Research Summary

Our lab's goal is to understand the genetic circuits that control human immune cell function in health and disease. We have begun to identify how genetic risk variants for autoimmune diseases disrupt immune cell circuits (Farh and Marson et al., Nature 2015; Simeonov et al., Nature, 2017), and how pathogenic circuits may be targeted with novel therapeutics (Xiao et al., Immunity 2014). My lab has developed new tools for efficient CRISPR genome engineering in primary human T cells (Schumann et al., PNAS 2015; Roth et al., Nature 2018; Nguyen et al. Nature Biotech 2020). Now we are pursuing a comprehensive strategy to test how coding and non-coding genetic variation control essential programs in the human immune system (Simeonov et al. Nature, 2017; Shifrut et al., Cell 2018; Roth et al., Cell 2020; Cortez et al., Nature 2020; Schumann et al., Nature Immunology 2020). Genome engineered human T cells hold great potential for the next generation of cell-based therapies for cancer, autoimmunity and infectious diseases.

Research Funding

  • September 8, 2020 - August 31, 2025 - Inherited T cell defects: Diagnosis, Mechanisms and Treatments, Principal Investigator. Sponsor: NIH/NIAID, Sponsor Award ID: P01AI138962
  • September 18, 2018 - May 31, 2022 - Functional Molecular Investigation of Inflammatory Bowel Disease (IBD) Risk Variants, Principal Investigator. Sponsor: NIH/NIDDK, Sponsor Award ID: R01DK119979
  • September 30, 2016 - June 30, 2021 - Functional Interrogation of Non-Coding Type 1 Diabetes Risk Variants in Human Immune Cells and Beta Cells, Principal Investigator. Sponsor: NIH/NIDDK, Sponsor Award ID: DP3DK111914
  • June 1, 2016 - May 31, 2021 - Functional Testing of Host Genes that control HIV Latency in Primary Immune Cells, Principal Investigator. Sponsor: NIH/NIDA, Sponsor Award ID: DP2DA042423

Education

Harvard College, MA, A.B., 2001, Biology
Cambridge University, UK, M.Phil., 2003, Biological Sciences
Massachusetts Institute of Technology, MA, Ph.D., 2008, Biology
Harvard Medical School, MA, M.D., 2010, Medicine
Brigham and Women’s Hospital, Residency, 2012, Internal Medicine
UC San Francisco, Fellowship, 2016, Infectious Diseases

Honors & Awards

  • 1998
    Jacob Wendell Prize for most promising freshman scholar, Harvard College
  • 1998
    Detur Book Prize, Harvard College
  • 2000
    William and Mary Bossert Prize, Harvard College
  • 2001
    Phi Beta Kappa, Harvard College
  • 2001-2002
    Herchel Smith Harvard Scholar, Emmanuel College, Cambridge, UK
  • 2009
    Sigma Xi 2009, MIT
  • 2010
    James Tolbert Shipley Prize for excellence and accomplishment in research, Harvard Medical
  • 2016
    American Society of Clinical Investigation (ASCI) Young Physician Scientist Award
  • 2016
    Burroughs Wellcome Foundation Career Award for Medical Scientists
  • 2016
    NIDA/NIH Avenir New Innovator Award
  • 2017
    Named to first cohort of Chan Zuckerberg Biohub Investigators
  • 2018
    Elected Member, American Society of Clinical Investors (ASCI)
  • 2019
    Lloyd J. Old STAR Award, Cancer Research Institute (CRI)

Selected Publications

  1. Baglaenko Y, Macfarlane D, Marson A, Nigrovic PA, Raychaudhuri S. Genome editing to define the function of risk loci and variants in rheumatic disease. Nat Rev Rheumatol. 2021 Jun 29.  View on PubMed
  2. Gallman AE, Wolfreys FD, Nguyen DN, Sandy M, Xu Y, An J, Li Z, Marson A, Lu E, Cyster JG. Abcc1 and Ggt5 support lymphocyte guidance through export and catabolism of S-geranylgeranyl-l-glutathione. Sci Immunol. 2021 Jun 04; 6(60).  View on PubMed
  3. Hamilton JR, Tsuchida CA, Nguyen DN, Shy BR, McGarrigle ER, Sandoval Espinoza CR, Carr D, Blaeschke F, Marson A, Doudna JA. Targeted delivery of CRISPR-Cas9 and transgenes enables complex immune cell engineering. Cell Rep. 2021 Jun 01; 35(9):109207.  View on PubMed
  4. Hiatt J, Cavero DA, McGregor MJ, Zheng W, Budzik JM, Roth TL, Haas KM, Wu D, Rathore U, Meyer-Franke A, Bouzidi MS, Shifrut E, Lee Y, Kumar VE, Dang EV, Gordon DE, Wojcechowskyj JA, Hultquist JF, Fontaine KA, Pillai SK, Cox JS, Ernst JD, Krogan NJ, Marson A. Efficient generation of isogenic primary human myeloid cells using CRISPR-Cas9 ribonucleoproteins. Cell Rep. 2021 May 11; 35(6):109105.  View on PubMed
  5. Lee Y, Bogdanoff D, Wang Y, Hartoularos GC, Woo JM, Mowery CT, Nisonoff HM, Lee DS, Sun Y, Lee J, Mehdizadeh S, Cantlon J, Shifrut E, Ngyuen DN, Roth TL, Song YS, Marson A, Chow ED, Ye CJ. XYZeq: Spatially resolved single-cell RNA sequencing reveals expression heterogeneity in the tumor microenvironment. Sci Adv. 2021 Apr; 7(17).  View on PubMed
  6. Muller YD, Nguyen DP, Ferreira LMR, Ho P, Raffin C, Valencia RVB, Congrave-Wilson Z, Roth TL, Eyquem J, Van Gool F, Marson A, Perez L, Wells JA, Bluestone JA, Tang Q. The CD28-Transmembrane Domain Mediates Chimeric Antigen Receptor Heterodimerization With CD28. Front Immunol. 2021; 12:639818.  View on PubMed
  7. Siddiqui S, Johansson K, Joo A, Bonser LR, Koh KD, Le Tonqueze O, Bolourchi S, Bautista RA, Zlock L, Roth TL, Marson A, Bhakta NR, Ansel KM, Finkbeiner WE, Erle DJ, Woodruff PG. Epithelial miR-141 regulates IL-13-induced airway mucus production. JCI Insight. 2021 Mar 08; 6(5).  View on PubMed
  8. Taylor KE, Ansel KM, Marson A, Criswell LA, Farh KK. PICS2: Next-generation fine mapping via probabilistic identification of causal SNPs. Bioinformatics. 2021 Feb 24.  View on PubMed
  9. Mathewson ND, Ashenberg O, Tirosh I, Gritsch S, Perez EM, Marx S, Jerby-Arnon L, Chanoch-Myers R, Hara T, Richman AR, Ito Y, Pyrdol J, Friedrich M, Schumann K, Poitras MJ, Gokhale PC, Gonzalez Castro LN, Shore ME, Hebert CM, Shaw B, Cahill HL, Drummond M, Zhang W, Olawoyin O, Wakimoto H, Rozenblatt-Rosen O, Brastianos PK, Liu XS, Jones PS, Cahill DP, Frosch MP, Louis DN, Freeman GJ, Ligon KL, Marson A, Chiocca EA, Reardon DA, Regev A, Suvà ML, Wucherpfennig KW. Inhibitory CD161 receptor identified in glioma-infiltrating T cells by single-cell analysis. Cell. 2021 Mar 04; 184(5):1281-1298.e26.  View on PubMed
  10. Rutishauser RL, Deguit CDT, Hiatt J, Blaeschke F, Roth TL, Wang L, Raymond KA, Starke CE, Mudd JC, Chen W, Smullin C, Matus-Nicodemos R, Hoh R, Krone M, Hecht FM, Pilcher CD, Martin JN, Koup RA, Douek DC, Brenchley JM, Sékaly RP, Pillai SK, Marson A, Deeks SG, McCune JM, Hunt PW. TCF-1 regulates HIV-specific CD8+ T cell expansion capacity. JCI Insight. 2021 02 08; 6(3).  View on PubMed
  11. Roth TL, Marson A. Genetic Disease and Therapy. Annu Rev Pathol. 2021 01 24; 16:145-166.  View on PubMed
  12. Baars MJD, Douma T, Simeonov DR, Myers DR, Kulhanek K, Banerjee S, Zwakenberg S, Baltissen MP, Amini M, de Roock S, van Wijk F, Vermeulen M, Marson A, Roose JP, Vercoulen Y. Dysregulated RASGRP1 expression through RUNX1 mediated transcription promotes autoimmunity. Eur J Immunol. 2021 02; 51(2):471-482.  View on PubMed
  13. Kang HM, Subramaniam M, Targ S, Nguyen M, Maliskova L, McCarthy E, Wan E, Wong S, Byrnes L, Lanata CM, Gate RE, Mostafavi S, Marson A, Zaitlen N, Criswell LA, Ye CJ. Author Correction: Multiplexed droplet single-cell RNA-sequencing using natural genetic variation. Nat Biotechnol. 2020 Nov; 38(11):1356.  View on PubMed
  14. Schumann K, Raju SS, Lauber M, Kolb S, Shifrut E, Cortez JT, Skartsis N, Nguyen VQ, Woo JM, Roth TL, Yu R, Nguyen MLT, Simeonov DR, Nguyen DN, Targ S, Gate RE, Tang Q, Bluestone JA, Spitzer MH, Ye CJ, Marson A. Functional CRISPR dissection of gene networks controlling human regulatory T cell identity. Nat Immunol. 2020 11; 21(11):1456-1466.  View on PubMed
  15. Mahuron KM, Moreau JM, Glasgow JE, Boda DP, Pauli ML, Gouirand V, Panjabi L, Grewal R, Luber JM, Mathur AN, Feldman RM, Shifrut E, Mehta P, Lowe MM, Alvarado MD, Marson A, Singer M, Wells J, Jupp R, Daud AI, Rosenblum MD. Layilin augments integrin activation to promote antitumor immunity. J Exp Med. 2020 09 07; 217(9).  View on PubMed
  16. Tan C, Hiwa R, Mueller JL, Vykunta V, Hibiya K, Noviski M, Huizar J, Brooks JF, Garcia J, Heyn C, Li Z, Marson A, Zikherman J. NR4A nuclear receptors restrain B cell responses to antigen when second signals are absent or limiting. Nat Immunol. 2020 10; 21(10):1267-1279.  View on PubMed
  17. Whitman JD, Hiatt J, Mowery CT, Shy BR, Yu R, Yamamoto TN, Rathore U, Goldgof GM, Whitty C, Woo JM, Gallman AE, Miller TE, Levine AG, Nguyen DN, Bapat SP, Balcerek J, Bylsma SA, Lyons AM, Li S, Wong AW, Gillis-Buck EM, Steinhart ZB, Lee Y, Apathy R, Lipke MJ, Smith JA, Zheng T, Boothby IC, Isaza E, Chan J, Acenas DD, Lee J, Macrae TA, Kyaw TS, Wu D, Ng DL, Gu W, York VA, Eskandarian HA, Callaway PC, Warrier L, Moreno ME, Levan J, Torres L, Farrington LA, Loudermilk RP, Koshal K, Zorn KC, Garcia-Beltran WF, Yang D, Astudillo MG, Bernstein BE, Gelfand JA, Ryan ET, Charles RC, Iafrate AJ, Lennerz JK, Miller S, Chiu CY, Stramer SL, Wilson MR, Manglik A, Ye CJ, Krogan NJ, Anderson MS, Cyster JG, Ernst JD, Wu AHB, Lynch KL, Bern C, Hsu PD, Marson A. Evaluation of SARS-CoV-2 serology assays reveals a range of test performance. Nat Biotechnol. 2020 10; 38(10):1174-1183.  View on PubMed
  18. Dong R, Libby KA, Blaeschke F, Fuchs W, Marson A, Vale RD, Su X. Rewired signaling network in T cells expressing the chimeric antigen receptor (CAR). EMBO J. 2020 08 17; 39(16):e104730.  View on PubMed
  19. Whitman JD, Hiatt J, Mowery CT, Shy BR, Yu R, Yamamoto TN, Rathore U, Goldgof GM, Whitty C, Woo JM, Gallman AE, Miller TE, Levine AG, Nguyen DN, Bapat SP, Balcerek J, Bylsma SA, Lyons AM, Li S, Wong AW, Gillis-Buck EM, Steinhart ZB, Lee Y, Apathy R, Lipke MJ, Smith JA, Zheng T, Boothby IC, Isaza E, Chan J, Acenas DD, Lee J, Macrae TA, Kyaw TS, Wu D, Ng DL, Gu W, York VA, Eskandarian HA, Callaway PC, Warrier L, Moreno ME, Levan J, Torres L, Farrington LA, Loudermilk R, Koshal K, Zorn KC, Garcia-Beltran WF, Yang D, Astudillo MG, Bernstein BE, Gelfand JA, Ryan ET, Charles RC, Iafrate AJ, Lennerz JK, Miller S, Chiu CY, Stramer SL, Wilson MR, Manglik A, Ye CJ, Krogan NJ, Anderson MS, Cyster JG, Ernst JD, Wu AHB, Lynch KL, Bern C, Hsu PD, Marson A. Test performance evaluation of SARS-CoV-2 serological assays. medRxiv. 2020 May 17.  View on PubMed
  20. Cortez JT, Montauti E, Shifrut E, Gatchalian J, Zhang Y, Shaked O, Xu Y, Roth TL, Simeonov DR, Zhang Y, Chen S, Li Z, Woo JM, Ho J, Vogel IA, Prator GY, Zhang B, Lee Y, Sun Z, Ifergan I, Van Gool F, Hargreaves DC, Bluestone JA, Marson A, Fang D. CRISPR screen in regulatory T cells reveals modulators of Foxp3. Nature. 2020 06; 582(7812):416-420.  View on PubMed

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