Research Summary

The focus of my research group is the protein homeostasis network, a network of over 1,000 different factors in human cells that maintain the proteome in a functional and balanced state. Our goal is to understand how this network functions, and how it is challenged and rewired in disease states, in particular cancer and neurodegenerative diseases. Our functional genomics platform enables us to elucidate dynamic networks and to pinpoint nodes that are potential therapeutic targets. Using biochemistry, biophysics and cell biology, we then "zoom in" on individual nodes of the network to reveal their mechanism of action.

As a postdoctoral scholar in Jonathan Weissman’s lab at UCSF, I spearheaded the development of a functional genomics platform for robust genome-wide screens and genetic interaction maps in mammalian cells (see section C.2 below). Very recently, we have enhanced this platform by developing CRISPR-based technologies enabling both loss-of-function and gain-of-function screens in mammalian cells (see section C.3 below). I am confident that our functional genomics approach will have a particularly high impact in its application to elucidate cancer-relevant cellular pathways. We have successfully used this approach to identify cellular factors that control the sensitivity of cancer cells to drugs (see section C.4 below). This approach can identify biomarkers predictive of clinical responses, predict possible mechanisms of drug resistance, and guide the development of combination therapies.

Research Funding

  • September 1, 2021 - August 31, 2026 - Uncovering the genetic mechanisms of the Chromosome 17q21.31 Tau haplotype on neurodegeneration risk in FTD and PSP , Co-Principal Investigator . Sponsor: NIH, Sponsor Award ID: U54NS123746
  • July 1, 2021 - June 30, 2026 - Investigating the Functional Impact of AD Risk Genes on Neuro-Vascular Interactions , Co-Principal Investigator . Sponsor: NIH, Sponsor Award ID: U01AG072464
  • September 5, 2018 - June 30, 2023 - The Psychiatric Cell Map Initiative: Connecting Genomics, Subcellular Networks, and Higher Order Phenotypes , Co-Principal Investigator . Sponsor: NIH, Sponsor Award ID: U01MH115747
  • September 30, 2018 - May 31, 2023 - Systematic elucidation of endosomal trafficking as a therapeutic opportunity in AD using CRISPR-based functional genomics , Principal Investigator . Sponsor: NIH, Sponsor Award ID: R01AG062359


Marburg University, Germany, Pre-diploma, 06/00, Human Biology
Cambridge University, UK, B.A. (M.A.), First Class Honours, 06/03, Biochemistry
The Rockefeller University, Ph.D., 06/09, Biological Sciences (Cell Biology, Biophysics)
University of California, San Francisco, Postdoctoral Fellow, 07/15, Systems Biology

Honors & Awards

  • 2017
    Named to first cohort of Chan Zuckerberg Biohub Investigators
  • 2016
    Innovative Research Grant, Stand Up To Cancer
  • 2015
    New Innovator Award, NIH
  • 2015
    Allen Distinguished Investigator Award, Paul G. Allen Family Foundation
  • 2014
    Pathway to Independence Award (K99/R00), NIH
  • 2012
    Finalist, Deloitte QB3 Award for Innovation, California Institute for Quantitative Biosciences
  • 2012
    PBBR Postdoctoral Research Fellowship, Program for Breakthrough Biomedical Research, UCSF
  • 2010
    Postdoctoral Fellowship, Jane Coffin Childs Memorial Fund
  • 2006
    David Rockefeller Fellowship, The Rockefeller University
  • 2003
    Predoctoral Fellowship, Howard Hughes Medical Institute

Selected Publications

  1. de Majo M, Koontz M, Marsan E, Salinas N, Ramsey A, Kuo YM, Seo K, Li H, Dr?ger N, Leng K, Gonzales SL, Kurnellas M, Miyaoka Y, Klim JR, Kampmann M, Ward ME, Huang EJ, Ullian EM. Granulin loss of function in human mature brain organoids implicates astrocytes in TDP-43 pathology. Stem Cell Reports. 2023 Feb 14.  View on PubMed
  2. Rheinberger M, Costa AL, Kampmann M, Glavas D, Shytaj IL, Sreeram S, Penzo C, Tibroni N, Garcia-Mesa Y, Leskov K, Fackler OT, Vlahovicek K, Karn J, Lucic B, Herrmann C, Lusic M. Genomic profiling of HIV-1 integration in microglia cells links viral integration to the topologically associated domains. Cell Rep. 2023 Feb 13; 42(2):112110.  View on PubMed
  3. You SF, Brase L, Filipello F, Iyer AK, Del-Aguila J, He J, D'Oliveira Albanus R, Budde J, Norton J, Gentsch J, Dr?ger NM, Sattler SM, Kampmann M, Piccio L, Morris JC, Perrin RJ, McDade E, Dominantly Inherited Alzheimer Network, Paul SM, Cashikar AG, Benitez BA, Harari O, Karch CM. MS4A4A modifies the risk of Alzheimer disease by regulating lipid metabolism and immune response in a unique microglia state. medRxiv. 2023 Feb 08.  View on PubMed
  4. Oltion K, Carelli JD, Yang T, See SK, Wang HY, Kampmann M, Taunton J. An E3 ligase network engages GCN1 to promote the degradation of translation factors on stalled ribosomes. Cell. 2023 Jan 19; 186(2):346-362.e17.  View on PubMed
  5. Replogle JM, Bonnar JL, Pogson AN, Liem CR, Maier NK, Ding Y, Russell BJ, Wang X, Leng K, Guna A, Norman TM, Pak RA, Ramos DM, Ward ME, Gilbert LA, Kampmann M, Weissman JS, Jost M. Maximizing CRISPRi efficacy and accessibility with dual-sgRNA libraries and optimal effectors. Elife. 2022 12 28; 11.  View on PubMed
  6. Pantazis CB, Yang A, Lara E, McDonough JA, Blauwendraat C, Peng L, Oguro H, Kanaujiya J, Zou J, Sebesta D, Pratt G, Cross E, Blockwick J, Buxton P, Kinner-Bibeau L, Medura C, Tompkins C, Hughes S, Santiana M, Faghri F, Nalls MA, Vitale D, Ballard S, Qi YA, Ramos DM, Anderson KM, Stadler J, Narayan P, Papademetriou J, Reilly L, Nelson MP, Aggarwal S, Rosen LU, Kirwan P, Pisupati V, Coon SL, Scholz SW, Priebe T, ?ttl M, Dong J, Meijer M, Janssen LJM, Lourenco VS, van der Kant R, Crusius D, Paquet D, Raulin AC, Bu G, Held A, Wainger BJ, Gabriele RMC, Casey JM, Wray S, Abu-Bonsrah D, Parish CL, Beccari MS, Cleveland DW, Li E, Rose IVL, Kampmann M, Calatayud Aristoy C, Verstreken P, Heinrich L, Chen MY, Sch?le B, Dou D, Holzbaur ELF, Zanellati MC, Basundra R, Deshmukh M, Cohen S, Khanna R, Raman M, Nevin ZS, Matia M, Van Lent J, Timmerman V, Conklin BR, Johnson Chase K, Zhang K, Funes S, Bosco DA, Erlebach L, Welzer M, Kronenberg-Versteeg D, Lyu G, Arenas E, Coccia E, Sarrafha L, Ahfeldt T, Marioni JC, Skarnes WC, Cookson MR, Ward ME, Merkle FT. A reference human induced pluripotent stem cell line for large-scale collaborative studies. Cell Stem Cell. 2022 12 01; 29(12):1685-1702.e22.  View on PubMed
  7. Leng K, Kampmann M. Towards elucidating disease-relevant states of neurons and glia by CRISPR-based functional genomics. Genome Med. 2022 11 18; 14(1):130.  View on PubMed
  8. Kim H, Leng K, Park J, Sorets AG, Kim S, Shostak A, Embalabala RJ, Mlouk K, Katdare KA, Rose IVL, Sturgeon SM, Neal EH, Ao Y, Wang S, Sofroniew MV, Brunger JM, McMahon DG, Schrag MS, Kampmann M, Lippmann ES. Reactive astrocytes transduce inflammation in a blood-brain barrier model through a TNF-STAT3 signaling axis and secretion of alpha 1-antichymotrypsin. Nat Commun. 2022 11 02; 13(1):6581.  View on PubMed
  9. Leng K, Rose IVL, Kim H, Xia W, Romero-Fernandez W, Rooney B, Koontz M, Li E, Ao Y, Wang S, Krawczyk M, Tcw J, Goate A, Zhang Y, Ullian EM, Sofroniew MV, Fancy SPJ, Schrag MS, Lippmann ES, Kampmann M. CRISPRi screens in human iPSC-derived astrocytes elucidate regulators of distinct inflammatory reactive states. Nat Neurosci. 2022 11; 25(11):1528-1542.  View on PubMed
  10. Krause GJ, Diaz A, Jafari M, Khawaja RR, Agullo-Pascual E, Santiago-Fernández O, Richards AL, Chen KH, Dmitriev P, Sun Y, See SK, Abdelmohsen K, Mazan-Mamczarz K, Krogan NJ, Gorospe M, Swaney DL, Sidoli S, Bravo-Cordero JJ, Kampmann M, Cuervo AM. Reduced endosomal microautophagy activity in aging associates with enhanced exocyst-mediated protein secretion. Aging Cell. 2022 Oct; 21(10):e13713.  View on PubMed
  11. Cooper YA, Teyssier N, Dräger NM, Guo Q, Davis JE, Sattler SM, Yang Z, Patel A, Wu S, Kosuri S, Coppola G, Kampmann M, Geschwind DH. Functional regulatory variants implicate distinct transcriptional networks in dementia. Science. 2022 Aug 19; 377(6608):eabi8654.  View on PubMed
  12. Dr?ger NM, Sattler SM, Huang CT, Teter OM, Leng K, Hashemi SH, Hong J, Aviles G, Clelland CD, Zhan L, Udeochu JC, Kodama L, Singleton AB, Nalls MA, Ichida J, Ward ME, Faghri F, Gan L, Kampmann M. A CRISPRi/a platform in human iPSC-derived microglia uncovers regulators of disease states. Nat Neurosci. 2022 09; 25(9):1149-1162.  View on PubMed
  13. Chin MY, Ang KH, Davies J, Alquezar C, Garda VG, Rooney B, Leng K, Kampmann M, Arkin MR, Kao AW. 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.  View on PubMed
  14. Bristow RG, Engel J, Jayasinghe I, Kampmann M, James Sansom O, Bryant DM. Conversations with LGBT+ scientists about visibility, leadership and climbing the career ladder. J Cell Sci. 2022 02 15; 135(4).  View on PubMed
  15. Shkedi A, Taylor IR, Echtenkamp F, Ramkumar P, Alshalalfa M, Rivera-M?rquez GM, Moses MA, Shao H, Karnes RJ, Neckers L, Feng F, Kampmann M, Gestwicki JE. Selective vulnerabilities in the proteostasis network of castration-resistant prostate cancer. Cell Chem Biol. 2022 03 17; 29(3):490-501.e4.  View on PubMed
  16. Biebl MM, Delhommel F, Faust O, Zak KM, Agam G, Guo X, M?hlhofer M, Dahiya V, Hillebrand D, Popowicz GM, Kampmann M, Lamb DC, Rosenzweig R, Sattler M, Buchner J. NudC guides client transfer between the Hsp40/70 and Hsp90 chaperone systems. Mol Cell. 2022 02 03; 82(3):555-569.e7.  View on PubMed
  17. Samelson AJ, Tran QD, Robinot R, Carrau L, Rezelj VV, Kain AM, Chen M, Ramadoss GN, Guo X, Lim SA, Lui I, Nu?ez JK, Rockwood SJ, Wang J, Liu N, Carlson-Stevermer J, Oki J, Maures T, Holden K, Weissman JS, Wells JA, Conklin BR, TenOever BR, Chakrabarti LA, Vignuzzi M, Tian R, Kampmann M. BRD2 inhibition blocks SARS-CoV-2 infection by reducing transcription of the host cell receptor ACE2. Nat Cell Biol. 2022 01; 24(1):24-34.  View on PubMed
  18. Guo X, Kampmann M. CRISPR-Based Screening for Stress Response Factors in Mammalian Cells. Methods Mol Biol. 2022; 2428:19-40.  View on PubMed
  19. Dellweg A, Kampmann M, Tschopp K. Evaluation of a nasopharyngeal stent in patients with obstructive sleep-related breathing disorders. J Int Med Res. 2022 Jan; 50(1):3000605211073302.  View on PubMed
  20. Alexander J. Ehrenberg, Kun Leng, Kaitlin Letourneau, Israel Hernandez, Caroline Lew, William W. Seeley, Salvatore Spina, Bruce L. Miller, Helmut Heinsen, Martin Kampmann, Kenneth S. Kosik, Lea T. Grinberg. The Lonafarnib target, Rhes, is uniquely dysregulated in tauopathies: A human postmortem study. Alzheimer's & Dementia. 2021 Dec 31; 17(S5).  View on PubMed

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