Upregulation of the ESCRT pathway and multivesicular bodies accelerates degradation of proteins associated with neurodegeneration.

ESCRT Huntington NCT-504 Neurodegeneration Tau huntingtin multivesicular bodies (MVB)

Journal

Autophagy reports
ISSN: 2769-4127
Titre abrégé: Autophagy Rep
Pays: United States
ID NLM: 9918383885906676

Informations de publication

Date de publication:
2023
Historique:
medline: 18 4 2023
entrez: 17 4 2023
pubmed: 18 4 2023
Statut: ppublish

Résumé

Many neurodegenerative diseases, including Huntington's disease (HD) and Alzheimer's disease (AD), occur due to an accumulation of aggregation-prone proteins, which results in neuronal death. Studies in animal and cell models show that reducing the levels of these proteins mitigates disease phenotypes. We previously reported a small molecule, NCT-504, which reduces cellular levels of mutant huntingtin (mHTT) in patient fibroblasts as well as mouse striatal and cortical neurons from an Hdh

Identifiants

pubmed: 37064812
doi: 10.1080/27694127.2023.2166722
pmc: PMC10101321
mid: NIHMS1884781
pii:
doi:

Types de publication

Journal Article

Langues

eng

Subventions

Organisme : NINDS NIH HHS
ID : R01 NS099340
Pays : United States
Organisme : NINDS NIH HHS
ID : R35 NS122302
Pays : United States

Références

Exp Mol Med. 2015 Mar 13;47:e147
pubmed: 25766616
Bioorg Med Chem. 2020 Jan 1;28(1):115175
pubmed: 31767406
J Cell Biol. 2014 Apr 14;205(1):33-49
pubmed: 24711499
Nature. 2009 Oct 1;461(7264):654-8
pubmed: 19794493
Nat Commun. 2021 Apr 14;12(1):2238
pubmed: 33854069
Nat Commun. 2021 Mar 5;12(1):1461
pubmed: 33674575
J Neurochem. 2002 Oct;83(1):176-85
pubmed: 12358741
Bioorg Med Chem Lett. 2018 Feb 15;28(4):707-710
pubmed: 29366651
Hum Mol Genet. 2003 Dec 15;12(24):3231-44
pubmed: 14570716
Cell Chem Biol. 2020 Jun 18;27(6):763
pubmed: 32559502
Nat Rev Neurol. 2020 Oct;16(10):529-546
pubmed: 32796930
Science. 2009 Jul 24;325(5939):473-7
pubmed: 19556463
Sci Transl Med. 2017 Jan 25;9(374):
pubmed: 28123067
Cell Death Dis. 2015 Nov 05;6:e1970
pubmed: 26539917
EMBO Mol Med. 2014 Jul 28;6(9):1142-60
pubmed: 25069841
J Cell Biol. 2005 May 9;169(3):425-34
pubmed: 15866887
J Cell Biol. 2018 Oct 1;217(10):3640-3655
pubmed: 30018090
Theranostics. 2021 Mar 11;11(11):5279-5295
pubmed: 33859747
J Cell Biol. 2009 Dec 28;187(7):1083-99
pubmed: 20026656
J Cell Biol. 2007 Nov 5;179(3):485-500
pubmed: 17984323
Mol Plant. 2020 Nov 2;13(11):1556-1569
pubmed: 32919085
FEBS J. 2022 Jun;289(12):3304-3316
pubmed: 33914417
Front Cell Neurosci. 2022 Apr 29;16:844211
pubmed: 35573838
Methods Enzymol. 2009;452:143-64
pubmed: 19200881
Biophys J. 2017 Sep 19;113(6):1342-1352
pubmed: 28629620
Genome Biol. 2014;15(12):550
pubmed: 25516281
Front Mol Neurosci. 2014 Sep 29;7:77
pubmed: 25324717
Neuron. 2016 Oct 19;92(2):407-418
pubmed: 27720485
Prog Neurobiol. 2013 Jun;105:49-59
pubmed: 23528736
FEBS Lett. 2001 Nov 30;509(1):22-6
pubmed: 11734199
Commun Integr Biol. 2012 Nov 1;5(6):566-71
pubmed: 23336026
Biochemistry. 2009 Oct 13;48(40):9321-3
pubmed: 19673488
Angew Chem Int Ed Engl. 2017 Sep 11;56(38):11530-11533
pubmed: 28703441
Prog Mol Subcell Biol. 2018;57:107-135
pubmed: 30097773
Hum Mol Genet. 2011 Oct 1;20(19):3852-66
pubmed: 21752829
J Neurosci. 2007 Aug 22;27(34):8989-98
pubmed: 17715336
Science. 2008 Sep 5;321(5894):1354-7
pubmed: 18687924
Hum Mol Genet. 2006 Mar 15;15(6):965-77
pubmed: 16467349
Cell. 1997 Aug 8;90(3):537-48
pubmed: 9267033
Nat Biotechnol. 2018 Oct 22;:
pubmed: 30346940
Autophagy. 2012 Jun;8(6):903-14
pubmed: 22576015
Curr Biol. 2014 Mar 17;24(6):R215-20
pubmed: 24650902
Autophagy. 2015;11(10):1833-48
pubmed: 26325487
Science. 2015 Dec 18;350(6267):1548-51
pubmed: 26634441
Mol Biol Cell. 2001 Aug;12(8):2556-66
pubmed: 11514635
EMBO J. 2018 Oct 15;37(20):
pubmed: 30166454
Eur J Med Chem. 2018 Feb 25;146:251-259
pubmed: 29407955
Proc Natl Acad Sci U S A. 2014 Apr 15;111(15):5706-11
pubmed: 24706802
J Biol Chem. 2016 Aug 26;291(35):18096-106
pubmed: 27405763
Hum Mol Genet. 2010 Oct 1;19(19):3702-20
pubmed: 20616151
Bioinformatics. 2021 Nov 5;37(21):3992-3994
pubmed: 34478488
CNS Neurosci Ther. 2011 Oct;17(5):514-24
pubmed: 20553310
Arch Biochem Biophys. 2002 Dec 1;408(1):103-10
pubmed: 12485608
Cell Mol Life Sci. 2007 Sep;64(17):2219-33
pubmed: 17604998
J Mol Biol. 2020 Apr 3;432(8):2799-2821
pubmed: 31887286
Neurosci Bull. 2010 Apr;26(2):153-62
pubmed: 20332821
Annu Rev Cell Dev Biol. 2007;23:519-47
pubmed: 17506697
Biochem J. 2012 May 1;443(3):681-9
pubmed: 22352297
Trends Biochem Sci. 2022 Sep;47(9):759-771
pubmed: 35508449
Biol Psychiatry. 2022 Jul 1;92(1):10-24
pubmed: 35000752
FEBS J. 2008 Sep;275(17):4263-70
pubmed: 18637946
Mol Biol Cell. 2022 Nov 1;33(13):ar125
pubmed: 36044348
J Cell Biol. 2019 Jun 3;218(6):1908-1927
pubmed: 31010855
Autophagy. 2020 Jan;16(1):185-187
pubmed: 31690177
BioDrugs. 2022 Mar;36(2):105-119
pubmed: 35254632
Cell Rep. 2015 Sep 22;12(11):1876-86
pubmed: 26344761
Elife. 2017 Dec 26;6:
pubmed: 29256861
J Cell Sci. 2020 Sep 16;133(18):
pubmed: 32938689
Proteomics. 2017 Feb;17(3-4):
pubmed: 27860250
Science. 2020 Aug 7;369(6504):
pubmed: 32764038
Autophagy. 2020 May;16(5):826-841
pubmed: 31366282
Neuron. 2017 Mar 8;93(5):1015-1034
pubmed: 28279350
Sci Rep. 2017 Mar 21;7:44849
pubmed: 28322270
Semin Cell Dev Biol. 2018 Feb;74:21-28
pubmed: 28807884
Biomolecules. 2014 Sep 23;4(3):862-84
pubmed: 25250704
J Biol Chem. 2005 Mar 25;280(12):11635-40
pubmed: 15664989
Annu Rev Neurosci. 2019 Jul 8;42:385-406
pubmed: 31283897
Biochem Soc Trans. 2012 Aug;40(4):644-52
pubmed: 22817709
FEBS J. 2016 Sep;283(18):3288-302
pubmed: 26910595
Nat Rev Mol Cell Biol. 2018 Nov;19(11):679-696
pubmed: 30194414
Elife. 2016 Oct 25;5:
pubmed: 27779468
PLoS One. 2020 Mar 24;15(3):e0230026
pubmed: 32208437
J Cell Biol. 2002 Mar 4;156(5):843-54
pubmed: 11864992
J Neurosci. 2009 Mar 11;29(10):3200-5
pubmed: 19279257
Elife. 2019 Mar 25;8:
pubmed: 30907729
Cell Chem Biol. 2016 Apr 21;23(4):453-61
pubmed: 27105281
J Biol Chem. 2018 Apr 13;293(15):5414-5424
pubmed: 29247007
Autophagy. 2019 Jan;15(1):182-183
pubmed: 30295124
Nat Commun. 2021 Oct 26;12(1):6173
pubmed: 34702852
Annu Rev Biochem. 1998;67:425-79
pubmed: 9759494
Cell. 2018 Feb 8;172(4):650-665
pubmed: 29425488
Oral Oncol. 2013 Apr;49(4):293-8
pubmed: 23312237
J Cell Sci. 2009 Jul 1;122(Pt 13):2179-83
pubmed: 19535733

Auteurs

Ron Benyair (R)

Cell and Developmental Biology, University of Michigan, Ann Arbor, United States; Life Sciences Institute, University of Michigan, Ann Arbor, Michigan, United States.

Sai Srinivas Panapakkam Giridharan (SSP)

Cell and Developmental Biology, University of Michigan, Ann Arbor, United States; Life Sciences Institute, University of Michigan, Ann Arbor, Michigan, United States.

Pilar Rivero-Ríos (P)

Cell and Developmental Biology, University of Michigan, Ann Arbor, United States; Life Sciences Institute, University of Michigan, Ann Arbor, Michigan, United States.

Junya Hasegawa (J)

Cell and Developmental Biology, University of Michigan, Ann Arbor, United States; Life Sciences Institute, University of Michigan, Ann Arbor, Michigan, United States.

Emily Bristow (E)

Cell and Developmental Biology, University of Michigan, Ann Arbor, United States; Life Sciences Institute, University of Michigan, Ann Arbor, Michigan, United States.

Eeva-Liisa Eskelinen (EL)

Institute of Biomedicine, University of Turku, Turku, Finland.

Merav D Shmueli (MD)

Department of Systems Immunology, Weizmann Institute of Science, Rehovot, Israel.

Vered Fishbain-Yoskovitz (V)

Department of Systems Immunology, Weizmann Institute of Science, Rehovot, Israel.

Yifat Merbl (Y)

Department of Systems Immunology, Weizmann Institute of Science, Rehovot, Israel.

Lisa M Sharkey (LM)

Department of Neurology, University of Michigan, Ann Arbor, Michigan, United States.

Henry L Paulson (HL)

Department of Neurology, University of Michigan, Ann Arbor, Michigan, United States.

Phyllis I Hanson (PI)

Department of Biological Chemistry, University of Michigan School of Medicine, 1150 W. Medical Center Drive, Ann Arbor, Michigan, United States.

Samarjit Patnaik (S)

Division of Pre-Clinical Innovation, National Center for Advancing Translational Sciences, National Institutes of Health, 9800 Medical Center Drive, Rockville, Maryland 20850, United States.

Ismael Al-Ramahi (I)

Department of Molecular and Human Genetics, Department of Molecular and Cellular Biology, Jan and Dan Duncan Neurological Research Institute, Houston, Texas, United States.

Juan Botas (J)

Department of Molecular and Human Genetics, Department of Molecular and Cellular Biology, Jan and Dan Duncan Neurological Research Institute, Houston, Texas, United States.

Juan Marugan (J)

Division of Pre-Clinical Innovation, National Center for Advancing Translational Sciences, National Institutes of Health, 9800 Medical Center Drive, Rockville, Maryland 20850, United States.

Lois S Weisman (LS)

Cell and Developmental Biology, University of Michigan, Ann Arbor, United States; Life Sciences Institute, University of Michigan, Ann Arbor, Michigan, United States.

Classifications MeSH