Antisense Oligonucleotide Therapies for Neurodegenerative Diseases.
Huntington's disease
RNA
amyotrophic lateral sclerosis
antisense oligonucleotides
spinal muscular atrophy
Journal
Annual review of neuroscience
ISSN: 1545-4126
Titre abrégé: Annu Rev Neurosci
Pays: United States
ID NLM: 7804039
Informations de publication
Date de publication:
08 07 2019
08 07 2019
Historique:
entrez:
9
7
2019
pubmed:
10
7
2019
medline:
12
9
2020
Statut:
ppublish
Résumé
Antisense oligonucleotides represent a novel therapeutic platform for the discovery of medicines that have the potential to treat most neurodegenerative diseases. Antisense drugs are currently in development for the treatment of amyotrophic lateral sclerosis, Huntington's disease, and Alzheimer's disease, and multiple research programs are underway for additional neurodegenerative diseases. One antisense drug, nusinersen, has been approved for the treatment of spinal muscular atrophy. Importantly, nusinersen improves disease symptoms when administered to symptomatic patients rather than just slowing the progression of the disease. In addition to the benefit to spinal muscular atrophy patients, there are discoveries from nusinersen that can be applied to other neurological diseases, including method of delivery, doses, tolerability of intrathecally delivered antisense drugs, and the biodistribution of intrathecal dosed antisense drugs. Based in part on the early success of nusinersen, antisense drugs hold great promise as a therapeutic platform for the treatment of neurological diseases.
Identifiants
pubmed: 31283897
doi: 10.1146/annurev-neuro-070918-050501
pmc: PMC7427431
mid: NIHMS1612328
doi:
Substances chimiques
Oligonucleotides
0
Oligonucleotides, Antisense
0
nusinersen
5Z9SP3X666
Types de publication
Journal Article
Research Support, N.I.H., Extramural
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
385-406Subventions
Organisme : NIGMS NIH HHS
ID : R37 GM042699
Pays : United States
Organisme : NINDS NIH HHS
ID : R01 NS027036
Pays : United States
Organisme : NIGMS NIH HHS
ID : R01 GM042699
Pays : United States
Organisme : NCI NIH HHS
ID : P01 CA013106
Pays : United States
Organisme : NINDS NIH HHS
ID : R01 NS112503
Pays : United States
Références
Nat Rev Dis Primers. 2017 Oct 05;3:17071
pubmed: 28980624
Neurology. 2012 Oct 30;79(18):1889-97
pubmed: 23077013
Am J Hum Genet. 1999 May;64(5):1340-56
pubmed: 10205265
JAMA Neurol. 2015 Mar;72(3):316-24
pubmed: 25580592
Neuron. 2016 Nov 23;92(4):796-812
pubmed: 27974162
Neuron. 2011 Oct 20;72(2):257-68
pubmed: 21944779
Nat Med. 2005 Apr;11(4):423-8
pubmed: 15768028
Am J Hum Genet. 2006 Jan;78(1):63-77
pubmed: 16385450
Nat Genet. 2003 Aug;34(4):460-3
pubmed: 12833158
J Neurosci. 2015 Mar 11;35(10):4440-51
pubmed: 25762686
Cell. 2012 Aug 31;150(5):895-908
pubmed: 22939619
Nature. 2017 Apr 20;544(7650):362-366
pubmed: 28405024
N Engl J Med. 2018 Feb 15;378(7):625-635
pubmed: 29443664
J Pediatr Orthop. 2018 Nov/Dec;38(10):e610-e617
pubmed: 30134351
Adv Drug Deliv Rev. 2015 Jun 29;87:46-51
pubmed: 25666165
Sci Transl Med. 2017 Jan 25;9(374):
pubmed: 28123067
Sci Transl Med. 2013 Oct 23;5(208):208ra149
pubmed: 24154603
Neurotherapeutics. 2013 Jul;10(3):459-72
pubmed: 23549647
Neurotherapeutics. 2008 Oct;5(4):499-506
pubmed: 19019300
Neurobiol Dis. 2012 Jan;45(1):356-61
pubmed: 21889984
Adv Exp Med Biol. 2018;1049:175-195
pubmed: 29427103
Mol Ther. 2015 Nov;23(11):1759-1771
pubmed: 26201449
Nat Genet. 2002 Apr;30(4):377-84
pubmed: 11925564
Nat Rev Drug Discov. 2015 Nov;14(11):759-80
pubmed: 26338154
Mol Ther. 2014 Dec;22(12):2093-106
pubmed: 25101598
Lancet Neurol. 2012 Apr;11(4):323-30
pubmed: 22406228
PLoS One. 2011;6(9):e24308
pubmed: 21909428
Nucleic Acids Res. 2017 Sep 19;45(16):9528-9546
pubmed: 28934489
J Pharmacol Exp Ther. 2000 Feb;292(2):489-96
pubmed: 10640284
Proc Natl Acad Sci U S A. 2007 Oct 23;104(43):17204-9
pubmed: 17940007
PLoS Biol. 2007 Apr;5(4):e73
pubmed: 17355180
Hum Genet. 2006 May;119(4):422-8
pubmed: 16508748
Neuron. 2016 May 4;90(3):535-50
pubmed: 27112497
Mol Ther. 2011 Dec;19(12):2178-85
pubmed: 21971427
Nature. 1993 Mar 4;362(6415):59-62
pubmed: 8446170
Science. 2014 Sep 5;345(6201):1139-45
pubmed: 25081482
Mol Ther. 2011 Dec;19(12):2152-62
pubmed: 22031240
Cell. 1995 Jan 13;80(1):155-65
pubmed: 7813012
Lancet. 2017 Dec 17;388(10063):3017-3026
pubmed: 27939059
Bioinformatics. 2013 Feb 15;29(4):499-503
pubmed: 23329412
Genes Dev. 2012 Aug 15;26(16):1874-84
pubmed: 22895255
Hum Mol Genet. 1999 Jul;8(7):1177-83
pubmed: 10369862
Mol Ther. 2009 Jun;17(6):1053-63
pubmed: 19240687
Biochemistry. 2010 Nov 30;49(47):10166-78
pubmed: 21028906
N Engl J Med. 2018 Jan 25;378(4):391-392
pubmed: 29365295
Annu Rev Neurosci. 2004;27:723-49
pubmed: 15217349
Hum Mol Genet. 2007 Dec 15;16(24):3149-59
pubmed: 17884807
JAMA Neurol. 2018 May 1;75(5):548-556
pubmed: 29435558
Curr Biol. 2009 May 12;19(9):774-8
pubmed: 19361997
Neurology. 2018 Oct 2;91(14):e1295-e1306
pubmed: 30217935
Proc Natl Acad Sci U S A. 2005 Apr 19;102(16):5820-5
pubmed: 15811941
Sci Transl Med. 2018 Oct 3;10(461):
pubmed: 30282695
J Pharmacol Exp Ther. 2001 Jun;297(3):1113-21
pubmed: 11356936
J Clin Invest. 2011 Feb;121(2):476-83
pubmed: 21285520
N Engl J Med. 2017 Nov 2;377(18):1723-1732
pubmed: 29091570
Acta Neuropathol. 2013 Dec;126(6):829-44
pubmed: 24129584
Neuron. 2013 Oct 16;80(2):415-28
pubmed: 24139042
J Pharmacol Exp Ther. 2001 Mar;296(3):890-7
pubmed: 11181921
Clin Genet. 2009 Aug;76(2):168-78
pubmed: 19780763
Nucleic Acids Res. 2001 Mar 15;29(6):1293-9
pubmed: 11238995
Proc Natl Acad Sci U S A. 2013 Nov 19;110(47):E4530-9
pubmed: 24170860
Proc Natl Acad Sci U S A. 2011 Jan 4;108(1):260-5
pubmed: 21173221
Neuron. 2011 Oct 20;72(2):245-56
pubmed: 21944778
Mol Ther. 2018 Sep 5;26(9):2163-2177
pubmed: 30007561
Genes Dev. 2015 Feb 1;29(3):288-97
pubmed: 25583329
Neurology. 2019 May 21;92(21):e2492-e2506
pubmed: 31019106
Neuron. 2012 Jun 21;74(6):1031-44
pubmed: 22726834
Mol Cell Biol. 2006 Feb;26(4):1333-46
pubmed: 16449646
Nat Rev Neurol. 2011 Oct 11;7(11):603-15
pubmed: 21989245
Am J Hum Genet. 2008 Apr;82(4):834-48
pubmed: 18371932
Cell. 2009 Feb 20;136(4):777-93
pubmed: 19239895
Nucleic Acids Res. 2013 Nov;41(21):9634-50
pubmed: 23963702
J Child Neurol. 2007 Aug;22(8):979-89
pubmed: 17761653
Nat Rev Drug Discov. 2012 Jan 20;11(2):125-40
pubmed: 22262036
Neurology. 2014 Aug 26;83(9):810-7
pubmed: 25080519
Hum Mol Genet. 2016 Apr 15;25(R1):R53-64
pubmed: 26503961
J Pharmacol Exp Ther. 2014 Jul;350(1):46-55
pubmed: 24784568
J Clin Invest. 2018 Aug 1;128(8):3558-3567
pubmed: 30010620
Annu Rev Pharmacol Toxicol. 2017 Jan 6;57:81-105
pubmed: 27732800
Sci Transl Med. 2011 Mar 2;3(72):72ra18
pubmed: 21368223
Neuron. 2013 Feb 20;77(4):639-46
pubmed: 23415312
Mol Ther Nucleic Acids. 2015 Jun 30;4:e245
pubmed: 26125484
Science. 1995 Jul 21;269(5222):407-10
pubmed: 7618107
Nat Biotechnol. 2016 Aug;34(8):875-80
pubmed: 27398791
Neuromuscul Disord. 2008 Jul;18(7):541-4
pubmed: 18579378
Mol Ther. 2013 Dec;21(12):2148-59
pubmed: 24008656
Nature. 2011 Oct 05;478(7367):123-6
pubmed: 21979052
Nat Rev Neurol. 2018 Sep;14(9):544-558
pubmed: 30120348
Hum Mol Genet. 2017 Oct 15;26(20):4093-4094
pubmed: 28973528
Hum Mol Genet. 2010 Oct 15;19(20):3895-905
pubmed: 20639395
Hum Mol Genet. 2010 Oct 15;19(20):3906-18
pubmed: 20693262
Science. 2014 Sep 5;345(6201):1192-1194
pubmed: 25103406
Hum Mol Genet. 2011 Oct 1;20(19):3811-21
pubmed: 21729883
Annu Rev Pharmacol Toxicol. 2010;50:259-93
pubmed: 20055705
Nat Med. 2014 May;20(5):536-41
pubmed: 24784230
Neurology. 2016 Mar 8;86(10):890-7
pubmed: 26865511
Nat Biotechnol. 2016 Feb;34(2):164-6
pubmed: 26655495
Nature. 2010 Aug 26;466(7310):1069-75
pubmed: 20740007
Nat Chem Biol. 2015 Jul;11(7):511-7
pubmed: 26030728
JAMA Neurol. 2018 May 1;75(5):536-538
pubmed: 29435570
Physiol Rev. 2010 Jul;90(3):905-81
pubmed: 20664076
Nat Struct Biol. 2003 Feb;10(2):120-5
pubmed: 12524529
Gene Ther. 2017 Sep;24(9):527-528
pubmed: 28737743
PLoS One. 2014 Sep 10;9(9):e107434
pubmed: 25207939
Nat Biotechnol. 2009 May;27(5):478-84
pubmed: 19412185
Sci Transl Med. 2017 Mar 29;9(383):
pubmed: 28356511
J Neurosci. 2013 Jul 31;33(31):12887-97
pubmed: 23904623
Cell Metab. 2006 Feb;3(2):87-98
pubmed: 16459310
Cell. 1993 Mar 26;72(6):971-83
pubmed: 8458085
Science. 2016 Mar 18;351(6279):1324-9
pubmed: 26989253
J Clin Invest. 2006 Aug;116(8):2290-6
pubmed: 16878173
Science. 2014 Aug 8;345(6197):688-93
pubmed: 25104390
Genes Dev. 2010 Aug 1;24(15):1634-44
pubmed: 20624852
Nat Neurosci. 2016 Aug;19(8):1085-92
pubmed: 27322420
Proc Natl Acad Sci U S A. 1999 May 25;96(11):6307-11
pubmed: 10339583
Nucleic Acids Res. 2012 Dec;40(22):11270-80
pubmed: 23042244
Eur J Hum Genet. 2012 Jan;20(1):27-32
pubmed: 21811307
Nat Genet. 1995 Oct;11(2):155-63
pubmed: 7550343
Med Genet. 2018;30(2):252-258
pubmed: 30220791
Nature. 2017 Apr 20;544(7650):367-371
pubmed: 28405022
Cell Metab. 2018 Apr 3;27(4):714-739
pubmed: 29617640
Neuropathol Appl Neurobiol. 1996 Oct;22(5):373-87
pubmed: 8930947
Curr Opin Genet Dev. 2017 Jun;44:117-124
pubmed: 28319737
Proc Natl Acad Sci U S A. 2016 Mar 22;113(12):3359-64
pubmed: 26951659
Nucleic Acid Ther. 2017 Apr;27(2):70-77
pubmed: 28080221
Lancet Neurol. 2013 May;12(5):435-42
pubmed: 23541756
Hum Genet. 2001 Mar;108(3):255-66
pubmed: 11354640
Science. 2006 Oct 6;314(5796):130-3
pubmed: 17023659
Neuron. 2012 Feb 23;73(4):685-97
pubmed: 22365544
PLoS Genet. 2017 Jul 17;13(7):e1006846
pubmed: 28715425
Nature. 2004 Sep 16;431(7006):343-9
pubmed: 15372041
Am J Hum Genet. 2009 Sep;85(3):408-13
pubmed: 19716110
Arch Neurol. 2011 Jun;68(6):779-86
pubmed: 21320981