Therapeutic effect of NLRP3 inhibition on hearing loss induced by systemic inflammation in a CAPS-associated mouse model

Masters, 2009, Horror autoinflammaticus: the molecular pathophysiology of autoinflammatory disease (*), Annu Rev Immunol, 27, 621, 10.1146/annurev.immunol.25.022106.141627 Balow, 1997, A high-resolution genetic map of the familial Mediterranean fever candidate region allows identification of haplotype-sharing among ethnic groups, Genomics, 44, 280, 10.1006/geno.1997.4860 Hoffman, 2001, Mutation of a new gene encoding a putative pyrin-like protein causes familial cold autoinflammatory syndrome and Muckle-Wells syndrome, Nat Genet, 29, 301, 10.1038/ng756 Heymann, 2013, Contribution of the inflammasomes to autoinflammatory diseases and recent mouse models as research tools, Clin Immunol, 147, 175, 10.1016/j.clim.2013.01.006 Yu, 2007, Pyrin activates the ASC pyroptosome in response to engagement by autoinflammatory PSTPIP1 mutants, Mol Cell, 28, 214, 10.1016/j.molcel.2007.08.029 Mariathasan, 2006, Cryopyrin activates the inflammasome in response to toxins and ATP, Nature, 440, 228, 10.1038/nature04515 Kanneganti, 2006, Bacterial RNA and small antiviral compounds activate caspase-1 through cryopyrin/Nalp3, Nature, 440, 233, 10.1038/nature04517 Schroder, 2010, The inflammasomes, Cell, 140, 821, 10.1016/j.cell.2010.01.040 Mangan, 2018, Targeting the NLRP3 inflammasome in inflammatory diseases, Nat Rev Drug Discov, 17, 588, 10.1038/nrd.2018.97 Heneka, 2018, Inflammasome signalling in brain function and neurodegenerative disease, Nat Rev Neurosci, 19, 610, 10.1038/s41583-018-0055-7 Jesus, 2014, IL-1 blockade in autoinflammatory syndromes, Annu Rev Med, 65, 223, 10.1146/annurev-med-061512-150641 Harrison, 2016, Anakinra as a diagnostic challenge and treatment option for systemic autoinflammatory disorders of undefined etiology, JCI Insight, 1, e86336, 10.1172/jci.insight.86336 Yu, 2016, Mitochondria and the NLRP3 inflammasome: physiological and pathological relevance, Arch Pharm Res, 39, 1503, 10.1007/s12272-016-0827-4 Neven, 2004, Molecular basis of the spectral expression of CIAS1 mutations associated with phagocytic cell-mediated autoinflammatory disorders CINCA/NOMID, MWS, and FCU, Blood, 103, 2809, 10.1182/blood-2003-07-2531 McDermott, 2007, From inflammasomes to fevers, crystals and hypertension: how basic research explains inflammatory diseases, Trends Mol Med, 13, 381, 10.1016/j.molmed.2007.07.005 Welzel, 2021, Diagnosis and management of the cryopyrin-associated periodic syndromes (CAPS): what do we know today?, J Clin Med, 10, 128, 10.3390/jcm10010128 Ahmadi, 2011, Cryopyrin-associated periodic syndromes: otolaryngologic and audiologic manifestations, Otolaryngol Head Neck Surg, 145, 295, 10.1177/0194599811402296 Kuemmerle-Deschner, 2015, Early detection of sensorineural hearing loss in Muckle–Wells-syndrome, Pediatr Rheumatol Online J, 13, 43, 10.1186/s12969-015-0041-9 Vambutas, 2014, Early efficacy trial of anakinra in corticosteroid-resistant autoimmune inner ear disease, J Clin Invest, 124, 4115, 10.1172/JCI76503 Nakanishi, 2017, NLRP3 mutation and cochlear autoinflammation cause syndromic and nonsyndromic hearing loss DFNA34 responsive to anakinra therapy, Proc Natl Acad Sci USA, 114, E7766, 10.1073/pnas.1702946114 Nakanishi, 2020, Genetic hearing loss associated with autoinflammation, Front Neurol, 11, 141, 10.3389/fneur.2020.00141 Ko, 2020, Non-invasive in vivo imaging of caspase-1 activation enables rapid and spatiotemporal detection of acute and chronic inflammatory disorders, Biomaterials, 226, 10.1016/j.biomaterials.2019.119543 Fernandes-Alnemri, 2010, The AIM2 inflammasome is critical for innate immunity to Francisella tularensis, Nat Immunol, 11, 385, 10.1038/ni.1859 Fernandes-Alnemri, 2009, AIM2 activates the inflammasome and cell death in response to cytoplasmic DNA, Nature, 458, 509, 10.1038/nature07710 Feldmann, 2002, Chronic infantile neurological cutaneous and articular syndrome is caused by mutations in CIAS1, a gene highly expressed in polymorphonuclear cells and chondrocytes, Am J Hum Genet, 71, 198, 10.1086/341357 Aksentijevich, 2002, De novo CIAS1 mutations, cytokine activation, and evidence for genetic heterogeneity in patients with neonatal-onset multisystem inflammatory disease (NOMID): a new member of the expanding family of pyrin-associated autoinflammatory diseases, Arthritis Rheum, 46, 3340, 10.1002/art.10688 Hough, 2022, Macrophages in the cochlea; an immunological link between risk factors and progressive hearing loss, Glia, 70, 219, 10.1002/glia.24095 Kishimoto, 2019, Early development of resident macrophages in the mouse cochlea depends on yolk sac hematopoiesis, Front Neurol, 10, 1115, 10.3389/fneur.2019.01115 Yona, 2013, Fate mapping reveals origins and dynamics of monocytes and tissue macrophages under homeostasis, Immunity, 38, 79, 10.1016/j.immuni.2012.12.001 Tapia-Abellan, 2019, MCC950 closes the active conformation of NLRP3 to an inactive state, Nat Chem Biol, 15, 560, 10.1038/s41589-019-0278-6 Okano, 2014, Immune system of the inner ear as a novel therapeutic target for sensorineural hearing loss, Front Pharmacol, 5, 205, 10.3389/fphar.2014.00205 Fujioka, 2014, Inflammatory and immune responses in the cochlea: potential therapeutic targets for sensorineural hearing loss, Front Pharmacol, 5, 287, 10.3389/fphar.2014.00287 Bae, 2021, Neutrophils infiltrate into the spiral ligament but not the stria vascularis in the cochlea during lipopolysaccharide-induced inflammation, Theranostics, 11, 2522, 10.7150/thno.49121 Wree, 2014, NLRP3 inflammasome activation results in hepatocyte pyroptosis, liver inflammation, and fibrosis in mice, Hepatology, 59, 898, 10.1002/hep.26592 Brydges, 2009, Inflammasome-mediated disease animal models reveal roles for innate but not adaptive immunity, Immunity, 30, 875, 10.1016/j.immuni.2009.05.005 Sato, 2010, Expression of fractalkine receptor CX3CR1 on cochlear macrophages influences survival of hair cells following ototoxic injury, J Assoc Res Otolaryngol, 11, 223, 10.1007/s10162-009-0198-3 Hirose, 2014, Systemic lipopolysaccharide compromises the blood-labyrinth barrier and increases entry of serum fluorescein into the perilymph, J Assoc Res Otolaryngol, 15, 707, 10.1007/s10162-014-0476-6 Jiang, 2019, Inflammation up-regulates cochlear expression of TRPV1 to potentiate drug-induced hearing loss, Sci Adv, 5, eaaw1836, 10.1126/sciadv.aaw1836 Vethanayagam, 2016, Toll-like receptor 4 modulates the cochlear immune response to acoustic injury, Cell Death Dis, 7, e2245, 10.1038/cddis.2016.156 Frye, 2017, Dynamic activation of basilar membrane macrophages in response to chronic sensory cell degeneration in aging mouse cochleae, Hear Res, 344, 125, 10.1016/j.heares.2016.11.003 Zhang, 2013, Characterization and inflammatory response of perivascular-resident macrophage-like melanocytes in the vestibular system, J Assoc Res Otolaryngol, 14, 635, 10.1007/s10162-013-0403-2 Vande Walle, 2019, MCC950/CRID3 potently targets the NACHT domain of wild-type NLRP3 but not disease-associated mutants for inflammasome inhibition, PLoS Biol, 17, 10.1371/journal.pbio.3000354