Antisense therapy in a rat model of Alexander disease reverses GFAP pathology, white matter deficits, and motor impairment.

Alexander Disease / genetics Animals Astrocytes / metabolism Glial Fibrillary Acidic Protein / genetics Gliosis / pathology Motor Disorders / metabolism Mutation / genetics Rats White Matter / pathology

Journal

Science translational medicine
ISSN: 1946-6242
Titre abrégé: Sci Transl Med
Pays: United States
ID NLM: 101505086

Informations de publication

Date de publication:
17 11 2021
Historique:
entrez: 17 11 2021
pubmed: 18 11 2021
medline: 31 3 2022
Statut: ppublish

Résumé

Alexander disease (AxD) is a devastating leukodystrophy caused by gain-of-function mutations in

Identifiants

DOI: 10.1126/scitranslmed.abg4711 PMID: 34788075 PMC: PMC8730534
pubmed: 34788075
doi: 10.1126/scitranslmed.abg4711
pmc: PMC8730534
mid: NIHMS1761378
doi:

Substances chimiques

GFAP protein, rat 0
Glial Fibrillary Acidic Protein 0

Types de publication

Journal Article Research Support, N.I.H., Extramural Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

eabg4711

Subventions

Organisme : NICHD NIH HHS
ID : P30 HD003352
Pays : United States
Organisme : NICHD NIH HHS
ID : P01 HD076892
Pays : United States
Organisme : NINDS NIH HHS
ID : K08 NS109344
Pays : United States
Organisme : NINDS NIH HHS
ID : R01 NS110719
Pays : United States
Organisme : NICHD NIH HHS
ID : P50 HD103526
Pays : United States
Organisme : NICHD NIH HHS
ID : U54 HD090256
Pays : United States

Références

Neurology. 2011 Sep 27;77(13):1287-94
pubmed: 21917775
ASN Neuro. 2013 Nov 19;5(5):e00125
pubmed: 24102621
J Neurosci Res. 1993 Oct 1;36(2):183-90
pubmed: 8263971
J Neurosci. 2012 Apr 11;32(15):5017-23
pubmed: 22496548
Neuroscience. 2008 Apr 22;153(1):108-19
pubmed: 18358624
Cold Spring Harb Perspect Biol. 2014 Nov 07;7(2):a020420
pubmed: 25380660
EMBO J. 1995 Apr 18;14(8):1590-8
pubmed: 7737111
Acta Neuropathol. 2001 Jul;102(1):1-10
pubmed: 11547943
Proc Natl Acad Sci U S A. 1996 Jun 25;93(13):6361-6
pubmed: 8692820
Nature. 2014 May 8;509(7499):189-94
pubmed: 24776795
Ann N Y Acad Sci. 2016 Feb;1366(1):20-39
pubmed: 26096370
Exp Cell Res. 2009 Apr 15;315(7):1260-72
pubmed: 19146851
Clin Sci (Lond). 2017 Aug 10;131(17):2257-2274
pubmed: 28798076
Nat Neurosci. 2015 Jul;18(7):942-52
pubmed: 26108722
Brain Behav. 2018 Oct;8(10):e01110
pubmed: 30240148
ASN Neuro. 2013;5(1):e00109
pubmed: 23432455
Nature. 2017 Jan 26;541(7638):481-487
pubmed: 28099414
Exp Cell Res. 2011 Oct 1;317(16):2252-66
pubmed: 21756903
Nature. 2020 Sep;585(7825):397-403
pubmed: 32610343
AJNR Am J Neuroradiol. 2001 Mar;22(3):541-52
pubmed: 11237983
Am J Pathol. 2002 Jan;160(1):255-63
pubmed: 11786419
J Clin Invest. 2012 Nov;122(11):3900-13
pubmed: 23064363
J Neurosci. 2011 Mar 16;31(11):4166-77
pubmed: 21411657
Nat Commun. 2018 May 15;9(1):1899
pubmed: 29765022
Brain Res. 1997 Feb 21;749(1):82-7
pubmed: 9070630
J Neuroendocrinol. 2019 May;31(5):e12671
pubmed: 30561077
Sci Rep. 2016 Mar 30;6:23673
pubmed: 27026049
Glia. 2007 Jul;55(9):954-65
pubmed: 17487878
Neuroscience. 2004;129(4):905-13
pubmed: 15561407
J Neurosci. 2013 Nov 20;33(47):18698-706
pubmed: 24259590
ASN Neuro. 2020 Jan-Dec;12:1759091420949680
pubmed: 32811163
Neurology. 2006 Feb 28;66(4):494-8
pubmed: 16505300
J Neurosci. 2006 Oct 25;26(43):11162-73
pubmed: 17065456
Neuron. 1996 Oct;17(4):607-15
pubmed: 8893019
Hum Mol Genet. 2005 Aug 15;14(16):2443-58
pubmed: 16014634
J Neurosci Methods. 2002 Apr 15;115(2):169-79
pubmed: 11992668
Front Neurosci. 2020 Feb 06;14:82
pubmed: 32116525
Nature. 2019 Sep;573(7772):130-134
pubmed: 31413369
J Biol Chem. 2017 Apr 7;292(14):5814-5824
pubmed: 28223355
Elife. 2019 Nov 04;8:
pubmed: 31682229
Acta Neuropathol. 1991;81(5):503-9
pubmed: 1650112
J Vis Exp. 2009 Jun 12;(28):
pubmed: 19525918
Ann Neurol. 2018 Jan;83(1):27-39
pubmed: 29226998
J Neurosci Methods. 1996 Nov;69(2):123-36
pubmed: 8946315
Neuron. 1995 Jan;14(1):29-41
pubmed: 7826639
Bioessays. 2016 Mar;38(3):232-43
pubmed: 26763143
Neuron. 2018 Apr 18;98(2):306-319.e7
pubmed: 29606582
Neurology. 1976 Jul;26(7):607-14
pubmed: 180453
Sci Rep. 2018 Feb 2;8(1):2246
pubmed: 29396480
Brain Res. 2013 May 28;1512:97-105
pubmed: 23548599
Front Cell Neurosci. 2020 Feb 27;14:30
pubmed: 32180706
Am J Pathol. 1998 Feb;152(2):391-8
pubmed: 9466565
Neurology. 2014 Jan 7;82(1):49-56
pubmed: 24306001
Biochemistry. 1999 Aug 24;38(34):11156-63
pubmed: 10460172
Am J Pathol. 1991 Apr;138(4):875-85
pubmed: 1707236
PLoS One. 2013 Apr 10;8(4):e60537
pubmed: 23593236
Annu Rev Pathol. 2017 Jan 24;12:131-152
pubmed: 28135564
Cell Stem Cell. 2018 Aug 02;23(2):239-251.e6
pubmed: 30075130
Neurochem Res. 2010 Jan;35(1):114-21
pubmed: 19655246
J Microsc. 2006 Dec;224(Pt 3):213-32
pubmed: 17210054
J Neuropathol Exp Neurol. 2010 Apr;69(4):335-45
pubmed: 20448479
Annu Rev Neurosci. 2019 Jul 8;42:385-406
pubmed: 31283897
Brain Res. 2012 Apr 23;1450:102-15
pubmed: 22436850
FASEB J. 2004 Aug;18(11):1291-3
pubmed: 15208268
J Cell Sci. 2005 May 1;118(Pt 9):2057-65
pubmed: 15840648
Neuroscience. 2014 Sep 12;276:161-73
pubmed: 24231735
Biochim Biophys Acta. 2006 Aug;1758(8):1085-93
pubmed: 16564496
J Proteome Res. 2016 Jul 1;15(7):2265-82
pubmed: 27193225
Nat Genet. 2001 Jan;27(1):117-20
pubmed: 11138011
Sci Rep. 2015 Dec 09;5:17956
pubmed: 26648273
DNA Cell Biol. 2020 Feb;39(2):154-158
pubmed: 31821021
Pharmacol Biochem Behav. 1993 Aug;45(4):817-21
pubmed: 8415820
Nat Neurosci. 2014 Jul;17(7):908-10
pubmed: 24880214

Auteurs

Tracy L Hagemann (TL)

Waisman Center, University of Wisconsin-Madison, Madison, WI 53705, USA.
ORCID: 0000-0002-0237-9267

Berit Powers (B)

Ionis Pharmaceuticals, Carlsbad, CA 92010, USA.

Ni-Hsuan Lin (NH)

Institute of Molecular Medicine, College of Life Sciences, National Tsing Hua University, Hsinchu 30013, Taiwan.
ORCID: 0000-0003-4524-3615

Ahmed F Mohamed (AF)

Waisman Center, University of Wisconsin-Madison, Madison, WI 53705, USA.

Katerina L Dague (KL)

Waisman Center, University of Wisconsin-Madison, Madison, WI 53705, USA.
ORCID: 0000-0001-5613-9018

Seth C Hannah (SC)

Waisman Center, University of Wisconsin-Madison, Madison, WI 53705, USA.

Gemma Bachmann (G)

Ionis Pharmaceuticals, Carlsbad, CA 92010, USA.
ORCID: 0000-0002-3974-191X

Curt Mazur (C)

Ionis Pharmaceuticals, Carlsbad, CA 92010, USA.
ORCID: 0000-0003-3485-9086

Frank Rigo (F)

Ionis Pharmaceuticals, Carlsbad, CA 92010, USA.

Abby L Olsen (AL)

Department of Neurology, Brigham and Women's Hospital, Harvard Medical School, Boston, MA 02115, USA.
ORCID: 0000-0002-7680-1758

Mel B Feany (MB)

Department of Pathology, Brigham and Women's Hospital, Harvard Medical School, Boston, MA 02115, USA.
ORCID: 0000-0003-0315-7970

Ming-Der Perng (MD)

Institute of Molecular Medicine, College of Life Sciences, National Tsing Hua University, Hsinchu 30013, Taiwan.
ORCID: 0000-0002-1884-3714

Robert F Berman (RF)

Department of Neurological Surgery and M.I.N.D Institute, University of California, Davis, Davis, CA 95616, USA.
ORCID: 0000-0001-8530-3147

Albee Messing (A)

Waisman Center, University of Wisconsin-Madison, Madison, WI 53705, USA.
Department of Comparative Biosciences, School of Veterinary Medicine, University of Wisconsin-Madison, Madison, WI 53705, USA.
ORCID: 0000-0001-6049-0521

Articles similaires

Evaluating the efficacy of telesurgery with dual console SSI Mantra Surgical Robotic System: experiment on animal model and clinical trials.

Sudhir Prem Srivastava, Vishwajyoti Pascual Srivastava, Avinesh Singh et al.
1.00
Robotic Surgical Procedures Animals Humans Telemedicine Models, Animal

Odour generalisation and detection dog training.

Lyn Caldicott, Thomas W Pike, Helen E Zulch et al.
1.00
Animals Odorants Dogs Generalization, Psychological Smell

FBXO22 inhibits colitis and colorectal carcinogenesis by regulating the degradation of the S2448-phosphorylated form of mTOR.

Minle Li, Xuan Chen, Pengfei Qu et al.
1.00
Animals TOR Serine-Threonine Kinases Colorectal Neoplasms Colitis Mice

Use of organic material provided by an automatic enrichment device by weaner pigs and its influence on tail lesions.

Philipp Heseker, Jeanette Probst, Stefanie Ammer et al.
1.00
Animals Tail Swine Behavior, Animal Animal Husbandry

Classifications MeSH

questionsmedicales.fr › Maladies métaboliques et nutritionnelles › Maladies métaboliques › Erreurs innées du métabolisme › Encéphalopathies métaboliques congénitales › Maladies démyélinisantes héréditaires du système nerveux central › Maladie d'Alexander › Antisense therapy in a rat model of Alexander...
questionsmedicales.fr › Eucaryotes › Animaux › Antisense therapy in a rat model of Alexander...
questionsmedicales.fr › Cellules › Névroglie › Astrocytes › Antisense therapy in a rat model of Alexander...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Protéines du cytosquelette › Protéines de filaments intermédiaires › Protéine gliofibrillaire acide › Antisense therapy in a rat model of Alexander...
questionsmedicales.fr › États, signes et symptômes pathologiques › Processus pathologiques › Gliose › Antisense therapy in a rat model of Alexander...
questionsmedicales.fr › Troubles mentaux › Troubles moteurs › Antisense therapy in a rat model of Alexander...
questionsmedicales.fr › Phénomènes génétiques › Variation génétique › Mutation › Antisense therapy in a rat model of Alexander...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Rodentia › Muridae › Murinae › Rats › Antisense therapy in a rat model of Alexander...
questionsmedicales.fr › Système nerveux › Système nerveux central › Moelle spinale › Substance blanche › Antisense therapy in a rat model of Alexander...