Model Organisms in the Fight against Muscular Dystrophy: Lessons from Drosophila and Zebrafish

Myotonic dystrophies: An update on clinical aspects, genetic, pathology, and molecular pathomechanisms

(2015) Giovanni Meola et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Making Sense of Anti-Sense Data

(2015) Didier Y.R. Stainier et al.   DEVELOPMENTAL CELL

Molecular mechanisms in DM1 — a focus on foci

(2015) Olof Joakim Pettersson et al.   NUCLEIC ACIDS RESEARCH

Precise and efficient genome editing in zebrafish using the CRISPR/Cas9 system

(2014) U. Irion et al.   DEVELOPMENT

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

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

Recent advances using zebrafish animal models for muscle disease drug discovery

(2014) Lisa Maves   Expert Opinion on Drug Discovery

A CRISPR view of development

(2014) Melissa M. Harrison et al.   GENES & DEVELOPMENT

Fluoxetine prevents dystrophic changes in a zebrafish model of Duchenne muscular dystrophy

(2014) T. A. Waugh et al.   HUMAN MOLECULAR GENETICS

Modifications to toxic CUG RNAs induce structural stability, rescue mis-splicing in a myotonic dystrophy cell model and reduce toxicity in a myotonic dystrophy zebrafish model

(2014) Elaine deLorimier et al.   NUCLEIC ACIDS RESEARCH

Transcriptional changes and developmental abnormalities in a zebrafish model of myotonic dystrophy type 1

(2013) P. K. Todd et al.   Disease Models & Mechanisms

Muscle dysfunction and structural defects of dystrophin-null sapje mutant zebrafish larvae are rescued by ataluren treatment

(2013) Mei Li et al.   FASEB JOURNAL

Repair or replace? Exploiting novel gene and cell therapy strategies for muscular dystrophies

(2013) Sara Benedetti et al.   FEBS Journal

Swimming into prominence: the zebrafish as a valuable tool for studying human myopathies and muscular dystrophies

(2013) Elizabeth M. Gibbs et al.   FEBS Journal

Novel Drosophila model of myotonic dystrophy type 1: phenotypic characterization and genome-wide view of altered gene expression

(2013) Lucie Picchio et al.   HUMAN MOLECULAR GENETICS

High-content screening identifies small molecules that remove nuclear foci, affect MBNL distribution and CELF1 protein levels via a PKC-independent pathway in myotonic dystrophy cell lines

(2013) Ami Ketley et al.   HUMAN MOLECULAR GENETICS

Dystrophic muscle improvement in zebrafish via increased heme oxygenase signaling

(2013) Genri Kawahara et al.   HUMAN MOLECULAR GENETICS

Muscular dystrophies

(2013) Eugenio Mercuri et al.   LANCET

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

(2013) Kerstin Howe et al.   NATURE

Animal models for metabolic, neuromuscular and ophthalmological rare diseases

(2013) Guillaume Vaquer et al.   NATURE REVIEWS DRUG DISCOVERY

Highly Efficient Targeted Mutagenesis of Drosophila with the CRISPR/Cas9 System

(2013) Andrew R. Bassett et al.   Cell Reports

Smaug/SAMD4A Restores Translational Activity of CUGBP1 and Suppresses CUG-Induced Myopathy

(2013) Maria de Haro et al.   PLoS Genetics

The ever-expanding spectrum of congenital muscular dystrophies

(2012) Eugenio Mercuri et al.   ANNALS OF NEUROLOGY

Genetic elevation of Sphingosine 1-phosphate suppresses dystrophic muscle phenotypes in Drosophila

(2012) M. Pantoja et al.   DEVELOPMENT

Loss of Drosophila A-type lamin C initially causes tendon abnormality including disintegration of cytoskeleton and nuclear lamina in muscular defects

(2012) Ryo Uchino et al.   DEVELOPMENTAL BIOLOGY

Zebrafish models flex their muscles to shed light on muscular dystrophies

(2012) J. Berger et al.   Disease Models & Mechanisms

Emerging drugs for Duchenne muscular dystrophy

(2012) Vinod Malik et al.   EXPERT OPINION ON EMERGING DRUGS

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

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

A Novel Inhibitor of Amyloid β (Aβ) Peptide Aggregation

(2012) Angela Fortner McKoy et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Hooked! Modeling human disease in zebrafish

(2012) Cristina Santoriello et al.   JOURNAL OF CLINICAL INVESTIGATION

A Splice Site Mutation in Laminin-α2 Results in a Severe Muscular Dystrophy and Growth Abnormalities in Zebrafish

(2012) Vandana A. Gupta et al.   PLoS One

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

(2012) M. Jinek et al.   SCIENCE

Gain of RNA function in pathological cases: Focus on myotonic dystrophy

(2011) A.F. Klein et al.   BIOCHIMIE

Lessons from morpholino-based screening in zebrafish

(2011) V. M. Bedell et al.   Briefings in Functional Genomics

Stress and muscular dystrophy: A genetic screen for Dystroglycan and Dystrophin interactors in Drosophila identifies cellular stress response components

(2011) Mariya M. Kucherenko et al.   DEVELOPMENTAL BIOLOGY

Zebrafish deficient for Muscleblind-like 2 exhibit features of myotonic dystrophy

(2011) L. E. Machuca-Tzili et al.   Disease Models & Mechanisms

The zebrafish dag1 mutant: a novel genetic model for dystroglycanopathies

(2011) Vandana Gupta et al.   HUMAN MOLECULAR GENETICS

Connexin 39.9 Protein Is Necessary for Coordinated Activation of Slow-twitch Muscle and Normal Behavior in Zebrafish

(2011) Hiromi Hirata et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Evaluation of exon-skipping strategies for Duchenne muscular dystrophy utilizing dystrophin-deficient zebrafish

(2011) Joachim Berger et al.   JOURNAL OF CELLULAR AND MOLECULAR MEDICINE

Human Disease Models in Drosophila melanogaster and the Role of the Fly in Therapeutic Drug Discovery

(2011) U. B. Pandey et al.   PHARMACOLOGICAL REVIEWS

High-Content Chemical and RNAi Screens for Suppressors of Neurotoxicity in a Huntington's Disease Model

(2011) Joost Schulte et al.   PLoS One

In vivo discovery of a peptide that prevents CUG-RNA hairpin formation and reverses RNA toxicity in myotonic dystrophy models

(2011) A. Garcia-Lopez et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Drug screening in a zebrafish model of Duchenne muscular dystrophy

(2011) G. Kawahara et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Congenital Muscular Dystrophies: A Brief Review

(2011) Enrico Bertini et al.   Seminars in Pediatric Neurology

Flightless flies: Drosophila models of neuromuscular disease

(2010) Thomas E. Lloyd et al.   Annals of the New York Academy of Sciences

High-throughput screening and small animal models, where are we?

(2010) Jean Giacomotto et al.   BRITISH JOURNAL OF PHARMACOLOGY

TDP-43 Mediates Degeneration in a Novel Drosophila Model of Disease Caused by Mutations in VCP/p97

(2010) G. P. Ritson et al.   JOURNAL OF NEUROSCIENCE

Increased Apoptosis of Myoblasts in Drosophila Model for the Walker-Warburg Syndrome

(2010) Morio Ueyama et al.   PLoS One

Chronic Hypoxia Impairs Muscle Function in the Drosophila Model of Duchenne's Muscular Dystrophy (DMD)

(2010) Matias Mosqueira et al.   PLoS One

Dystrophin deficiency in Drosophila reduces lifespan and causes a dilated cardiomyopathy phenotype

(2008) Ouarda Taghli-Lamallem et al.   AGING CELL

Genetic isolation and characterization of a splicing mutant of zebrafish dystrophin

(2008) Jeffrey R. Guyon et al.   HUMAN MOLECULAR GENETICS

Synaptic Defects in a Drosophila Model of Congenital Muscular Dystrophy

(2008) Y. P. Wairkar et al.   JOURNAL OF NEUROSCIENCE

Identification of small molecules rescuing fragile X syndrome phenotypes in Drosophila

(2008) Shuang Chang et al.   Nature Chemical Biology

Genetic and Chemical Modifiers of a CUG Toxicity Model in Drosophila

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

Genetic Modifier Screens Reveal New Components that Interact with the Drosophila Dystroglycan-Dystrophin Complex

(2008) Mariya M. Kucherenko et al.   PLoS One

Modeling Spinal Muscular Atrophy in Drosophila

(2008) Howard Chia-Hao Chang et al.   PLoS One