Zebrafish disease models in drug discovery: from preclinical modelling to clinical trials

Melanoma models for the next generation of therapies

(2021) E. Elizabeth Patton et al.   CANCER CELL

Advancing RAS/RASopathy therapies: An NCI‐sponsored intramural and extramural collaboration for the study of RASopathies

(2020) Andrea M. Gross et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A

Discovery of Acylsulfonohydrazide-Derived Lysine Acetyltransferase (KAT6A) Inhibitors as Potent Senescence-Inducing Anti-Cancer Agents

(2020) Daniel L. Priebbenow et al.   JOURNAL OF MEDICINAL CHEMISTRY

Allosteric Activators of Protein Phosphatase 2A Display Broad Antitumor Activity Mediated by Dephosphorylation of MYBL2

(2020) Ken Morita et al.   CELL

Evaluating drug targets through human loss-of-function genetic variation

(2020) Eric Vallabh Minikel et al.   NATURE

RNA helicase DDX21 mediates nucleotide stress responses in neural crest and melanoma cells

(2020) Cristina Santoriello et al.   NATURE CELL BIOLOGY

Zebrafish B cell acute lymphoblastic leukemia: new findings in an old model

(2020) Gilseung Park et al.   Oncotarget

Zebrafish Microenvironment Elevates EMT and CSC-Like Phenotype of Engrafted Prostate Cancer Cells

(2020) Lanpeng Chen et al.   Cells

From Tank to Treatment: Modeling Melanoma in Zebrafish

(2020) William Tyler Frantz et al.   Cells

PRL3-DDX21 Transcriptional Control of Endolysosomal Genes Restricts Melanocyte Stem Cell Differentiation

(2020) Jeanette A. Johansson et al.   DEVELOPMENTAL CELL

The identification of dual protective agents against cisplatin-induced oto-and nephrotoxicity using the zebrafish model

(2020) Jaime N Wertman et al.   eLife

Zebrafish Xenografts Unveil Sensitivity to Olaparib beyond BRCA Status

(2020) Ana Beatriz Varanda et al.   Cancers

Zebrafish xenografts as a fast screening platform for bevacizumab cancer therapy

(2020) Cátia Rebelo de Almeida et al.   Communications Biology

Cercosporamide inhibits bone morphogenetic protein receptor type I kinase activity in zebrafish

(2020) Jelmer Hoeksma et al.   Disease Models & Mechanisms

The Stress-Like Cancer Cell State Is a Consistent Component of Tumorigenesis

(2020) Maayan Baron et al.   Cell Systems

Deciphering melanoma cell states and plasticity with zebrafish models

(2020) Jana Travnickova et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

Water Waves to Sound Waves: Using Zebrafish to Explore Hair Cell Biology

(2019) Sarah B. Pickett et al.   JARO-JOURNAL OF THE ASSOCIATION FOR RESEARCH IN OTOLARYNGOLOGY

High-Throughput Video Processing of Heart Rate Responses in Multiple Wild-type Embryonic Zebrafish per Imaging Field

(2019) W. Kyle Martin et al.   Scientific Reports

Zebrafish as an Emerging Model for Osteoporosis: A Primary Testing Platform for Screening New Osteo-Active Compounds

(2019) Dylan J. M. Bergen et al.   Frontiers in Endocrinology

Impact of post-hatching maturation on the pharmacokinetics of paracetamol in zebrafish larvae

(2019) Rob C. van Wijk et al.   Scientific Reports

Screening drugs for myocardial disease in vivo with zebrafish: an expert update

(2019) Yanbin Zhao et al.   Expert Opinion on Drug Discovery

Dissecting metabolism using zebrafish models of disease

(2019) Talhah M. Salmi et al.   BIOCHEMICAL SOCIETY TRANSACTIONS

Germline-Activating RRAS2 Mutations Cause Noonan Syndrome

(2019) Tetsuya Niihori et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Patterns of substrate affinity, competition and degradation kinetics underlie biological activity of thalidomide analogs

(2019) Adam S. Sperling et al.   BLOOD

Visualizing Engrafted Human Cancer and Therapy Responses in Immunodeficient Zebrafish

(2019) Chuan Yan et al.   CELL

Whole organism small molecule screen identifies novel regulators of pancreatic endocrine development

(2019) Christian S. M. Helker et al.   DEVELOPMENT

Automated in vivo screen in zebrafish identifies Clotrimazole as targeting a metabolic vulnerability in a melanoma model

(2019) Francesca Precazzini et al.   DEVELOPMENTAL BIOLOGY

Modeling Cancer with Flies and Fish

(2019) Ross L. Cagan et al.   DEVELOPMENTAL CELL

Mechanistic and quantitative understanding of pharmacokinetics in zebrafish larvae through nanoscale blood sampling and metabolite modelling of paracetamol

(2019) Rob C van Wijk et al.   JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS

ARAF recurrent mutation causes central conducting lymphatic anomaly treatable with a MEK inhibitor

(2019) Dong Li et al.   NATURE MEDICINE

Insights into pediatric rhabdomyosarcoma research: Challenges and goals

(2019) Marielle E. Yohe et al.   PEDIATRIC BLOOD & CANCER

Deep learning enables automated volumetric assessments of cardiac function in zebrafish

(2019) Alexander A. Akerberg et al.   Disease Models & Mechanisms

Maintenance of Melanocyte Stem Cell Quiescence by GABA-A Signaling in Larval Zebrafish

(2019) James R. Allen et al.   GENETICS

Mutations in RABL3 alter KRAS prenylation and are associated with hereditary pancreatic cancer

(2019) Sahar Nissim et al.   NATURE GENETICS

Adult immune compromised zebrafish for xenograft cell transplantation studies

(2019) Chuan Yan et al.   EBioMedicine

Zebrafish MITF-Low Melanoma Subtype Models Reveal Transcriptional Subclusters and MITF-Independent Residual Disease

(2019) Jana Travnickova et al.   CANCER RESEARCH

Use of Zebrafish in Drug Discovery Toxicology

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

Zebrafish earn their drug discovery stripes

(2019) Megan Cully   NATURE REVIEWS DRUG DISCOVERY

A NF-ĸB-Activin A signaling axis enhances prostate cancer metastasis

(2019) Lanpeng Chen et al.   ONCOGENE

Zebrafish behavioural profiling identifies GABA and serotonin receptor ligands related to sedation and paradoxical excitation

(2019) Matthew N. McCarroll et al.   Nature Communications

Identification of pathways that regulate circadian rhythms using a larval zebrafish small molecule screen

(2019) Eric A. Mosser et al.   Scientific Reports

A new perspective on fungal metabolites: identification of bioactive compounds from fungi using zebrafish embryogenesis as read-out

(2019) Jelmer Hoeksma et al.   Scientific Reports

The sixth international RASopathies symposium: Precision medicine—From promise to practice

(2019) Karen W. Gripp et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A

Humanized zebrafish enhance human hematopoietic stem cell survival and promote acute myeloid leukemia clonal diversity

(2019) Vinothkumar Rajan et al.   HAEMATOLOGICA

Internal state dynamics shape brainwide activity and foraging behaviour

(2019) João C. Marques et al.   NATURE

Mosaic RAS/MAPK variants cause sporadic vascular malformations which respond to targeted therapy

(2018) Lara Al-Olabi et al.   JOURNAL OF CLINICAL INVESTIGATION

Transplantation of Human-Induced Pluripotent Stem Cell-Derived Neural Precursors into Early-Stage Zebrafish Embryos

(2018) J. Strnadel et al.   JOURNAL OF MOLECULAR NEUROSCIENCE

A whole-animal platform to advance a clinical kinase inhibitor into new disease space

(2018) Masahiro Sonoshita et al.   Nature Chemical Biology

Chemical probes and drug leads from advances in synthetic planning and methodology

(2018) Christopher J. Gerry et al.   NATURE REVIEWS DRUG DISCOVERY

Somatic Activating KRAS Mutations in Arteriovenous Malformations of the Brain

(2018) Sergey I. Nikolaev et al.   NEW ENGLAND JOURNAL OF MEDICINE

Target identification of small molecules using large-scale CRISPR-Cas mutagenesis scanning of essential genes

(2018) Jasper Edgar Neggers et al.   Nature Communications

Donated chemical probes for open science

(2018) Susanne Müller et al.   eLife

An automated high-resolution in vivo screen in zebrafish to identify chemical regulators of myelination

(2018) Jason J Early et al.   eLife

Preclinical Animal Models for Dravet Syndrome: Seizure Phenotypes, Comorbidities and Drug Screening

(2018) Aliesha Griffin et al.   Frontiers in Pharmacology

Zebrafish blastomere screen identifies retinoic acid suppression of MYB in adenoid cystic carcinoma

(2018) Joseph Mandelbaum et al.   JOURNAL OF EXPERIMENTAL MEDICINE

Inhibitors of histone acetyltransferases KAT6A/B induce senescence and arrest tumour growth

(2018) Jonathan B. Baell et al.   NATURE

Rare Genetic Blood Disease Modeling in Zebrafish

(2018) Alberto Rissone et al.   Frontiers in Genetics

Targeting Processive Transcription Elongation via SEC Disruption for MYC-Induced Cancer Therapy

(2018) Kaiwei Liang et al.   CELL

Zebrafish knock-ins swim into the mainstream

(2018) Sergey V. Prykhozhij et al.   Disease Models & Mechanisms

Human tumor genomics and zebrafish modeling identify SPRED1 loss as a driver of mucosal melanoma

(2018) Julien Ablain et al.   SCIENCE

Grxcr1 Promotes Hair Bundle Development by Destabilizing the Physical Interaction between Harmonin and Sans Usher Syndrome Proteins

(2018) Bernardo Blanco-Sánchez et al.   Cell Reports

Zebrafish Models of Human Disease: Gaining Insight into Human Disease at ZFIN

(2017) Yvonne M. Bradford et al.   ILAR JOURNAL

Dissecting hematopoietic and renal cell heterogeneity in adult zebrafish at single-cell resolution using RNA sequencing

(2017) Qin Tang et al.   JOURNAL OF EXPERIMENTAL MEDICINE

Phenotypic Optimization of Urea–Thiophene Carboxamides To Yield Potent, Well Tolerated, and Orally Active Protective Agents against Aminoglycoside-Induced Hearing Loss

(2017) Sarwat Chowdhury et al.   JOURNAL OF MEDICINAL CHEMISTRY

Divergent effects of intrinsically active MEK variants on developmental Ras signaling

(2017) Yogesh Goyal et al.   NATURE GENETICS

Opportunities and challenges in phenotypic drug discovery: an industry perspective

(2017) John G. Moffat et al.   NATURE REVIEWS DRUG DISCOVERY

PRMT5 as a druggable target for glioblastoma therapy

(2017) Yeshavanth Kumar Banasavadi-Siddegowda et al.   NEURO-ONCOLOGY

Single-cell functional and chemosensitive profiling of combinatorial colorectal therapy in zebrafish xenografts

(2017) Rita Fior et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

In vivo severity ranking of Ras pathway mutations associated with developmental disorders

(2017) Granton A. Jindal et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Small molecule screen in embryonic zebrafish using modular variations to target segmentation

(2017) Sandra Richter et al.   Nature Communications

A chemical screen in zebrafish embryonic cells establishes that Akt activation is required for neural crest development

(2017) Christie Ciarlo et al.   eLife

Structural and Functional View of Polypharmacology

(2017) Aurelio Moya-García et al.   Scientific Reports

Leveraging Large-scale Behavioral Profiling in Zebrafish to Explore Neuroactive Polypharmacology

(2016) Matthew N. McCarroll et al.   ACS Chemical Biology

Single-cell imaging of normal and malignant cell engraftment into optically clear prkdc -null SCID zebrafish

(2016) John C. Moore et al.   JOURNAL OF EXPERIMENTAL MEDICINE

Rapid Discovery and Structure–Activity Relationships of Pyrazolopyrimidines That Potently Suppress Breast Cancer Cell Growth via SRC Kinase Inhibition with Exceptional Selectivity over ABL Kinase

(2016) Craig Fraser et al.   JOURNAL OF MEDICINAL CHEMISTRY

Natural Products as Sources of New Drugs from 1981 to 2014

(2016) David J. Newman et al.   JOURNAL OF NATURAL PRODUCTS

Stress from Nucleotide Depletion Activates the Transcriptional Regulator HEXIM1 to Suppress Melanoma

(2016) Justin L. Tan et al.   MOLECULAR CELL

Zebrafish behavioral profiling identifies multitarget antipsychotic-like compounds

(2016) Giancarlo Bruni et al.   Nature Chemical Biology

Cornerstones of CRISPR–Cas in drug discovery and therapy

(2016) Christof Fellmann et al.   NATURE REVIEWS DRUG DISCOVERY

A zebrafish melanoma model reveals emergence of neural crest identity during melanoma initiation

(2016) C. K. Kaufman et al.   SCIENCE

Visualizing Human Hematopoietic Stem Cell Trafficking In Vivo Using a Zebrafish Xenograft Model

(2016) Frank J.T. Staal et al.   STEM CELLS AND DEVELOPMENT

MEK Inhibitors Reverse Growth of Embryonal Brain Tumors Derived from Oligoneural Precursor Cells

(2016) Katarzyna Modzelewska et al.   Cell Reports

MEK Inhibitors Reverse cAMP-Mediated Anxiety in Zebrafish

(2015) Pia R. Lundegaard et al.   CHEMISTRY & BIOLOGY

The melanocyte lineage in development and disease

(2015) R. L. Mort et al.   DEVELOPMENT

Challenges in understanding psychiatric disorders and developing therapeutics: a role for zebrafish

(2015) J. M. McCammon et al.   Disease Models & Mechanisms

RASopathies: unraveling mechanisms with animal models

(2015) G. A. Jindal et al.   Disease Models & Mechanisms

Zebrafish: An Important Tool for Liver Disease Research

(2015) Wolfram Goessling et al.   GASTROENTEROLOGY

Zebrafish as tools for drug discovery

(2015) Calum A. MacRae et al.   NATURE REVIEWS DRUG DISCOVERY

The re-emergence of natural products for drug discovery in the genomics era

(2015) Alan L. Harvey et al.   NATURE REVIEWS DRUG DISCOVERY

Inhibition of the prostaglandin-degrading enzyme 15-PGDH potentiates tissue regeneration

(2015) Y. Zhang et al.   SCIENCE

Clonal Evolution Enhances Leukemia-Propagating Cell Frequency in T Cell Acute Lymphoblastic Leukemia through Akt/mTORC1 Pathway Activation

(2014) Jessica S. Blackburn et al.   CANCER CELL

Zebrafish models in translational research: tipping the scales toward advancements in human health

(2014) J. B. Phillips et al.   Disease Models & Mechanisms

Transgenic Zebrafish Reveal Tissue-Specific Differences in Estrogen Signaling in Response to Environmental Water Samples

(2014) Daniel A. Gorelick et al.   ENVIRONMENTAL HEALTH PERSPECTIVES

Focused chemical genomics using zebrafish xenotransplantation as a pre-clinical therapeutic platform for T-cell acute lymphoblastic leukemia

(2014) V. L. Bentley et al.   HAEMATOLOGICA

Glycogen synthase kinase 3 inhibitors induce the canonical WNT/ -catenin pathway to suppress growth and self-renewal in embryonal rhabdomyosarcoma

(2014) E. Y. Chen et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Visnagin protects against doxorubicin-induced cardiomyopathy through modulation of mitochondrial malate dehydrogenase

(2014) Y. Liu et al.   Science Translational Medicine

Selectivity of natural, synthetic and environmental estrogens for zebrafish estrogen receptors

(2014) Caroline Pinto et al.   TOXICOLOGY AND APPLIED PHARMACOLOGY

Extracellular palladium-catalysed dealkylation of 5-fluoro-1-propargyl-uracil as a bioorthogonally activated prodrug approach

(2014) Jason T. Weiss et al.   Nature Communications

S-Nitrosothiol Signaling Regulates Liver Development and Improves Outcome following Toxic Liver Injury

(2014) Andrew G. Cox et al.   Cell Reports

Prostaglandin E2 enhances long-term repopulation but does not permanently alter inherent stem cell competitiveness

(2013) J. Hoggatt et al.   BLOOD

Prostaglandin-modulated umbilical cord blood hematopoietic stem cell transplantation

(2013) C. Cutler et al.   BLOOD

AML1-ETO mediates hematopoietic self-renewal and leukemogenesis through a COX/ -catenin signaling pathway

(2013) Y. Zhang et al.   BLOOD

A novel chemical screening strategy in zebrafish identifies common pathways in embryogenesis and rhabdomyosarcoma development

(2013) X. Le et al.   DEVELOPMENT

Dopamine from the Brain Promotes Spinal Motor Neuron Generation during Development and Adult Regeneration

(2013) Michell M. Reimer et al.   DEVELOPMENTAL CELL

The zebrafish reference genome sequence and its relationship to the human genome

(2013) Kerstin Howe et al.   NATURE

Lenalidomide Causes Selective Degradation of IKZF1 and IKZF3 in Multiple Myeloma Cells

(2013) J. Kronke et al.   SCIENCE

The Myeloma Drug Lenalidomide Promotes the Cereblon-Dependent Destruction of Ikaros Proteins

(2013) G. Lu et al.   SCIENCE

Towards a Comprehensive Catalog of Zebrafish Behavior 1.0 and Beyond

(2013) Allan V. Kalueff et al.   Zebrafish

Drug screening in Scn1a zebrafish mutant identifies clemizole as a potential Dravet syndrome treatment

(2013) Scott C. Baraban et al.   Nature Communications

Zebrafish screen identifies novel compound with selective toxicity against leukemia

(2012) S. Ridges et al.   BLOOD

ALDH2 Mediates 5-Nitrofuran Activity in Multiple Species

(2012) Linna Zhou et al.   CHEMISTRY & BIOLOGY

Rargb regulates organ laterality in a zebrafish model of right atrial isomerism

(2012) Maija K. Garnaas et al.   DEVELOPMENTAL BIOLOGY

Identification of Modulators of Hair Cell Regeneration in the Zebrafish Lateral Line

(2012) P. Namdaran et al.   JOURNAL OF NEUROSCIENCE

Neutrophil-mediated experimental metastasis is enhanced by VEGFR inhibition in a zebrafish xenograft model

(2012) Shuning He et al.   JOURNAL OF PATHOLOGY

Chemical genetic discovery of targets and anti-targets for cancer polypharmacology

(2012) Arvin C. Dar et al.   NATURE

Whole-organism screening for gluconeogenesis identifies activators of fasting metabolism

(2012) Philipp Gut et al.   Nature Chemical Biology

Automated Whole Animal Bio-Imaging Assay for Human Cancer Dissemination

(2012) Veerander P. S. Ghotra et al.   PLoS One

Prostaglandin E2 Enhances Human Cord Blood Stem Cell Xenotransplants and Shows Long-Term Safety in Preclinical Nonhuman Primate Transplant Models

(2011) Wolfram Goessling et al.   Cell Stem Cell

DHODH modulates transcriptional elongation in the neural crest and melanoma

(2011) Richard Mark White et al.   NATURE

Chemical informatics and target identification in a zebrafish phenotypic screen

(2011) Christian Laggner et al.   Nature Chemical Biology

How were new medicines discovered?

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

A new zebrafish model for experimental leukemia therapy

(2010) Igor V. Mizgirev et al.   CANCER BIOLOGY & THERAPY

Combined zebrafish-yeast chemical-genetic screens reveal gene-copper-nutrition interactions that modulate melanocyte pigmentation

(2010) H. Ishizaki et al.   Disease Models & Mechanisms

Distinct Biological Network Properties between the Targets of Natural Products and Disease Genes

(2010) Vlado Dančík et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

PGE2-regulated wnt signaling and N-acetylcysteine are synergistically hepatoprotective in zebrafish acetaminophen injury

(2010) T. E. North et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Zebrafish Behavioral Profiling Links Drugs to Biological Targets and Rest/Wake Regulation

(2010) J. Rihel et al.   SCIENCE

Identification of a Primary Target of Thalidomide Teratogenicity

(2010) T. Ito et al.   SCIENCE

Prostaglandin E2 enhances hematopoietic stem cell homing, survival, and proliferation

(2009) J. Hoggatt et al.   BLOOD

Genetic Interaction of PGE2 and Wnt Signaling Regulates Developmental Specification of Stem Cells and Regeneration

(2009) Wolfram Goessling et al.   CELL

Mechanisms of Action of Antipsychotic Drugs of Different Classes, Refractoriness to Therapeutic Effects of Classical Neuroleptics, and Individual Variation in Sensitivity to their Actions: PART I

(2009) R. Miller   Current Neuropharmacology

Mechanisms of Action of Antipsychotic Drugs of Different Classes, Refractoriness to Therapeutic Effects of Classical Neuroleptics, and Individual Variation in Sensitivity to their Actions: PART II

(2009) R. Miller   Current Neuropharmacology

Kinase-activating and kinase-impaired cardio-facio-cutaneous syndrome alleles have activity during zebrafish development and are sensitive to small molecule inhibitors

(2009) C. Anastasaki et al.   HUMAN MOLECULAR GENETICS

Discovering chemical modifiers of oncogene-regulated hematopoietic differentiation

(2009) Jing-Ruey J Yeh et al.   Nature Chemical Biology

Recurrent fusion of MYB and NFIB transcription factor genes in carcinomas of the breast and head and neck

(2009) M. Persson et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

BMP type I receptor inhibition reduces heterotopic ossification

(2008) Paul B Yu et al.   NATURE MEDICINE

Identification of Genetic and Chemical Modulators of Zebrafish Mechanosensory Hair Cell Death

(2008) Kelly N. Owens et al.   PLoS Genetics