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 …
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
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 …
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 …
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 …
Mitochondrial dysfunction has been recognized as an early event in Alzheimer's disease
(AD) pathology, preceding and inducing neurodegeneration and memory loss. The …
(AD) pathology, preceding and inducing neurodegeneration and memory loss. The …
Sodium (23Na) MRI detects elevated muscular sodium concentration in Duchenne muscular dystrophy
Objective: In boys with Duchenne muscular dystrophy (DMD), 1H MRI suggested muscular
edema before fatty degeneration. Using specific 23Na MRI sequences, we tested the …
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 …
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
Defects in neuronal connectivity of the brain are well documented among schizophrenia
patients. Although the schizophrenia susceptibility gene Disrupted-in-Schizophrenia 1 …
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 …
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 …
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 …
the dystrophin gene. The subcellular mechanisms of DMD remain poorly understood and …