Aimee Kao, MD, PhD

John Douglas French Foundation Endowed Professor

+1 415 502-7123

Fields of Interest:

Aimee Kao, MD, PhD, is a Professor of Neurology at the University of California, San Francisco and the John Douglas French Foundation Endowed Professor. She leads an NIH-supported Tau Center Without Walls and directs the UCSF Tau Consortium Human Fibroblast Bank. Dr. Kao’s clinical expertise includes the diagnosis and treatment of Alzheimer’s disease, vascular dementia, Lewy body disease and frontotemporal lobar degeneration. Her basic science laboratory studies how age, stress and pH changes affect protein homeostasis and contribute to sporadic and familial neurodegenerative disorders. She has received the Paul G. Allen Family Foundation Distinguished Investigator Award in Neurodegenerative Diseases, the Glenn Award for Research in the Biological Mechanisms of Aging and the Derek Denny Brown Young Neurological Scholar Award.

Publications:

The progranulin cleavage product granulin 3 exerts a dominant negative effect on animal fitness.

Human molecular genetics

Wang AL, Mambou EA, Kao AW

Correction: Mutations in α-synuclein, TDP-43 and tau prolong protein half-life through diminished degradation by lysosomal proteases.

Molecular neurodegeneration

Sampognaro PJ, Arya S, Knudsen GM, Gunderson EL, Sandoval-Perez A, Hodul M, Bowles K, Craik CS, Jacobson MP, Kao AW

Mutations in a-synuclein, TDP-43 and tau prolong protein half-life through diminished degradation by lysosomal proteases.

Molecular neurodegeneration

Sampognaro PJ, Arya S, Knudsen GM, Gunderson EL, Sandoval-Perez A, Hodul M, Bowles K, Craik CS, Jacobson MP, Kao AW

Tuberous sclerosis complex is associated with a novel human tauopathy.

Acta neuropathologica

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

Postmortem Human Dura Mater Cells Exhibit Phenotypic, Transcriptomic and Genetic Abnormalities that Impact their Use for Disease Modeling.

Stem cell reviews and reports

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

Phenotypic Screening Using High-Content Imaging to Identify Lysosomal pH Modulators in a Neuronal Cell Model.

ACS chemical neuroscience

Chin MY, Ang KH, Davies J, Alquezar C, Garda VG, Rooney B, Leng K, Kampmann M, Arkin MR, Kao AW

TSC1 loss increases risk for tauopathy by inducing tau acetylation and preventing tau clearance via chaperone-mediated autophagy.

Science advances

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

Processing of progranulin into granulins involves multiple lysosomal proteases and is affected in frontotemporal lobar degeneration.

Molecular neurodegeneration

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

Genetically Encoded, pH-Sensitive mTFP1 Biosensor for Probing Lysosomal pH.

ACS sensors

Chin MY, Patwardhan AR, Ang KH, Wang AL, Alquezar C, Welch M, Nguyen PT, Grabe M, Molofsky AV, Arkin MR, Kao AW

Tau Post-translational Modifications: Dynamic Transformers of Tau Function, Degradation, and Aggregation.

Frontiers in neurology

Alquezar C, Arya S, Kao AW

Progranulin Adsorbs to Polypropylene Tubes and Disrupts Functional Assays: Implications for Research, Biomarker Studies, and Therapeutics.

Frontiers in neuroscience

Gururaj S, Sampognaro PJ, Argouarch AR, Kao AW

Association of Cognitive and Behavioral Features Between Adults With Tuberous Sclerosis and Frontotemporal Dementia.

JAMA neurology

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

Heavy metals contaminating the environment of a progressive supranuclear palsy cluster induce tau accumulation and cell death in cultured neurons.

Scientific reports

Alquezar C, Felix JB, McCandlish E, Buckley BT, Caparros-Lefebvre D, Karch CM, Golbe LI, Kao AW

Protons in small spaces: Discrete simulations of vesicle acidification.

PLoS computational biology

Singh A, Marcoline FV, Veshaguri S, Kao AW, Bruchez M, Mindell JA, Stamou D, Grabe M

A Comprehensive Resource for Induced Pluripotent Stem Cells from Patients with Primary Tauopathies.

Stem cell reports

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

Lifespan-increasing drug nordihydroguaiaretic acid inhibits p300 and activates autophagy.

NPJ aging and mechanisms of disease

Tezil T, Chamoli M, Ng CP, Simon RP, Butler VJ, Jung M, Andersen J, Kao AW, Verdin E

Age- and stress-associated C. elegans granulins impair lysosomal function and induce a compensatory HLH-30/TFEB transcriptional response.

PLoS genetics

Butler VJ, Gao F, Corrales CI, Cortopassi WA, Caballero B, Vohra M, Ashrafi K, Cuervo AM, Jacobson MP, Coppola G, Kao AW

Multi-Granulin Domain Peptides Bind to Pro-Cathepsin D and Stimulate Its Enzymatic Activity More Effectively Than Progranulin in Vitro.

Biochemistry

Butler VJ, Cortopassi WA, Gururaj S, Wang AL, Pierce OM, Jacobson MP, Kao AW

Tau/MAPT disease-associated variant A152T alters tau function and toxicity via impaired retrograde axonal transport.

Human molecular genetics

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

Progranulin Stimulates the in vitro Maturation of pro-Cathepsin D at Acidic pH.

Journal of molecular biology

Butler VJ, Cortopassi WA, Argouarch AR, Ivry SL, Craik CS, Jacobson MP, Kao AW

Tauopathy-Associated PERK Alleles are Functional Hypomorphs that Increase Neuronal Vulnerability to ER Stress.

Human molecular genetics

Yuan SH, Hiramatsu N, Liu Q, Sun XV, Lenh D, Chan P, Chiang K, Koo EH, Kao AS, Litvan I, Lin JH

Regionally specific TSC1 and TSC2 gene expression in tuberous sclerosis complex.

Scientific reports

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

The Use of Caenorhabditis elegans to Study Progranulin in the Regulation of Programmed Cell Death and Stress Response.

Methods in molecular biology (Clifton, N.J.)

Hsu TY, Butler VJ, Kao AW

Clinicopathological correlations in behavioural variant frontotemporal dementia.

Brain : a journal of neurology

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

Linking tuberous sclerosis complex, excessive mTOR signaling, and age-related neurodegeneration: a new association between TSC1 mutation and frontotemporal dementia.

Acta neuropathologica

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

Progranulin, lysosomal regulation and neurodegenerative disease.

Nature reviews. Neuroscience

Kao AW, McKay A, Singh PP, Brunet A, Huang EJ

Animal models of dementia.

Erik D. Roberson,Aimee W. Kao

MCP-1 and eotaxin-1 selectively and negatively associate with memory in MCI and Alzheimer's disease dementia phenotypes.

Alzheimer's & dementia (Amsterdam, Netherlands)

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

The Progranulin Cleavage Products, Granulins, Exacerbate TDP-43 Toxicity and Increase TDP-43 Levels.

The Journal of neuroscience : the official journal of the Society for Neuroscience

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

A shift to organismal stress resistance in programmed cell death mutants.

PLoS genetics

Judy ME, Nakamura A, Huang A, Grant H, McCurdy H, Weiberth KF, Gao F, Coppola G, Kenyon C, Kao AW

The advantages of frontotemporal degeneration drug development (part 2 of frontotemporal degeneration: the next therapeutic frontier).

Alzheimer's & dementia : the journal of the Alzheimer's Association

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

Frontotemporal degeneration, the next therapeutic frontier: molecules and animal models for frontotemporal degeneration drug development.

Alzheimer's & dementia : the journal of the Alzheimer's Association

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