The cholesterol depleting agent, (2-Hydroxypropyl)-ß-cyclodextrin, does not affect disease progression in SOD1^G93A mice

References

• Niedermeyer S, Murn M, Choi PJ. Respiratory failure in amyotrophic lateral sclerosis. Chest 2019;155:401–8.
   PubMed Web of Science ®Google Scholar
• Chio A, Logroscino G, Hardiman O, Swingler R, Mitchell D, Beghi E, et al. Prognostic factors in ALS: A critical review. Amyotroph Lateral Scler. 2009;10:310–23.
   PubMed Web of Science ®Google Scholar
• Mejzini R, Flynn LL, Pitout IL, Fletcher S, Wilton SD, Akkari PA. ALS genetics, mechanisms, and therapeutics: where are we now? Front Neurosci. 2019;13:1310.
   PubMed Web of Science ®Google Scholar
• Dupuis L, Corcia P, Fergani A, Gonzalez De Aguilar JL, Bonnefont-Rousselot D, Bittar R, et al. Dyslipidemia is a protective factor in amyotrophic lateral sclerosis. Neurology 2008;70:1004–9.
   PubMed Web of Science ®Google Scholar
• Boillée S, Vande Velde C, Cleveland DW. ALS: a disease of motor neurons and their nonneuronal neighbors. Neuron 2006;52:39–59.
   PubMed Web of Science ®Google Scholar
• Loeffler JP, Picchiarelli G, Dupuis L, Gonzalez De Aguilar JL. The role of skeletal muscle in amyotrophic lateral sclerosis. Brain Pathol. 2016;26:227–36.
   PubMed Web of Science ®Google Scholar
• Bryson JB, Hobbs C, Parsons MJ, Bosch KD, Pandraud A, Walsh FS, et al. Amyloid precursor protein (APP) contributes to pathology in the SOD1(G93A) mouse model of amyotrophic lateral sclerosis. Hum Mol Genet. 2012;21:3871–82.
   PubMed Web of Science ®Google Scholar
• Truong PH, Crouch PJ, Hilton JBW, McLean CA, Cappai R, Ciccotosto GD. Sex-dependent effects of amyloid precursor-like protein 2 in the SOD1-G37R transgenic mouse model of MND. Cell Mol Life Sci. 2021;78:6605–30.
   PubMed Web of Science ®Google Scholar
• Calingasan NY, Chen J, Kiaei M, Beal MF. Beta-amyloid 42 accumulation in the lumbar spinal cord motor neurons of amyotrophic lateral sclerosis patients. Neurobiol Dis. 2005;19:340–7.
   PubMed Web of Science ®Google Scholar
• Rabinovich-Toidman P, Rabinovich-Nikitin I, Ezra A, Barbiro B, Fogel H, Slutsky I, et al. Mutant SOD1 Increases APP Expression and Phosphorylation in Cellular and Animal Models of ALS. PLoS One. 2015;10:e0143420.
   PubMed Web of Science ®Google Scholar
• Hartmann T, Kuchenbecker J, Grimm MO. Alzheimer’s disease: the lipid connection. J Neurochem. 2007;103 Suppl 1: Suppl 159–70.
   PubMed Web of Science ®Google Scholar
• Corder EH, Saunders AM, Strittmatter WJ, Schmechel DE, Gaskell PC, Small GW, et al. Gene dose of apolipoprotein E type 4 allele and the risk of Alzheimer’s disease in late onset families. Science. 1993;261:921–3.
   PubMed Web of Science ®Google Scholar
• Djelti F, Braudeau J, Hudry E, Dhenain M, Varin J, Bièche I, et al. CYP46A1 inhibition, brain cholesterol accumulation and neurodegeneration pave the way for Alzheimer’s disease. Brain. 2015;138:2383–98.
   PubMed Web of Science ®Google Scholar
• Carstea ED, Morris JA, Coleman KG, Loftus SK, Zhang D, Cummings C, et al. Niemann-Pick C1 disease gene: homology to mediators of cholesterol homeostasis. Science. 1997;277:228–31.
   PubMed Web of Science ®Google Scholar
• Nixon RA. Niemann-Pick Type C disease and Alzheimer’s disease: the APP-endosome connection fattens up. Am J Pathol. 2004;164:757–61.
   PubMed Web of Science ®Google Scholar
• Maulik M, Ghoshal B, Kim J, Wang Y, Yang J, Westaway D, et al. Mutant human APP exacerbates pathology in a mouse model of NPC and its reversal by a β-cyclodextrin. Hum Mol Genet. 2012;21:4857–75.
   PubMed Web of Science ®Google Scholar
• Yao J, Ho D, Calingasan NY, Pipalia NH, Lin MT, Beal MF. Neuroprotection by cyclodextrin in cell and mouse models of Alzheimer disease. J Exp Med. 2012;209:2501–13.
   PubMed Web of Science ®Google Scholar
• Liu B, Li H, Repa JJ, Turley SD, Dietschy JM. Genetic variations and treatments that affect the lifespan of the NPC1 mouse. J Lipid Res. 2008;49:663–9.
   PubMed Web of Science ®Google Scholar
• Davidson CD, Ali NF, Micsenyi MC, Stephney G, Renault S, Dobrenis K, et al. Chronic cyclodextrin treatment of murine Niemann-Pick C disease ameliorates neuronal cholesterol and glycosphingolipid storage and disease progression. PLoS One. 2009;4:e6951.
   PubMed Web of Science ®Google Scholar
• Delaye JB, Patin F, Piver E, Bruno C, Vasse M, Vourc’h P, et al. Low IDL-B and high LDL-1 subfraction levels in serum of ALS patients. J Neurol Sci. 2017;380:124–7.
   PubMed Web of Science ®Google Scholar
• Johnson B, Kokkinis A, Gai N, Shamim EA, Blackstone C, Fischbeck KH, et al. Nonalcoholic fatty liver disease in patients with inherited and sporadic motor neuron degeneration. Genes. 2022;13:936.
   PubMed Web of Science ®Google Scholar
• Lütjohann D, Papassotiropoulos A, Björkhem I, Locatelli S, Bagli M, Oehring RD, et al. Plasma 24S-hydroxycholesterol (cerebrosterol) is increased in Alzheimer and vascular demented patients. J Lipid Res. 2000;41:195–8.
   PubMed Web of Science ®Google Scholar
• Kim SM, Noh MY, Kim H, Cheon SY, Lee KM, Lee J, et al. 25-Hydroxycholesterol is involved in the pathogenesis of amyotrophic lateral sclerosis. Oncotarget 2017;8:11855–67.
   PubMedGoogle Scholar
• Abdel-Khalik J, Yutuc E, Crick PJ, Gustafsson J, Warner M, Roman G, et al. Defective cholesterol metabolism in amyotrophic lateral sclerosis. J Lipid Res. 2017;58:267–78.
   PubMed Web of Science ®Google Scholar
• Andersson S, Gustafsson N, Warner M, Gustafsson JA. Inactivation of liver X receptor beta leads to adult-onset motor neuron degeneration in male mice. Proc Natl Acad Sci U S A. 2005;102:3857–62.
   PubMed Web of Science ®Google Scholar
• Wang Y, Muneton S, Sjovall J, Jovanovic JN, Griffiths WJ. The effect of 24S-hydroxycholesterol on cholesterol homeostasis in neurons: quantitative changes to the cortical neuron proteome. J Proteome Res. 2008;7:1606–14.
   PubMed Web of Science ®Google Scholar
• Kacher R, Mounier C, Caboche J, Betuing S. Altered cholesterol homeostasis in huntington’s disease. Front Aging Neurosci. 2022;14:797220.
   PubMed Web of Science ®Google Scholar
• Boussicault L, Alves S, Lamazière A, Planques A, Heck N, Moumné L, et al. CYP46A1, the rate-limiting enzyme for cholesterol degradation, is neuroprotective in Huntington’s disease. Brain. 2016;139:953–70.
   PubMed Web of Science ®Google Scholar
• Griffin LD, Gong W, Verot L, Mellon SH. Niemann-Pick type C disease involves disrupted neurosteroidogenesis and responds to allopregnanolone. Nat Med. 2004;10:704–11.
   PubMed Web of Science ®Google Scholar
• Antonini A, Caioli S, Saba L, Vindigni G, Biocca S, Canu N, et al. Membrane cholesterol depletion in cortical neurons highlights altered NMDA receptor functionality in a mouse model of amyotrophic lateral sclerosis. Biochim Biophys Acta Mol Basis Dis. 2018;1864:509–19.
   PubMed Web of Science ®Google Scholar
• Dupuis L, Pradat P-F, Ludolph AC, Loeffler J-P. Energy metabolism in amyotrophic lateral sclerosis. Lancet Neurol. 2011;10:75–82.
   PubMed Web of Science ®Google Scholar
• Bai L, Wang Y, Huo J, Li S, Wen Y, Liu Q, et al. Simvastatin accelerated motoneurons death in SOD1(G93A) mice through inhibiting Rab7-mediated maturation of late autophagic vacuoles. Cell Death Dis. 2021;12:392.
   PubMed Web of Science ®Google Scholar
• Levine L. Statins stimulate arachidonic acid release and prostaglandin I2 production in rat liver cells. Lipids Health Dis. 2003;2:1.
   PubMedGoogle Scholar
• Lee H, Lee JJ, Park NY, Dubey SK, Kim T, Ruan K, et al. Multi-omic analysis of selectively vulnerable motor neuron subtypes implicates altered lipid metabolism in ALS. Nat Neurosci. 2021;24:1673–85.
   PubMed Web of Science ®Google Scholar
• Latorre-Leal M, Rodriguez-Rodriguez P, Franchini L, Daniilidou M, Eroli F, Winblad B, et al. Sex-dependent effects of CYP46A1 overexpression on cognitive function during aging. bioRxiv 2021.
   Google Scholar