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essai préclinique
Commentaire :
Étude expérimentale menée en laboratoire soit sur l'animal (in vivo), soit in vitro, qui est préalable à l'administration d'un nouveau produit ou à l'utilisation d'un nouveau dispositif médical chez l'homme. (Grand dictionnaire terminologique, 19/10/2006)
Synonyme(s)phase préclinique ;preclinical studies test préclinique |
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Recent developments in the field of cachexia, sarcopenia, and muscle wasting: highlights from the 12th Cachexia Conference
Ebner N, Anker SD, von Haehling S
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RyR1-targeted drug discovery pipeline integrating FRET-based high-throughput screening and human myofiber dynamic Ca(2+) assays
Rebbeck RT, Singh DP, Janicek KA, et al.
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Metabolism and Disposition of Ataluren following Oral Administration to Mice, Rats, Dogs, and Humans
Kong R, Ma J, Hwang S, et al.
Drug metabolism and disposition: the biological fate of chemicals, 2020
Titin splicing regulates cardiotoxicity associated with calpain 3 gene therapy for limb-girdle muscular dystrophy type 2A
Lostal W, Roudaut C, Faivre M, et al.
Science translational medicine, 2019, 11, 520
Low fat diets increase survival of a mouse model of spinal muscular atrophy
Deguise MO, Chehade L, Tierney A, et al.
Annals of clinical and translational neurology, 2019
Metformin attenuates autoimmune disease of the neuromotor system in animal models of myasthenia gravis
Cui Y, Chang L, Wang C, et al.
International immunopharmacology, 2019, 75
rAAVrh74.MCK.GALGT2 Demonstrates Safety and Widespread Muscle Glycosylation after Intravenous Delivery in C57BL/6J Mice
Zygmunt DA, Xu R, Jia Y, et al.
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A CTG repeat-selective chemical screen identifies microtubule inhibitors as selective modulators of toxic CUG RNA levels
Reddy K, Jenquin JR, McConnell OL, et al.
Proceedings of the national Academy of sciences of the United States of America, 2019, 116, 42
Selective modulation of the androgen receptor AF2 domain rescues degeneration in spinal bulbar muscular atrophy
Badders NM, Korff A, Miranda HC, et al.
Nature medicine, 2018
Les cellules souches : Comprendre la recherche
Dupuy-Maury F
VLM. Vaincre les myopathies, 2018, 184, p. 23
Long-Term Sustained Effect of Liver-Targeted Adeno-Associated Virus Gene Therapy for Mitochondrial Neurogastrointestinal Encephalomyopathy
Torres-Torronteras J, Cabrera-Pérez R, Vila-Julia F, et al.
Human gene therapy, 2018
Acetylcholine receptor antibody-mediated animal models of myasthenia gravis and the role of complement
Kusner LL, Sengupta M, Kaminski HJ
Annals of the New York Academy of Sciences, 2018, 1413, 1, p 136
Effect of a long-term treatment with metformin in dystrophic mdx mice: A reconsideration of its potential clinical interest in Duchenne muscular dystrophy
Mantuano P, Sanarica F, Conte E, et al.
Biochemical pharmacology, 2018, 154, p 89
Overview of Current Drugs and Molecules in Development for Spinal Muscular Atrophy Therapy
Shorrock HK, Gillingwater TH, Groen EJN
Drugs, 2018, 78, 3, p 293
Ryanodine channel complex stabilizer compound S48168/ARM210 as a disease modifier in dystrophin-deficient mdx mice: proof-of-concept study and independent validation of efficacy
Capogrosso RF, Mantuano P, Uaesoontrachoon K, et al.
FASEB journal : official publication of the Federation of American Societies for Experimental Biology, 2018, 32, 2, p 1025
Dual AAV gene therapy for Duchenne muscular dystrophy with a 7-kb mini-dystrophin gene in the canine model
Kodippili K, Hakim C, Pan X, et al.
Human gene therapy, 2018, 29, 3, p 299
Test of Antifibrotic Drugs in a Cellular Model of Fibrosis Based on Muscle-Derived Fibroblasts from Duchenne Muscular Dystrophy Patients
Zanotti S, Mora M
Methods in molecular biology, 2018, 1687, p 205
A Neuron-Specific Gene Therapy Relieves Motor Deficits in Pompe Disease Mice
Lee NC, Hwu WL, Muramatsu SI, et al.
Molecular neurobiology, 2018, 55, 6, p 5299
RNA-Seq Analysis of an Antisense Sequence Optimized for Exon Skipping in Duchenne Patients Reveals No Off-Target Effect
Domenger C, Allais M, François V, et al.
Molecular therapy. Nucleic acids, 2018, 10, p 277
Peripheral neuropathies: Antisense therapy for Charcot-Marie-Tooth disease?
Ridler C
Nature reviews. Neurology, 2018, 14, 2, p 64
Finalizing a plan to guarantee quality in translational research for neuromuscular diseases : 227th ENMC International Workshop - Heemskerk, Netherlands, 10-11 February 2017
Willmann R, Buccella F, de Luca A, et al.
Neuromuscular disorders : NMD, 2018, 28, 2, p 185
A dystrophic Duchenne mouse model for testing human antisense oligonucleotides
Veltrop M, van Vliet L, Hulsker M, et al.
PLoS ONE, 2018, 13, 2
Antisense oligonucleotides offer hope to patients with Charcot-Marie-Tooth disease type 1A
Shy ME
The Journal of clinical investigation, 2018, 128, 1, p 110
PMP22 antisense oligonucleotides reverse Charcot-Marie-Tooth disease type 1A features in rodent models
Zhao HT, Damle S, Ikeda-Lee K, et al.
The Journal of clinical investigation, 2018, 128, 1, p 359
