Chronic lithium administration in a mouse model for Krabbe disease.

Krabbe Twitcher autophagy globoid cell leukodystrophy lithium psychosine

Journal

JIMD reports
ISSN: 2192-8304
Titre abrégé: JIMD Rep
Pays: United States
ID NLM: 101568557

Informations de publication

Date de publication:
Jan 2022
Historique:
received: 25 08 2021
revised: 11 10 2021
accepted: 14 10 2021
entrez: 14 1 2022
pubmed: 15 1 2022
medline: 15 1 2022
Statut: epublish

Résumé

Krabbe disease (KD; or globoid cell leukodystrophy) is an autosomal recessive lysosomal storage disorder caused by deficiency of the galactosylceramidase (GALC) enzyme. No cure is currently available for KD. Clinical applied treatments are supportive only. Recently, we demonstrated that two differently acting autophagy inducers (lithium and rapamycin) can improve some KD hallmarks in-vitro, laying the foundation for their in-vivo pre-clinical testing. Here, we test lithium carbonate in-vivo, in the spontaneous mouse model for KD, the Twitcher (TWI) mouse. The drug is administered ad libitum via drinking water (600 mg/L) starting from post natal day 20. We longitudinally monitor the mouse motor performance through the grip strength, the hanging wire and the rotarod tests, and a set of biochemical parameters related to the KD pathogenesis [i.e., GALC enzymatic activity, psychosine (PSY) accumulation and astrogliosis]. Additionally, we investigate the expression of some crucial markers related to the two pathways that could be altered by lithium: the autophagy and the β-catenin-dependent pathways. Results demonstrate that lithium has not a significant rescue effect on the TWI phenotype, although it can slightly and transiently improves muscle strength. We also show that lithium, with this administration protocol, is unable to stimulate autophagy in the TWI mice central nervous system, whereas results suggest that it can restore the β-catenin activation status in the TWI sciatic nerve. Overall, these data provide intriguing inputs for further evaluations of lithium treatment in TWI mice.

Identifiants

pubmed: 35028271
doi: 10.1002/jmd2.12258
pii: JMD212258
pmc: PMC8743347
doi:

Types de publication

Journal Article

Langues

eng

Pagination

50-65

Informations de copyright

© 2021 The Authors. JIMD Reports published by John Wiley & Sons Ltd on behalf of SSIEM.

Déclaration de conflit d'intérêts

The authors declare that they have no competing interests.

Références

Mol Genet Metab. 2014 Feb;111(2):172-83
pubmed: 24094551
Biomolecules. 2020 Dec 23;11(1):
pubmed: 33374753
J Neurosci Res. 2016 Nov;94(11):990-1006
pubmed: 27638584
J Neurosci. 2013 Jun 12;33(24):10048-56
pubmed: 23761900
Brain Res. 1988 Jun 28;454(1-2):340-6
pubmed: 3409017
PLoS One. 2015 Nov 25;10(11):e0142267
pubmed: 26605788
Mol Ther. 2015 Nov;23(11):1681-1690
pubmed: 26329589
Cell Death Differ. 2009 Jan;16(1):46-56
pubmed: 18636076
Metabolites. 2020 Mar 12;10(3):
pubmed: 32178227
Sci Rep. 2018 Aug 20;8(1):12462
pubmed: 30127535
Hum Mol Genet. 2017 May 1;26(9):1643-1655
pubmed: 28334757
Int J Neuropsychopharmacol. 2014 Dec 28;18(6):
pubmed: 25548109
Sci Adv. 2019 Nov 20;5(11):eaax7462
pubmed: 31799395
J Neurosci Res. 2016 Nov;94(11):1031-6
pubmed: 27638588
Cell Death Dis. 2014 Nov 20;5:e1529
pubmed: 25412308
Neurobiol Dis. 2006 Aug;23(2):273-80
pubmed: 16759875
Hum Mutat. 2010 Dec;31(12):E1894-914
pubmed: 20886637
J Neurosci Res. 2016 Nov;94(11):1246-60
pubmed: 27638607
Neurobiol Dis. 2018 Dec;120:51-62
pubmed: 30176352
Mol Ther. 2021 Feb 3;29(2):691-701
pubmed: 33388420
Cells. 2021 Jan 25;10(2):
pubmed: 33503974
JIMD Rep. 2019;44:43-54
pubmed: 29995202
J Neurosci. 2015 Apr 22;35(16):6495-505
pubmed: 25904800
Nat Rev Neurosci. 2009 Jul;10(7):519-29
pubmed: 19513088
Mol Ther. 2021 May 5;29(5):1883-1902
pubmed: 33508430
Cells. 2019 Oct 05;8(10):
pubmed: 31590363
Sci Rep. 2017 Jun 6;7(1):2858
pubmed: 28588216
Gene. 2015 Jan 15;555(1):2-13
pubmed: 25260228
J Pathol. 2014 Apr;232(5):509-21
pubmed: 24415155
Cells. 2020 May 01;9(5):
pubmed: 32370022
Neurobiol Dis. 2019 Sep;129:195-207
pubmed: 31108173
Psychopharmacology (Berl). 2015 Sep;232(18):3455-67
pubmed: 26141192
Dev Neurosci. 1991;13(4-5):240-4
pubmed: 1817027
Annu Rev Pathol. 2012;7:185-217
pubmed: 22313379
Dev Biol. 2008 Aug 1;320(1):60-71
pubmed: 18508042
Bioconjug Chem. 2018 Jul 18;29(7):2225-2231
pubmed: 29894633
Pediatr Res. 2021 Jul;90(1):82-92
pubmed: 33173184
J Lipid Res. 1980 Jan;21(1):53-64
pubmed: 7354254
Anat Rec (Hoboken). 2008 Sep;291(9):1088-96
pubmed: 18727075
J Neuropathol Exp Neurol. 1993 Sep;52(5):490-8
pubmed: 8360702
J Neurosci Res. 2016 Nov;94(11):1049-61
pubmed: 27638591
Biofactors. 2020 May;46(3):411-420
pubmed: 31960520
Mol Ther. 2018 Mar 7;26(3):874-889
pubmed: 29433937
J Neurosci Res. 2016 Nov;94(11):1328-32
pubmed: 27638614
Neuroscience. 2015 May 21;294:101-8
pubmed: 25743255
J Biophotonics. 2017 Mar;10(3):385-393
pubmed: 26990139
Hum Mol Genet. 2015 Jun 15;24(12):3372-89
pubmed: 25749991
Oncotarget. 2017 Apr 4;8(14):23539-23550
pubmed: 28212563
J Neurosci Res. 2016 Nov;94(11):1359-67
pubmed: 27037626
Clin Chim Acta. 2015 Oct 23;450:6-10
pubmed: 26232157
J Cell Biol. 2002 Sep 16;158(6):1097-107
pubmed: 12221071
Mol Cell Proteomics. 2019 Jun;18(6):1227-1241
pubmed: 30926673
Front Cell Neurosci. 2020 Dec 23;14:619712
pubmed: 33424556
Stem Cell Reports. 2017 Dec 12;9(6):1853-1867
pubmed: 29198828
J Neuroinflammation. 2016 Feb 08;13:32
pubmed: 26856696
PLoS One. 2012;7(3):e33292
pubmed: 22428012
Hum Gene Ther. 2018 Jul;29(7):785-801
pubmed: 29316812
J Neurochem. 1996 Mar;66(3):1118-24
pubmed: 8769874
ACS Chem Neurosci. 2014 Jun 18;5(6):434-42
pubmed: 24738557
Clin Chim Acta. 1992 Jan 31;205(1-2):87-96
pubmed: 1521344
Brain Res. 1980 Dec 8;202(2):479-83
pubmed: 7437911
Proc Natl Acad Sci U S A. 2019 Oct 1;116(40):20097-20103
pubmed: 31527255
J Pain. 2019 Nov;20(11):1338-1352
pubmed: 31075529
Adv Exp Med Biol. 1976;68:115-26
pubmed: 937104

Auteurs

Ambra Del Grosso (A)

NEST, Istituto Nanoscienze-CNR and Scuola Normale Superiore, Piazza San Silvestro Pisa Italy.

Gabriele Parlanti (G)

NEST, Istituto Nanoscienze-CNR and Scuola Normale Superiore, Piazza San Silvestro Pisa Italy.

Lucia Angella (L)

NEST, Istituto Nanoscienze-CNR and Scuola Normale Superiore, Piazza San Silvestro Pisa Italy.

Nadia Giordano (N)

Scuola Normale Superiore, Piazza dei Cavalieri Pisa Italy.
CNR Neuroscience Institute Pisa Italy.

Ilaria Tonazzini (I)

NEST, Istituto Nanoscienze-CNR and Scuola Normale Superiore, Piazza San Silvestro Pisa Italy.

Elisa Ottalagana (E)

NEST, Istituto Nanoscienze-CNR and Scuola Normale Superiore, Piazza San Silvestro Pisa Italy.

Sara Carpi (S)

NEST, Istituto Nanoscienze-CNR and Scuola Normale Superiore, Piazza San Silvestro Pisa Italy.

Roberto Maria Pellegrino (RM)

Department of Chemistry, Biology, and Biotechnologies University of Perugia Perugia Italy.

Husam B R Alabed (HBR)

Department of Chemistry, Biology, and Biotechnologies University of Perugia Perugia Italy.

Carla Emiliani (C)

Department of Chemistry, Biology, and Biotechnologies University of Perugia Perugia Italy.

Matteo Caleo (M)

Scuola Normale Superiore, Piazza dei Cavalieri Pisa Italy.
CNR Neuroscience Institute Pisa Italy.
Department of Biomedical Sciences University of Padua Padova Italy.

Marco Cecchini (M)

NEST, Istituto Nanoscienze-CNR and Scuola Normale Superiore, Piazza San Silvestro Pisa Italy.

Classifications MeSH