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A Community-Informed Toolkit for Physician-Scientist Competencies and Milestones: Bridging the Worlds of Medicine and Research. Acad Med. 2026 Feb 28. Estrada L, Baca JT, Baron MH, Brass LF, Craft CS, Cavazos JE, Kao AW, Roghmann MC, Yellin J, McCormack WT. PMID: 41766344.
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Lysosomal protease-mediated APP degradation is pH-dependent, mutation-sensitive, and facilitates tau proteolysis. Mol Neurodegener Adv. 2026; 2(1):10. Ackley C, Liau Z, Arya S, Antee T, Cheang E, Knudsen GM, Lane-Donovan C, Sampognaro PJ, Kao AW. PMID: 41675590; PMCID: PMC12886278.
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Prosaposin Is Cleaved Into Saposins by Multiple Cathepsins in a Progranulin-Regulated Fashion. J Neurochem. 2026 Jan; 170(1):e70357. Hodul M, Lane-Donovan C, Cheang ES, Gao V, Sampognaro PJ, Mambou EA, Yang Z, Kao AW. PMID: 41572567; PMCID: PMC12828107.
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Integrative Genomic and Functional Analyses Reveal NINL as a Modulator of Tau Aggregation. bioRxiv. 2025 Dec 16. Swift SK, Huang G, Cochran JN, Minaya MA, Castruita PA, Miller KJ, Starr E, Galasso G, Marsh JA, Kao AW, Yokoyama JS, Karch CM. PMID: 41446180; PMCID: PMC12724438.
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Drug-induced phospholipidosis as an artifact in antiviral drug repurposing. bioRxiv. 2025 Nov 21. Glenn IS, Paris L, White AD, Garda VG, Montano M, Khalid MM, Kao AW, Ott M, Shoichet BK. PMID: 41332603; PMCID: PMC12667853.
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The lysosome and proteostatic stress at the intersection of pediatric neurological disorders and adult neurodegenerative diseases. Prog Neurobiol. 2025 Dec; 255:102854. Lane-Donovan C, Paredes M, Kao AW. PMID: 41253210.
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Lysosomal Protease-Mediated APP Degradation is pH-Dependent, Mutation-Sensitive, and Facilitates Tau Proteolysis. Res Sq. 2025 Jul 10. Ackley C, Liau Z, Arya S, Antee T, Knudsen GM, Lane-Donovan C, Sampognaro PJ, Kao AW. PMID: 40671818; PMCID: PMC12265180.
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GRAMD1B is a regulator of lipid homeostasis, autophagic flux and phosphorylated tau. Nat Commun. 2025 Apr 09; 16(1):3312. Acosta Ingram D, Turkes E, Kim TY, Vo S, Sweeney N, Bonte MA, Rutherford R, Julian DL, Pan M, Marsh J, Argouarch AR, Wu M, Scharre DW, Bell EH, Honig LS, Vonsattel JP, Serrano GE, Beach TG, Karch CM, Kao AW, Hester ME, Han X, Fu H. PMID: 40204713; PMCID: PMC11982250.
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The relationship between caloric intake and clinical outcomes in critically ill patients: A retrospective study. Clin Nutr ESPEN. 2025 Feb; 65:9-15. Lin YR, Chen PC, Li WT, Huang MH, Huang SF, Wang CJ, Chien YW, Kao AW, Shan YS. PMID: 39551353.
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mTOR activation induces endolysosomal remodeling and nonclassical secretion of IL-32 via exosomes in inflammatory reactive astrocytes. J Neuroinflammation. 2024 Aug 08; 21(1):198. Leng K, Rooney B, McCarthy F, Xia W, Rose IVL, Bax S, Chin M, Fathi S, Herrington KA, Leonetti M, Kao A, Fancy SPJ, Elias JE, Kampmann M. PMID: 39118084; PMCID: PMC11312292.
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Second international symposium on the chaperone code, 2023. Cell Stress Chaperones. 2024 Feb; 29(1):88-96. Buchner J, Alasady MJ, Backe SJ, Blagg BSJ, Carpenter RL, Colombo G, Gelis I, Gewirth DT, Gierasch LM, Houry WA, Johnson JL, Kang BH, Kao AW, LaPointe P, Mattoo S, McClellan AJ, Neckers LM, Prodromou C, Rasola A, Sager RA, Theodoraki MA, Truman AW, Truttman MC, Zachara NE, Bourboulia D, Mollapour M, Woodford MR. PMID: 38316354; PMCID: PMC10939070.
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The progranulin cleavage product granulin 3 exerts a dominant negative effect on animal fitness. Hum Mol Genet. 2024 01 20; 33(3):245-253. Wang AL, Mambou EA, Kao AW. PMID: 37903062; PMCID: PMC10800025.
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Correction: Mutations in α-synuclein, TDP-43 and tau prolong protein half-life through diminished degradation by lysosomal proteases. Mol Neurodegener. 2023 May 30; 18(1):34. Sampognaro PJ, Arya S, Knudsen GM, Gunderson EL, Sandoval-Perez A, Hodul M, Bowles K, Craik CS, Jacobson MP, Kao AW. PMID: 37254128; PMCID: PMC10228009.
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Mutations in α-synuclein, TDP-43 and tau prolong protein half-life through diminished degradation by lysosomal proteases. Mol Neurodegener. 2023 05 02; 18(1):29. Sampognaro PJ, Arya S, Knudsen GM, Gunderson EL, Sandoval-Perez A, Hodul M, Bowles K, Craik CS, Jacobson MP, Kao AW. PMID: 37131250; PMCID: PMC10155372.
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Tuberous sclerosis complex is associated with a novel human tauopathy. Acta Neuropathol. 2023 01; 145(1):1-12. Hwang JL, Perloff OS, Gaus SE, Benitez C, Alquezar C, Cosme CQ, Nana AL, Vatsavayai SC, Ramos EM, Geschwind DH, Miller BL, Kao AW, Seeley WW. PMID: 36469115; PMCID: PMC10244026.
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Postmortem Human Dura Mater Cells Exhibit Phenotypic, Transcriptomic and Genetic Abnormalities that Impact their Use for Disease Modeling. Stem Cell Rev Rep. 2022 12; 18(8):3050-3065. Argouarch AR, Schultz N, Yang AC, Jang Y, Garcia K, Cosme CG, Corrales CI, Nana AL, Karydas AM, Spina S, Grinberg LT, Miller B, Wyss-Coray T, Abyzov A, Goodarzi H, Seeley WW, Kao AW. PMID: 35809166; PMCID: PMC9622518.
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Phenotypic Screening Using High-Content Imaging to Identify Lysosomal pH Modulators in a Neuronal Cell Model. ACS Chem Neurosci. 2022 05 18; 13(10):1505-1516. Chin MY, Ang KH, Davies J, Alquezar C, Garda VG, Rooney B, Leng K, Kampmann M, Arkin MR, Kao AW. PMID: 35522480; PMCID: PMC9121341.
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TSC1 loss increases risk for tauopathy by inducing tau acetylation and preventing tau clearance via chaperone-mediated autophagy. Sci Adv. 2021 Nov 05; 7(45):eabg3897. Alquezar C, Schoch KM, Geier EG, Ramos EM, Scrivo A, Li KH, Argouarch AR, Mlynarski EE, Dombroski B, DeTure M, Dickson DW, Yokoyama JS, Cuervo AM, Burlingame AL, Schellenberg GD, Miller TM, Miller BL, Kao AW. PMID: 34739309; PMCID: PMC8570595.
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Processing of progranulin into granulins involves multiple lysosomal proteases and is affected in frontotemporal lobar degeneration. Mol Neurodegener. 2021 08 03; 16(1):51. Mohan S, Sampognaro PJ, Argouarch AR, Maynard JC, Welch M, Patwardhan A, Courtney EC, Zhang J, Mason A, Li KH, Huang EJ, Seeley WW, Miller BL, Burlingame A, Jacobson MP, Kao AW. PMID: 34344440; PMCID: PMC8330050.
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Genetically Encoded, pH-Sensitive mTFP1 Biosensor for Probing Lysosomal pH. ACS Sens. 2021 06 25; 6(6):2168-2180. Chin MY, Patwardhan AR, Ang KH, Wang AL, Alquezar C, Welch M, Nguyen PT, Grabe M, Molofsky AV, Arkin MR, Kao AW. PMID: 34102054; PMCID: PMC8240087.
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Tau Post-translational Modifications: Dynamic Transformers of Tau Function, Degradation, and Aggregation. Front Neurol. 2020; 11:595532. Alquezar C, Arya S, Kao AW. PMID: 33488497; PMCID: PMC7817643.
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Progranulin Adsorbs to Polypropylene Tubes and Disrupts Functional Assays: Implications for Research, Biomarker Studies, and Therapeutics. Front Neurosci. 2020; 14:602235. Gururaj S, Sampognaro PJ, Argouarch AR, Kao AW. PMID: 33381010; PMCID: PMC7768044.
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Association of Cognitive and Behavioral Features Between Adults With Tuberous Sclerosis and Frontotemporal Dementia. JAMA Neurol. 2020 03 01; 77(3):358-366. Liu AJ, Staffaroni AM, Rojas-Martinez JC, Olney NT, Alquezar-Burillo C, Ljubenkov PA, La Joie R, Fong JC, Taylor J, Karydas A, Ramos EM, Coppola G, Boxer AL, Rabinovici GD, Miller BL, Kao AW. PMID: 31860018; PMCID: PMC6990672.
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Heavy metals contaminating the environment of a progressive supranuclear palsy cluster induce tau accumulation and cell death in cultured neurons. Sci Rep. 2020 01 17; 10(1):569. Alquezar C, Felix JB, McCandlish E, Buckley BT, Caparros-Lefebvre D, Karch CM, Golbe LI, Kao AW. PMID: 31953414; PMCID: PMC6969162.
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Protons in small spaces: Discrete simulations of vesicle acidification. PLoS Comput Biol. 2019 12; 15(12):e1007539. Singh A, Marcoline FV, Veshaguri S, Kao AW, Bruchez M, Mindell JA, Stamou D, Grabe M. PMID: 31869334; PMCID: PMC6946529.
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A Comprehensive Resource for Induced Pluripotent Stem Cells from Patients with Primary Tauopathies. Stem Cell Reports. 2019 11 12; 13(5):939-955. Karch CM, Kao AW, Karydas A, Onanuga K, Martinez R, Argouarch A, Wang C, Huang C, Sohn PD, Bowles KR, Spina S, Silva MC, Marsh JA, Hsu S, Pugh DA, Ghoshal N, Norton J, Huang Y, Lee SE, Seeley WW, Theofilas P, Grinberg LT, Moreno F, McIlroy K, Boeve BF, Cairns NJ, Crary JF, Haggarty SJ, Ichida JK, Kosik KS, Miller BL, Gan L, Goate AM, Temple S, Tau Consortium Stem Cell Group. PMID: 31631020; PMCID: PMC6895712.
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Lifespan-increasing drug nordihydroguaiaretic acid inhibits p300 and activates autophagy. NPJ Aging Mech Dis. 2019; 5:7. Tezil T, Chamoli M, Ng CP, Simon RP, Butler VJ, Jung M, Andersen J, Kao AW, Verdin E. PMID: 31602311; PMCID: PMC6775102.
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Age- and stress-associated C. elegans granulins impair lysosomal function and induce a compensatory HLH-30/TFEB transcriptional response. PLoS Genet. 2019 08; 15(8):e1008295. Butler VJ, Gao F, Corrales CI, Cortopassi WA, Caballero B, Vohra M, Ashrafi K, Cuervo AM, Jacobson MP, Coppola G, Kao AW. PMID: 31398187; PMCID: PMC6703691.
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Multi-Granulin Domain Peptides Bind to Pro-Cathepsin D and Stimulate Its Enzymatic Activity More Effectively Than Progranulin in Vitro. Biochemistry. 2019 06 11; 58(23):2670-2674. Butler VJ, Cortopassi WA, Gururaj S, Wang AL, Pierce OM, Jacobson MP, Kao AW. PMID: 31099551; PMCID: PMC6666309.
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Tau/MAPT disease-associated variant A152T alters tau function and toxicity via impaired retrograde axonal transport. Hum Mol Genet. 2019 05 01; 28(9):1498-1514. Butler VJ, Salazar DA, Soriano-Castell D, Alves-Ferreira M, Dennissen FJA, Vohra M, Oses-Prieto JA, Li KH, Wang AL, Jing B, Li B, Groisman A, Gutierrez E, Mooney S, Burlingame AL, Ashrafi K, Mandelkow EM, Encalada SE, Kao AW. PMID: 30590647; PMCID: PMC6489414.
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Progranulin Stimulates the In Vitro Maturation of Pro-Cathepsin D at Acidic pH. J Mol Biol. 2019 03 01; 431(5):1038-1047. Butler VJ, Cortopassi WA, Argouarch AR, Ivry SL, Craik CS, Jacobson MP, Kao AW. PMID: 30690031; PMCID: PMC6613950.
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Tauopathy-associated PERK alleles are functional hypomorphs that increase neuronal vulnerability to ER stress. Hum Mol Genet. 2018 11 15; 27(22):3951-3963. Yuan SH, Hiramatsu N, Liu Q, Sun XV, Lenh D, Chan P, Chiang K, Koo EH, Kao AW, Litvan I, Lin JH. PMID: 30137327; PMCID: PMC6216228.
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Regionally specific TSC1 and TSC2 gene expression in tuberous sclerosis complex. Sci Rep. 2018 09 06; 8(1):13373. Li Y, Barkovich MJ, Karch CM, Nillo RM, Fan CC, Broce IJ, Tan CH, Cuneo D, Hess CP, Dillon WP, Glenn OA, Glastonbury CM, Olney N, Yokoyama JS, Bonham LW, Miller B, Kao A, Schmansky N, Fischl B, Andreassen OA, Jernigan T, Dale A, Barkovich AJ, Desikan RS, Sugrue LP. PMID: 30190613; PMCID: PMC6127129.
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The Use of Caenorhabditis elegans to Study Progranulin in the Regulation of Programmed Cell Death and Stress Response. Methods Mol Biol. 2018; 1806:193-206. Hsu TY, Butler VJ, Kao AW. PMID: 29956278.
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Clinicopathological correlations in behavioural variant frontotemporal dementia. Brain. 2017 Dec 01; 140(12):3329-3345. Perry DC, Brown JA, Possin KL, Datta S, Trujillo A, Radke A, Karydas A, Kornak J, Sias AC, Rabinovici GD, Gorno-Tempini ML, Boxer AL, De May M, Rankin KP, Sturm VE, Lee SE, Matthews BR, Kao AW, Vossel KA, Tartaglia MC, Miller ZA, Seo SW, Sidhu M, Gaus SE, Nana AL, Vargas JNS, Hwang JL, Ossenkoppele R, Brown AB, Huang EJ, Coppola G, Rosen HJ, Geschwind D, Trojanowski JQ, Grinberg LT, Kramer JH, Miller BL, Seeley WW. PMID: 29053860; PMCID: PMC5841140.
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Linking tuberous sclerosis complex, excessive mTOR signaling, and age-related neurodegeneration: a new association between TSC1 mutation and frontotemporal dementia. Acta Neuropathol. 2017 11; 134(5):813-816. Olney NT, Alquezar C, Ramos EM, Nana AL, Fong JC, Karydas AM, Taylor JB, Stephens ML, Argouarch AR, Van Berlo VA, Dokuru DR, Sherr EH, Jicha GA, Dillon WP, Desikan RS, De May M, Seeley WW, Coppola G, Miller BL, Kao AW. PMID: 28828560; PMCID: PMC5645431.
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Progranulin, lysosomal regulation and neurodegenerative disease. Nat Rev Neurosci. 2017 06; 18(6):325-333. Kao AW, McKay A, Singh PP, Brunet A, Huang EJ. PMID: 28435163; PMCID: PMC6040832.
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Animal models of dementia. The Behavioral Neurology of Dementia. 2017 Jan 1; 77-93. Roberson RE, Kao KA. .
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MCP-1 and eotaxin-1 selectively and negatively associate with memory in MCI and Alzheimer's disease dementia phenotypes. Alzheimers Dement (Amst). 2016; 3:91-7. Bettcher BM, Fitch R, Wynn MJ, Lalli MA, Elofson J, Jastrzab L, Mitic L, Miller ZA, Rabinovici GD, Miller BL, Kao AW, Kosik KS, Kramer JH. PMID: 27453930; PMCID: PMC4941041.
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The Progranulin Cleavage Products, Granulins, Exacerbate TDP-43 Toxicity and Increase TDP-43 Levels. J Neurosci. 2015 Jun 24; 35(25):9315-28. Salazar DA, Butler VJ, Argouarch AR, Hsu TY, Mason A, Nakamura A, McCurdy H, Cox D, Ng R, Pan G, Seeley WW, Miller BL, Kao AW. PMID: 26109656; PMCID: PMC4478251.
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A shift to organismal stress resistance in programmed cell death mutants. PLoS Genet. 2013; 9(9):e1003714. Judy ME, Nakamura A, Huang A, Grant H, McCurdy H, Weiberth KF, Gao F, Coppola G, Kenyon C, Kao AW. PMID: 24068943; PMCID: PMC3778000.
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The advantages of frontotemporal degeneration drug development (part 2 of frontotemporal degeneration: the next therapeutic frontier). Alzheimers Dement. 2013 Mar; 9(2):189-98. Boxer AL, Gold M, Huey E, Hu WT, Rosen H, Kramer J, Gao FB, Burton EA, Chow T, Kao A, Leavitt BR, Lamb B, Grether M, Knopman D, Cairns NJ, Mackenzie IR, Mitic L, Roberson ED, Van Kammen D, Cantillon M, Zahs K, Jackson G, Salloway S, Morris J, Tong G, Feldman H, Fillit H, Dickinson S, Khachaturian ZS, Sutherland M, Abushakra S, Lewcock J, Farese R, Kenet RO, Laferla F, Perrin S, Whitaker S, Honig L, Mesulam MM, Boeve B, Grossman M, Miller BL, Cummings JL. PMID: 23062850; PMCID: PMC3562382.
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Frontotemporal degeneration, the next therapeutic frontier: molecules and animal models for frontotemporal degeneration drug development. Alzheimers Dement. 2013 Mar; 9(2):176-88. Boxer AL, Gold M, Huey E, Gao FB, Burton EA, Chow T, Kao A, Leavitt BR, Lamb B, Grether M, Knopman D, Cairns NJ, Mackenzie IR, Mitic L, Roberson ED, Van Kammen D, Cantillon M, Zahs K, Salloway S, Morris J, Tong G, Feldman H, Fillit H, Dickinson S, Khachaturian Z, Sutherland M, Farese R, Miller BL, Cummings J. PMID: 23043900; PMCID: PMC3542408.
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A neurodegenerative disease mutation that accelerates the clearance of apoptotic cells. Proc Natl Acad Sci U S A. 2011 Mar 15; 108(11):4441-6. Kao AW, Eisenhut RJ, Martens LH, Nakamura A, Huang A, Bagley JA, Zhou P, de Luis A, Neukomm LJ, Cabello J, Farese RV, Kenyon C. PMID: 21368173; PMCID: PMC3060230.
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Cognitive and neuropsychiatric profile of the synucleinopathies: Parkinson disease, dementia with Lewy bodies, and multiple system atrophy. Alzheimer Dis Assoc Disord. 2009 Oct-Dec; 23(4):365-70. Kao AW, Racine CA, Quitania LC, Kramer JH, Christine CW, Miller BL. PMID: 19935145; PMCID: PMC2886667.
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Animal models of dementia. The Behavioral Neurology of Dementia. 2009 Jan 1; 131-141. Roberson RE, Kao KA. .
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Chemokine receptors, neural progenitor cells, and the AIDS dementia complex. J Infect Dis. 2004 Jul 15; 190(2):211-5. Kao AW, Price RW. PMID: 15216453.
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Functional comparison of the role of dynamin 2 splice variants on GLUT-4 endocytosis in 3T3L1 adipocytes. Am J Physiol Endocrinol Metab. 2000 May; 278(5):E825-31. Kao AW, Yang C, Pessin JE. PMID: 10780938.
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Dynamin is required for recombinant adeno-associated virus type 2 infection. J Virol. 1999 Dec; 73(12):10371-6. Duan D, Li Q, Kao AW, Yue Y, Pessin JE, Engelhardt JF. PMID: 10559355; PMCID: PMC113092.
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The role of glycogen synthase kinase 3beta in insulin-stimulated glucose metabolism. J Biol Chem. 1999 Jun 18; 274(25):17934-40. Summers SA, Kao AW, Kohn AD, Backus GS, Roth RA, Pessin JE, Birnbaum MJ. PMID: 10364240.
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Aldolase mediates the association of F-actin with the insulin-responsive glucose transporter GLUT4. J Biol Chem. 1999 Jun 18; 274(25):17742-7. Kao AW, Noda Y, Johnson JH, Pessin JE, Saltiel AR. PMID: 10364216.
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Expression of a dominant interfering dynamin mutant in 3T3L1 adipocytes inhibits GLUT4 endocytosis without affecting insulin signaling. J Biol Chem. 1998 Sep 25; 273(39):25450-7. Kao AW, Ceresa BP, Santeler SR, Pessin JE. PMID: 9738014.
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Inhibition of clathrin-mediated endocytosis selectively attenuates specific insulin receptor signal transduction pathways. Mol Cell Biol. 1998 Jul; 18(7):3862-70. Ceresa BP, Kao AW, Santeler SR, Pessin JE. PMID: 9632770; PMCID: PMC108970.
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The 66-kDa Shc isoform is a negative regulator of the epidermal growth factor-stimulated mitogen-activated protein kinase pathway. J Biol Chem. 1997 Oct 31; 272(44):28042-9. Okada S, Kao AW, Ceresa BP, Blaikie P, Margolis B, Pessin JE. PMID: 9346957.
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Insulin stimulates the phosphorylation of the 66- and 52-kilodalton Shc isoforms by distinct pathways. Endocrinology. 1997 Jun; 138(6):2474-80. Kao AW, Waters SB, Okada S, Pessin JE. PMID: 9165038.
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Insulin Stimulates the Phosphorylation of the 66- and 52-Kilodalton Shc Isoforms by Distinct Pathways*. Endocrinology. 1997 Jun 1; 138(6):2474-2480. Kao KA, Waters WS, Okada OS, Pessin PJ. .
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Insulin and epidermal growth factor receptors regulate distinct pools of Grb2-SOS in the control of Ras activation. J Biol Chem. 1996 Jul 26; 271(30):18224-30. Waters SB, Chen D, Kao AW, Okada S, Holt KH, Pessin JE. PMID: 8663461.
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Evidence for myosin motors on organelles in squid axoplasm. Proc Natl Acad Sci U S A. 1993 Dec 01; 90(23):11252-6. Bearer EL, DeGiorgis JA, Bodner RA, Kao AW, Reese TS. PMID: 8248236; PMCID: PMC47960.
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