Arnaud Klein
Chargé de recherche INSERM

Présentation
Chercheur dans l’équipe 4/ groupe Furling, mes projets portent sur le développement de stratégies thérapeutiques innovantes pour la Dystrophie Myotonique de type 1. Nous utilisons des approches de thérapie génique ou des oligonucléotides antisens que nous évaluons dans des modèles cellulaires (myoblastes, fibroblastes ou surexpression) et murins de la maladie.
Principales publications
- Klein, AF, Arandel, L, Marie, J, Furling, D. FISH Protocol for Myotonic Dystrophy Type 1 Cells. Methods Mol Biol. 2020;2056 :203-215. doi: 10.1007/978-1-4939-9784-8_13. PubMed PMID:31586350 .
- Klein, AF, Varela, MA, Arandel, L, Holland, A, Naouar, N, Arzumanov, A et al.. Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice. J Clin Invest. 2019;129 (11):4739-4744. doi: 10.1172/JCI128205. PubMed PMID:31479430 PubMed Central PMC6819114.
- Matloka, M, Klein, AF, Rau, F, Furling, D. Cells of Matter-In Vitro Models for Myotonic Dystrophy. Front Neurol. 2018;9 :361. doi: 10.3389/fneur.2018.00361. PubMed PMID:29875732 PubMed Central PMC5974047.
- Arandel, L, Polay Espinoza, M, Matloka, M, Bazinet, A, De Dea Diniz, D, Naouar, N et al.. Immortalized human myotonic dystrophy muscle cell lines to assess therapeutic compounds. Dis Model Mech. 2017;10 (4):487-497. doi: 10.1242/dmm.027367. PubMed PMID:28188264 PubMed Central PMC5399563.
- Laurent, FX, Sureau, A, Klein, AF, Trouslard, F, Gasnier, E, Furling, D et al.. New function for the RNA helicase p68/DDX5 as a modifier of MBNL1 activity on expanded CUG repeats. Nucleic Acids Res. 2012;40 (7):3159-71. doi: 10.1093/nar/gkr1228. PubMed PMID:22156369 PubMed Central PMC3326330.
- Fugier, C, Klein, AF, Hammer, C, Vassilopoulos, S, Ivarsson, Y, Toussaint, A et al.. Misregulated alternative splicing of BIN1 is associated with T tubule alterations and muscle weakness in myotonic dystrophy. Nat Med. 2011;17 (6):720-5. doi: 10.1038/nm.2374. PubMed PMID:21623381 .
- François, V, Klein, AF, Beley, C, Jollet, A, Lemercier, C, Garcia, L et al.. Selective silencing of mutated mRNAs in DM1 by using modified hU7-snRNAs. Nat Struct Mol Biol. 2011;18 (1):85-7. doi: 10.1038/nsmb.1958. PubMed PMID:21186365 .