AAV2/9-mediated overexpression of MIF inhibits SOD1 misfolding, delays disease onset, and extends survival in mouse models of ALS.
Age of Onset
Amyotrophic Lateral Sclerosis
/ genetics
Animals
Cells, Cultured
Dependovirus
Disease Models, Animal
Genetic Therapy
/ methods
Genetic Vectors
/ administration & dosage
Humans
Injections, Spinal
Intramolecular Oxidoreductases
/ genetics
Macrophage Migration-Inhibitory Factors
/ genetics
Mice
Mice, Transgenic
Motor Neurons
/ pathology
Mutation
Parvovirinae
/ genetics
Primary Cell Culture
Protein Aggregates
Protein Folding
Spinal Cord
/ cytology
Superoxide Dismutase-1
/ genetics
Time Factors
Transduction, Genetic
/ methods
Treatment Outcome
AAV
ALS
MIF
misfolded SOD1
mutant SOD1
Journal
Proceedings of the National Academy of Sciences of the United States of America
ISSN: 1091-6490
Titre abrégé: Proc Natl Acad Sci U S A
Pays: United States
ID NLM: 7505876
Informations de publication
Date de publication:
16 07 2019
16 07 2019
Historique:
pubmed:
3
7
2019
medline:
3
4
2020
entrez:
3
7
2019
Statut:
ppublish
Résumé
Mutations in superoxide dismutase (SOD1) cause amyotrophic lateral sclerosis (ALS), a neurodegenerative disease characterized by the loss of upper and lower motor neurons. Transgenic mice that overexpress mutant SOD1 develop paralysis and accumulate misfolded SOD1 onto the cytoplasmic faces of intracellular organelles, including mitochondria and endoplasmic reticulum (ER). Recently, macrophage migration inhibitory factor (MIF) was shown to directly inhibit mutant SOD1 misfolding and binding to intracellular membranes. In addition, complete elimination of endogenous MIF accelerated disease onset and late disease progression, as well as shortened the lifespan of mutant SOD1 mice with higher amounts of misfolded SOD1 detected within the spinal cord. Based on these findings, we used adeno-associated viral (AAV) vectors to overexpress MIF in the spinal cord of mutant SOD1
Identifiants
pubmed: 31262807
pii: 1904665116
doi: 10.1073/pnas.1904665116
pmc: PMC6642395
doi:
Substances chimiques
Macrophage Migration-Inhibitory Factors
0
Protein Aggregates
0
SOD1 protein, human
0
Superoxide Dismutase-1
EC 1.15.1.1
Intramolecular Oxidoreductases
EC 5.3.-
MIF protein, human
EC 5.3.2.1
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
14755-14760Déclaration de conflit d'intérêts
The authors declare no conflict of interest.
Références
J Biol Chem. 2002 Aug 16;277(33):29626-33
pubmed: 12050154
Acta Neuropathol. 2004 Jul;108(1):31-6
pubmed: 15067555
Neuron. 2004 Jul 8;43(1):5-17
pubmed: 15233913
J Neurol Sci. 2004 Oct 15;225(1-2):19-25
pubmed: 15465081
J Neurosci. 2005 Mar 9;25(10):2463-70
pubmed: 15758154
J Neurochem. 2005 May;93(4):875-82
pubmed: 15857390
Int J Biochem Cell Biol. 2006 Jan;38(1):43-55
pubmed: 16099194
Mol Ther. 2006 Mar;13(3):463-83
pubmed: 16412695
Neuroscience. 2006;139(3):1117-28
pubmed: 16504406
J Neurosci. 2006 Apr 19;26(16):4147-54
pubmed: 16624935
Proc Natl Acad Sci U S A. 2006 May 2;103(18):7142-7
pubmed: 16636275
Science. 2006 Jun 2;312(5778):1389-92
pubmed: 16741123
Nat Med. 2007 Jun;13(6):754-9
pubmed: 17486090
Ann Neurol. 2007 Dec;62(6):553-9
pubmed: 18074357
Nat Neurosci. 2008 Mar;11(3):251-3
pubmed: 18246065
Neurobiol Dis. 2008 Apr;30(1):42-55
pubmed: 18255302
Science. 2008 Feb 15;319(5865):916-9
pubmed: 18276881
Prog Neurobiol. 2008 May;85(1):94-134
pubmed: 18282652
Proc Natl Acad Sci U S A. 2008 Mar 11;105(10):4022-7
pubmed: 18296640
Genes Dev. 2008 Jun 1;22(11):1451-64
pubmed: 18519638
J Neurochem. 2008 Sep;106(5):2170-83
pubmed: 18624915
Mol Cell Biol. 2009 Apr;29(7):1922-32
pubmed: 19188446
J Immunol. 2009 Jun 1;182(11):6896-906
pubmed: 19454686
Hum Mol Genet. 2009 Sep 1;18(17):3217-26
pubmed: 19483195
Curr Opin Mol Ther. 2009 Aug;11(4):442-7
pubmed: 19649989
Ann Neurol. 2009 Jul;66(1):75-80
pubmed: 19670443
J Cell Biol. 2009 Dec 14;187(6):761-72
pubmed: 19951898
J Neurochem. 2010 Jun;113(5):1092-100
pubmed: 20067574
Acta Neuropathol. 2010 Mar;119(3):335-44
pubmed: 20111867
J Neurochem. 2010 Jun;113(5):1188-99
pubmed: 20345765
Hum Mol Genet. 2010 Aug 1;19(15):2974-86
pubmed: 20460269
PLoS One. 2010 Jul 14;5(7):e11552
pubmed: 20644736
Arch Biochem Biophys. 2010 Nov 15;503(2):175-82
pubmed: 20682279
Neuron. 2010 Aug 26;67(4):575-87
pubmed: 20797535
Nat Neurosci. 2010 Nov;13(11):1396-403
pubmed: 20953194
Proc Natl Acad Sci U S A. 2010 Dec 7;107(49):21146-51
pubmed: 21078990
Neurochem Int. 2011 Feb;58(3):344-53
pubmed: 21185345
J Neurochem. 2011 Apr;117(1):91-9
pubmed: 21226712
Acta Neuropathol. 2011 May;121(5):623-34
pubmed: 21287393
PLoS One. 2011;6(7):e22031
pubmed: 21779368
Curr Opin Neurobiol. 2011 Dec;21(6):904-19
pubmed: 21813273
Proc Natl Acad Sci U S A. 2011 Sep 27;108(39):16398-403
pubmed: 21930926
Blood. 2012 Mar 29;119(13):3038-41
pubmed: 22271447
Proc Natl Acad Sci U S A. 2012 Mar 27;109(13):5074-9
pubmed: 22416121
PLoS One. 2012;7(4):e35050
pubmed: 22493728
Ann Neurol. 2012 Nov;72(5):739-49
pubmed: 23280792
Proteins. 2013 Aug;81(8):1285-303
pubmed: 23508986
Arch Phys Med Rehabil. 2013 Aug;94(8):1498-507
pubmed: 23618747
Mol Ther. 2013 Dec;21(12):2148-59
pubmed: 24008656
Mol Cell. 2013 Oct 10;52(1):75-86
pubmed: 24076220
Proc Natl Acad Sci U S A. 2014 Mar 4;111(9):3620-5
pubmed: 24550511
Acta Neuropathol Commun. 2014 May 14;2:55
pubmed: 24887207
Arch Phys Med Rehabil. 2015 Apr;96(4):633-44
pubmed: 25461821
J Neurochem. 2015 May;133(3):452-64
pubmed: 25557022
Hum Gene Ther Clin Dev. 2015 Mar;26(1):5-14
pubmed: 25675273
Neuron. 2015 Apr 8;86(1):218-32
pubmed: 25801706
J Neuroinflammation. 2015 Jun 24;12:123
pubmed: 26104759
Proc Natl Acad Sci U S A. 2016 May 10;113(19):5424-8
pubmed: 27114530
Neurobiol Dis. 2016 Nov;95:168-78
pubmed: 27461051
Proc Natl Acad Sci U S A. 2016 Sep 6;113(36):10198-203
pubmed: 27551074
Acta Neuropathol. 2017 Jul;134(1):97-111
pubmed: 28247063
Hum Gene Ther Clin Dev. 2017 Jun;28(2):74-79
pubmed: 28279081
Exp Neurol. 2018 Mar;301(Pt B):83-91
pubmed: 28679106
ACS Chem Neurosci. 2017 Oct 18;8(10):2225-2234
pubmed: 28715630
N Engl J Med. 2017 Nov 2;377(18):1713-1722
pubmed: 29091557
Cell Death Dis. 2018 Jan 25;9(2):107
pubmed: 29371591
Brain Res Bull. 2018 May;139:203-210
pubmed: 29499331
Science. 1994 Jun 17;264(5166):1772-5
pubmed: 8209258
Nature. 1993 Mar 4;362(6415):59-62
pubmed: 8446170
J Mol Biol. 1998 Jul 3;280(1):85-102
pubmed: 9653033