent stem cells under xeno-free conditions

(2014) Hal X. Nguyen et al.   JOURNAL OF COMPARATIVE NEUROLOGY

Genome Editing of Isogenic Human Induced Pluripotent Stem Cells Recapitulates Long QT Phenotype for Drug Testing

(2014) Yongming Wang et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Generation of cerebral organoids from human pluripotent stem cells

(2014) Madeline A Lancaster et al.   Nature Protocols

The new frontier of genome engineering with CRISPR-Cas9

(2014) J. A. Doudna et al.   SCIENCE

A retinoic acid-enhanced, multicellular human blood-brain barrier model derived from stem cell sources

(2014) Ethan S. Lippmann et al.   Scientific Reports

Lipodystrophy-Linked LMNA p.R482W Mutation Induces Clinical Early Atherosclerosis and In Vitro Endothelial Dysfunction

(2013) Guillaume Bidault et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

High-Content Assays for Hepatotoxicity Using Induced Pluripotent Stem Cell–Derived Cells

(2013) Oksana Sirenko et al.   ASSAY AND DRUG DEVELOPMENT TECHNOLOGIES

The why and how of phenotypic small-molecule screens

(2013) Ulrike S Eggert   Nature Chemical Biology

Axonal transport deficits and neurodegenerative diseases

(2013) Stéphanie Millecamps et al.   NATURE REVIEWS NEUROSCIENCE

A fully defined and scalable 3D culture system for human pluripotent stem cell expansion and differentiation

(2013) Y. Lei et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

The determination and interpretation of the therapeutic index in drug development

(2012) Patrick Y. Muller et al.   NATURE REVIEWS DRUG DISCOVERY

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

(2012) M. Jinek et al.   SCIENCE

The challenge of new drug discovery for tuberculosis

(2011) Anil Koul et al.   NATURE

How were new medicines discovered?

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

TAL Effectors: Customizable Proteins for DNA Targeting

(2011) A. J. Bogdanove et al.   SCIENCE

Trends in Risks Associated With New Drug Development: Success Rates for Investigational Drugs

(2010) J A DiMasi et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Causes of blind and partial sight certifications in England and Wales: April 2007–March 2008

(2010) C Bunce et al.   EYE

LQTS gene LOVD database

(2010) Tao Zhang et al.   HUMAN MUTATION

Network medicine: a network-based approach to human disease

(2010) Albert-László Barabási et al.   NATURE REVIEWS GENETICS

Is Amyotrophic Lateral Sclerosis a Mitochondrial Channelopathy?

(2010) Virginia Le Verche et al.   NEURON

Alzheimer's Disease

(2010) Henry W. Querfurth et al.   NEW ENGLAND JOURNAL OF MEDICINE

Toxicogenetics: population-based testing of drug and chemical safety in mouse models

(2010) Ivan Rusyn et al.   PHARMACOGENOMICS

Cost-of-illness studies in systemic lupus erythematosus: A systematic review

(2010) Tracy Y. Zhu et al.   ARTHRITIS CARE & RESEARCH

Highly efficient neural conversion of human ES and iPS cells by dual inhibition of SMAD signaling

(2009) Stuart M Chambers et al.   NATURE BIOTECHNOLOGY

Autophagy and Exosomes in the Aged Retinal Pigment Epithelium: Possible Relevance to Drusen Formation and Age-Related Macular Degeneration

(2009) Ai Ling Wang et al.   PLoS One

Breaking the Code of DNA Binding Specificity of TAL-Type III Effectors

(2009) J. Boch et al.   SCIENCE

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

(2009) Saravanan Karumbayaram et al.   STEM CELLS

The pilocarpine model of temporal lobe epilepsy

(2008) Giulia Curia et al.   JOURNAL OF NEUROSCIENCE METHODS