Therapeutic Development in Charcot Marie Tooth Type 1 Disease.
CMT subtype
animal models
clinical trials
therapies
Journal
International journal of molecular sciences
ISSN: 1422-0067
Titre abrégé: Int J Mol Sci
Pays: Switzerland
ID NLM: 101092791
Informations de publication
Date de publication:
23 Jun 2021
23 Jun 2021
Historique:
received:
13
05
2021
revised:
11
06
2021
accepted:
15
06
2021
entrez:
2
7
2021
pubmed:
3
7
2021
medline:
27
7
2021
Statut:
epublish
Résumé
Charcot-Marie-Tooth disease (CMT) is the most frequent hereditary peripheral neuropathies. It is subdivided in two main groups, demyelinating (CMT1) and axonal (CMT2). CMT1 forms are the most frequent. The goal of this review is to present published data on 1-cellular and animal models having opened new potential therapeutic approaches. 2-exploration of these tracks, including clinical trials. The first conclusion is the great increase of publications on CMT1 subtypes since 2000. We discussed two points that should be considered in the therapeutic development toward a regulatory-approved therapy to be proposed to patients. The first point concerns long term safety if treatments will be a long-term process. The second point relates to the evaluation of treatment efficiency. Degradation of CMT clinical phenotype is not linear and progressive.
Identifiants
pubmed: 34201736
pii: ijms22136755
doi: 10.3390/ijms22136755
pmc: PMC8268813
pii:
doi:
Substances chimiques
Gangliosides
0
Neurotrophin 3
0
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Références
Glia. 2018 Jul;66(7):1302-1316
pubmed: 29423921
Hum Mol Genet. 2019 Nov 1;28(21):3528-3542
pubmed: 31411673
Brain. 2012 Jul;135(Pt 7):2032-47
pubmed: 22689911
Arch Neurol. 2003 Apr;60(4):605-9
pubmed: 12707076
Orphanet J Rare Dis. 2015 May 07;10:56
pubmed: 25947624
Am J Hum Genet. 2005 Nov;77(5):841-50
pubmed: 16252242
Orphanet J Rare Dis. 2014 Dec 10;9:201
pubmed: 25491744
Science. 2015 Apr 10;348(6231):239-42
pubmed: 25859045
Neurobiol Dis. 2000 Dec;7(6 Pt B):561-73
pubmed: 11114256
Neuromuscul Disord. 2007 Mar;17(3):248-53
pubmed: 17303424
Front Cell Neurosci. 2016 Mar 01;10:49
pubmed: 26973463
J Neurosci. 2011 Nov 23;31(47):17180-92
pubmed: 22114285
Neurosci Lett. 2016 Oct 6;632:33-8
pubmed: 27530546
J Neurosci. 2006 Feb 22;26(8):2358-68
pubmed: 16495463
J Neurol Neurosurg Psychiatry. 1997 Apr;62(4):310-8
pubmed: 9120441
Cell. 1991 Jul 26;66(2):219-32
pubmed: 1677316
Neurology. 2005 Sep 13;65(5):681-9
pubmed: 16157899
FEBS Lett. 2008 Oct 29;582(25-26):3614-8
pubmed: 18835269
Brain. 2011 Feb;134(Pt 2):585-601
pubmed: 21169333
J Neuropathol Exp Neurol. 2011 May;70(5):386-98
pubmed: 21487305
Glia. 2016 Apr;64(4):475-86
pubmed: 26250643
J Neurosci. 1989 Feb;9(2):625-38
pubmed: 2918381
Glia. 2012 Mar;60(3):457-64
pubmed: 22131286
Science. 1993 Dec 24;262(5142):2039-42
pubmed: 8266101
Nucleic Acids Res. 2020 Jan 10;48(1):130-140
pubmed: 31713617
Nat Commun. 2018 Aug 2;9(1):3025
pubmed: 30072689
Nat Med. 2004 Apr;10(4):396-401
pubmed: 15034573
J Neurosci. 1997 Jun 15;17(12):4545-51
pubmed: 9169515
Lancet Neurol. 2009 Jun;8(6):537-44
pubmed: 19427269
Nat Med. 2003 Dec;9(12):1533-7
pubmed: 14608378
Lancet Neurol. 2009 Dec;8(12):1103-10
pubmed: 19818690
Hum Mol Genet. 1998 Mar;7(3):449-58
pubmed: 9467003
Neurobiol Dis. 2016 Sep;93:201-14
pubmed: 27215377
Ann Neurol. 2002 Feb;51(2):190-201
pubmed: 11835375
Glia. 1998 Sep;24(1):8-20
pubmed: 9700485
Proc Natl Acad Sci U S A. 1991 Oct 1;88(19):8410-4
pubmed: 1717979
Front Neurosci. 2014 Jun 13;8:151
pubmed: 24982612
Free Radic Biol Med. 2020 Dec;161:246-262
pubmed: 32980538
Brain Res. 2020 Jan 15;1727:146533
pubmed: 31669284
Orphanet J Rare Dis. 2014 Dec 18;9:199
pubmed: 25519680
J Neurochem. 1998 Oct;71(4):1765-8
pubmed: 9751213
Neuron. 1996 May;16(5):1049-60
pubmed: 8630243
Hum Mol Genet. 2018 Apr 15;27(8):1460-1473
pubmed: 29462293
Sci Rep. 2019 Aug 6;9(1):11392
pubmed: 31388039
Nat Genet. 1992 Dec;2(4):288-91
pubmed: 1303281
Gene. 2000 May 2;248(1-2):223-31
pubmed: 10806367
Science. 2002 Jan 18;295(5554):499-502
pubmed: 11799245
J Clin Invest. 2018 Jan 2;128(1):359-368
pubmed: 29202483
Semin Cell Biol. 1992 Feb;3(1):3-16
pubmed: 1320430
Clin Genet. 1974;6(2):98-118
pubmed: 4430158
J Cell Biol. 2004 May 24;165(4):565-73
pubmed: 15148307
Nat Genet. 1995 Nov;11(3):281-6
pubmed: 7581451
Ann Clin Transl Neurol. 2018 Mar 10;5(4):445-455
pubmed: 29687021
J Neurol Neurosurg Psychiatry. 2015 Aug;86(8):873-8
pubmed: 25430934
Clin Neurophysiol. 2020 Oct;131(10):2440-2451
pubmed: 32829291
Traffic. 2004 Nov;5(11):821-37
pubmed: 15479448
Hum Mol Genet. 2019 Jan 1;28(1):124-132
pubmed: 30239779
Neuromuscul Disord. 1991;1(2):93-7
pubmed: 1822787
Hum Mol Genet. 1996 May;5(5):563-9
pubmed: 8733121
Nature. 1992 Mar 19;356(6366):241-4
pubmed: 1552943