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

J Giacomotto, L Ségalat - British journal of pharmacology, 2010 - Wiley Online Library
Current high‐throughput screening methods for drug discovery rely on the existence of
targets. Moreover, most of the hits generated during screenings turn out to be invalid after …

Duchenne muscular dystrophy animal models for high-throughput drug discovery and precision medicine

NB Wasala, SJ Chen, D Duan - Expert opinion on drug discovery, 2020 - Taylor & Francis
Introduction: Duchenne muscular dystrophy (DMD) is an X-linked handicapping disease due
to the loss of an essential muscle protein dystrophin. Dystrophin-null animals have been …

Affinity proteomics within rare diseases: a BIO‐NMD study for blood biomarkers of muscular dystrophies

B Ayoglu, A Chaouch, H Lochmüller… - EMBO molecular …, 2014 - embopress.org
Despite the recent progress in the broad‐scaled analysis of proteins in body fluids, there is
still a lack in protein profiling approaches for biomarkers of rare diseases. Scarcity of …

The carbonic anhydrase inhibitor methazolamide prevents amyloid beta-induced mitochondrial dysfunction and caspase activation protecting neuronal and glial cells …

S Fossati, P Giannoni, ME Solesio, SL Cocklin… - Neurobiology of …, 2016 - Elsevier
Mitochondrial dysfunction has been recognized as an early event in Alzheimer's disease
(AD) pathology, preceding and inducing neurodegeneration and memory loss. The …

Sodium (23Na) MRI detects elevated muscular sodium concentration in Duchenne muscular dystrophy

MA Weber, AM Nagel, K Jurkat-Rott, F Lehmann-Horn - Neurology, 2011 - AAN Enterprises
Objective: In boys with Duchenne muscular dystrophy (DMD), 1H MRI suggested muscular
edema before fatty degeneration. Using specific 23Na MRI sequences, we tested the …

[HTML][HTML] Duchenne muscular dystrophy hiPSC–derived myoblast drug screen identifies compounds that ameliorate disease in mdx mice

C Sun, IY Choi, YIR Gonzalez, P Andersen… - JCI insight, 2020 - ncbi.nlm.nih.gov
Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy. In the
present study, when human induced pluripotent stem cells (hiPSCs) were differentiated into …

Disrupted-in-Schizophrenia 1–mediated axon guidance involves TRIO-RAC-PAK small GTPase pathway signaling

SY Chen, PH Huang, HJ Cheng - Proceedings of the …, 2011 - National Acad Sciences
Defects in neuronal connectivity of the brain are well documented among schizophrenia
patients. Although the schizophrenia susceptibility gene Disrupted-in-Schizophrenia 1 …

A novel Caenorhabditis elegans allele, smn-1(cb131), mimicking a mild form of spinal muscular atrophy, provides a convenient drug screening platform highlighting …

JN Sleigh, SD Buckingham, B Esmaeili… - Human molecular …, 2011 - academic.oup.com
Spinal muscular atrophy (SMA), an autosomal recessive genetic disorder, is characterized
by the selective degeneration of lower motor neurons, leading to muscle atrophy and, in the …

Ultra-structural time-course study in the C. elegans model for Duchenne muscular dystrophy highlights a crucial role for sarcomere-anchoring structures and …

N Brouilly, C Lecroisey, E Martin… - Human molecular …, 2015 - academic.oup.com
Duchenne muscular dystrophy (DMD) is a genetic disease characterized by progressive
muscle degeneration due to mutations in the dystrophin gene. In spite of great advances in …

Chemical genetics unveils a key role of mitochondrial dynamics, cytochrome c release and IP3R activity in muscular dystrophy

J Giacomotto, N Brouilly, L Walter… - Human molecular …, 2013 - academic.oup.com
Duchenne muscular dystrophy (DMD) is a neuromuscular disease caused by mutations in
the dystrophin gene. The subcellular mechanisms of DMD remain poorly understood and …