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Current Medicinal Chemistry

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ISSN (Print): 0929-8673
ISSN (Online): 1875-533X

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Target Mutation-Driven Drug Discovery

Author(s): Concetta Altamura*, Diana Conte, Maria Rosaria Carratù and Jean-François Desaphy

Volume 29, Issue 31, 2022

Published on: 23 May, 2022

Page: [5156 - 5158] Pages: 3

DOI: 10.2174/1389450123666220418111200

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Lopes-Pacheco, M. CFTR Modulators: The changing face of cystic fibrosis in the era of precision medicine. Front. Pharmacol., 2020, 10, 1662.
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Maggi, L.; Bonanno, S.; Altamura, C.; Desaphy, J.F. ion channel gene mutations causing skeletal muscle disorders: Pathomechanisms and opportunities for therapy. Cells, 2021, 10(6), 1521.
[http://dx.doi.org/10.3390/cells10061521] [PMID: 34208776]
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Altamura, C.; Lucchiari, S.; Sahbani, D.; Ulzi, G.; Comi, G.P.; D’Ambrosio, P.; Petillo, R.; Politano, L.; Vercelli, L.; Mongini, T.; Dotti, M.T.; Cardani, R.; Meola, G.; Lo Monaco, M.; Matthews, E.; Hanna, M.G.; Carratù, M.R.; Conte, D.; Imbrici, P.; Desaphy, J.F. The analysis of myotonia congenita mutations discloses functional clusters of amino acids within the CBS2 domain and the C-terminal peptide of the ClC-1 channel. Hum. Mutat., 2018, 39(9), 1273-1283.
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Farinato, A.; Altamura, C.; Imbrici, P.; Maggi, L.; Bernasconi, P.; Mantegazza, R.; Pasquali, L.; Siciliano, G.; Lo Monaco, M.; Vial, C.; Sternberg, D.; Carratù, M.R.; Conte, D.; Desaphy, J.F. Pharmacogenetics of myotonic hNav1.4 sodium channel variants situated near the fast inactivation gate. Pharmacol. Res., 2019, 141, 224-235.
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Desaphy, J.F.; Modoni, A.; Lomonaco, M.; Camerino, D.C. Dramatic improvement of myotonia permanens with flecainide: A two-case report of a possible bench-to-bedside pharmacogenetics strategy. Eur. J. Clin. Pharmacol., 2013, 69(4), 1037-1039.
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Desaphy, J.F.; Carbonara, R.; D’Amico, A.; Modoni, A.; Roussel, J.; Imbrici, P.; Pagliarani, S.; Lucchiari, S.; Lo Monaco, M.; Conte Camerino, D. Translational approach to address therapy in myotonia permanens due to a new SCN4A mutation. Neurology, 2016, 86(22), 2100-2108.
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Portaro, S.; Rodolico, C.; Sinicropi, S.; Musumeci, O.; Valenzise, M.; Toscano, A. Flecainide-responsive myotonia permanens with SNEL Onset: A new case and literature review. Pediatrics, 2016, 137(4), e20153289.
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Lehmann-Horn, F.; D’Amico, A.; Bertini, E.; Lomonaco, M.; Merlini, L.; Nelson, K.R.; Philippi, H.; Siciliano, G.; Spaans, F.; Jurkat-Rott, K. Myotonia permanens with Nav1.4-G1306E displays varied phenotypes during course of life. Acta Myol., 2017, 36(3), 125-134.
[PMID: 29774303]
[15]
Altamura, C.; Mangiatordi, G.F.; Nicolotti, O.; Sahbani, D.; Farinato, A.; Leonetti, F.; Carratù, M.R.; Conte, D.; Desaphy, J.F.; Imbrici, P. Mapping ligand binding pockets in chloride ClC-1 channels through an integrated in silico and experimental approach using anthracene-9-carboxylic acid and niflumic acid. Br. J. Pharmacol., 2018, 175(10), 1770-1780.
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Pande, M.; Kundu, D.; Srivastava, R. Drugs repurposing against SARS-CoV2 and the new variant B.1.1.7 (alpha strain) targeting the spike protein: Molecular docking and simulation studies. Heliyon, 2021, 7(8), e07803.
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