University of California San Francisco

Publications

  1. Dual α-globin-truncated erythropoietin receptor knockin restores hemoglobin production in α-thalassemia-derived erythroid cells.
    Chu SN, Soupene E, Sharma D, Sinha R, McCreary T, Hernandez B, Shen H, Wienert B, Bowman C, Yin H, Lesch BJ, Jia K, Romero KA, Kostamo Z, Zhang Y, Tran T, Cordero M, Homma S, Hampton JP, Gardner JM, Conklin BR, MacKenzie TC, Sheehan VA, Porteus MH, Cromer MK
    Cell Rep. 2025 01 28; 44(1):115141.; 
    PubMed
  2. Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease.
    Lattanzi A, Camarena J, Lahiri P, Segal H, Srifa W, Vakulskas CA, Frock RL, Kenrick J, Lee C, Talbott N, Skowronski J, Cromer MK, Charlesworth CT, Bak RO, Mantri S, Bao G, DiGiusto D, Tisdale J, Wright JF, Bhatia N, Roncarolo MG, Dever DP, Porteus MH
    Sci Transl Med. 2021 06 16; 13(598).; 
    PubMed
  3. Neomorphic effects of recurrent somatic mutations in Yin Yang 1 in insulin-producing adenomas.
    Cromer MK, Choi M, Nelson-Williams C, Fonseca AL, Kunstman JW, Korah RM, Overton JD, Mane S, Kenney B, Malchoff CD, Stalberg P, Akerström G, Westin G, Hellman P, Carling T, Björklund P, Lifton RP
    Proc Natl Acad Sci U S A. 2015 Mar 31; 112(13):4062-7.; 
    PubMed
  4. Mobilization meets antibody conditioning: A path toward safer engraftment.
    Cromer MK
    Mol Ther. 2025 Oct 01; 33(10):4673-4675.; 
    PubMed
  5. Transient inhibition of 53BP1 increases the frequency of targeted integration in human hematopoietic stem and progenitor cells.
    Baik R, Cromer MK, Glenn SE, Vakulskas CA, Chmielewski KO, Dudek AM, Feist WN, Klermund J, Shipp S, Cathomen T, Dever DP, Porteus MH
    Nat Commun. 2024 01 02; 15(1):111.; 
    PubMed
  6. Improving the safety of human pluripotent stem cell therapies using genome-edited orthogonal safeguards.
    Martin RM, Fowler JL, Cromer MK, Lesch BJ, Ponce E, Uchida N, Nishimura T, Porteus MH, Loh KM
    Nat Commun. 2020 06 01; 11(1):2713.; 
    PubMed
  7. Highly efficient in vivo hematopoietic stem cell transduction using an optimized self-complementary adeno-associated virus.
    Charlesworth CT, Homma S, Amaya AK, Dib C, Vaidyanathan S, Tan TK, Miyauchi M, Nakauchi Y, Suchy FP, Wang S, Igarashi KJ, Cromer MK, Dudek AM, Amorin A, Czechowicz A, Wilkinson AC, Nakauchi H
    PubMed
  8. CRISPR nuclease off-target activity and mitigation strategies.
    Wienert B, Cromer MK
    Front Genome Ed. 2022; 4:1050507.; 
    PubMed
  9. Global Transcriptional Response to CRISPR/Cas9-AAV6-Based Genome Editing in CD34+ Hematopoietic Stem and Progenitor Cells.
    Cromer MK, Vaidyanathan S, Ryan DE, Curry B, Lucas AB, Camarena J, Kaushik M, Hay SR, Martin RM, Steinfeld I, Bak RO, Dever DP, Hendel A, Bruhn L, Porteus MH
    Mol Ther. 2018 10 03; 26(10):2431-2442.; 
    PubMed
  10. In vivo site-specific engineering to reprogram T cells.
    Nyberg WA, Bernard PL, Ngo W, Wang CH, Ark J, Rothrock A, Borgo GM, Kimmerly GR, Jung JH, Allain V, Hamilton JR, Baldwin A, Stickels R, Wyman S, Khan SH, Lang S, Marsh D, Almudhfar N, Novick C, Mortazavi Y, Zhang S, AbdElwakil MM, Sandoval LR, Hwang S, Chu SN, Jung H, Liu C, Sharma D, McCreary T, Li Z, Satpathy AT, Carnevale J, Rutishauser RL, Cromer MK, Roybal KT, Dodgson SE, Doudna JA, Asokan A, Eyquem J
    Nature. 2026 Mar 18.; 
    PubMed
  11. Enhancement of erythropoietic output by Cas9-mediated insertion of a natural variant in haematopoietic stem and progenitor cells.
    Luna SE, Camarena J, Hampton JP, Majeti KR, Charlesworth CT, Soupene E, Selvaraj S, Jia K, Sheehan VA, Cromer MK, Porteus MH
    Nat Biomed Eng. 2024 12; 8(12):1540-1552.; 
    PubMed
  12. Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells.
    Cromer MK, Camarena J, Martin RM, Lesch BJ, Vakulskas CA, Bode NM, Kurgan G, Collingwood MA, Rettig GR, Behlke MA, Lemgart VT, Zhang Y, Goyal A, Zhao F, Ponce E, Srifa W, Bak RO, Uchida N, Majeti R, Sheehan VA, Tisdale JF, Dever DP, Porteus MH
    Nat Med. 2021 04; 27(4):677-687.; 
    PubMed
  13. Identification of somatic mutations in parathyroid tumors using whole-exome sequencing.
    Cromer MK, Starker LF, Choi M, Udelsman R, Nelson-Williams C, Lifton RP, Carling T
    J Clin Endocrinol Metab. 2012 Sep; 97(9):E1774-81.; 
    PubMed
  14. Lentiviral vectors for hematopoietic stem cell gene therapy restore α-globin expression in α-thalassemia red blood cells.
    Segura EER, Hart K, Campo Fernandez B, Brown D, Tam K, Gutierrez Garcia A, Seigneurbieux E, Li K, Mulumba C, Blakely E, Masiuk K, Sinha R, Sharma D, Everett J, Hogenauer M, Cromer MK, Bushman F, MacKenzie TC, Kohn DB
    Cell Rep Med. 2025 Oct 21; 6(10):102362.; 
    PubMed
  15. High-efficiency transgene integration by homology-directed repair in human primary cells using DNA-PKcs inhibition.
    Selvaraj S, Feist WN, Viel S, Vaidyanathan S, Dudek AM, Gastou M, Rockwood SJ, Ekman FK, Oseghale AR, Xu L, Pavel-Dinu M, Luna SE, Cromer MK, Sayana R, Gomez-Ospina N, Porteus MH
    Nat Biotechnol. 2024 May; 42(5):731-744.; 
    PubMed
  16. Highly Efficient and Marker-free Genome Editing of Human Pluripotent Stem Cells by CRISPR-Cas9 RNP and AAV6 Donor-Mediated Homologous Recombination.
    Martin RM, Ikeda K, Cromer MK, Uchida N, Nishimura T, Romano R, Tong AJ, Lemgart VT, Camarena J, Pavel-Dinu M, Sindhu C, Wiebking V, Vaidyanathan S, Dever DP, Bak RO, Laustsen A, Lesch BJ, Jakobsen MR, Sebastiano V, Nakauchi H, Porteus MH
    Cell Stem Cell. 2019 05 02; 24(5):821-828.e5.; 
    PubMed
  17. Engineering synthetic signaling receptors to enable erythropoietin-free erythropoiesis.
    Shah AP, Majeti KR, Ekman FK, Selvaraj S, Sharma D, Sinha R, Soupene E, Chati P, Luna SE, Charlesworth CT, McCreary T, Lesch BJ, Tran T, Chu SN, Porteus MH, Kyle Cromer M
    Nat Commun. 2025 Jan 29; 16(1):1140.; 
    PubMed
  18. Ultra-deep sequencing validates safety of CRISPR/Cas9 genome editing in human hematopoietic stem and progenitor cells.
    Cromer MK, Barsan VV, Jaeger E, Wang M, Hampton JP, Chen F, Kennedy D, Xiao J, Khrebtukova I, Granat A, Truong T, Porteus MH
    Nat Commun. 2022 Aug 11; 13(1):4724.; 
    PubMed
  19. Priming Human Repopulating Hematopoietic Stem and Progenitor Cells for Cas9/sgRNA Gene Targeting.
    Charlesworth CT, Camarena J, Cromer MK, Vaidyanathan S, Bak RO, Carte JM, Potter J, Dever DP, Porteus MH
    Mol Ther Nucleic Acids. 2018 Sep 07; 12:89-104.; 
    PubMed
  20. Novel humanized loss-of-function NF1 mouse model of juvenile myelomonocytic leukemia.
    Sinha R, Patil RV, Romano R, Sharma D, Lee E, Perriman R, Takeda S, Lesch BJ, Yao Z, Liu YL, Cromer MK, Porteus MH, Bertaina A
    Blood Adv. 2026 Feb 10; 10(3):837-849.; 
    PubMed
  21. Lineage-tracing hematopoietic stem cell origins in vivo to efficiently make human HLF+ HOXA+ hematopoietic progenitors from pluripotent stem cells.
    Fowler JL, Zheng SL, Nguyen A, Chen A, Xiong X, Chai T, Chen JY, Karigane D, Banuelos AM, Niizuma K, Kayamori K, Nishimura T, Cromer MK, Gonzalez-Perez D, Mason C, Liu DD, Yilmaz L, Miquerol L, Porteus MH, Luca VC, Majeti R, Nakauchi H, Red-Horse K, Weissman IL, Ang LT, Loh KM
    PubMed
  22. Improved Genome Editing through Inhibition of FANCM and Members of the BTR Dissolvase Complex.
    de Alencastro G, Puzzo F, Pavel-Dinu M, Zhang F, Pillay S, Majzoub K, Tiffany M, Jang H, Sheikali A, Cromer MK, Meetei R, Carette JE, Porteus MH, Pekrun K, Kay MA
    Mol Ther. 2021 03 03; 29(3):1016-1027.; 
    PubMed
  23. Protocol for efficient CRISPR/AAV-mediated genome editing and erythroid differentiation of human hematopoietic stem and progenitor cells.
    Sharma D, Sinha R, Lesch BJ, Cromer MK
    STAR Protoc. 2025 Sep 19; 6(3):104018.; 
    PubMed
  24. Comparative analysis of CRISPR off-target discovery tools following ex vivo editing of CD34+ hematopoietic stem and progenitor cells.
    Cromer MK, Majeti KR, Rettig GR, Murugan K, Kurgan GL, Bode NM, Hampton JP, Vakulskas CA, Behlke MA, Porteus MH
    PubMed
  25. Identification of preexisting adaptive immunity to Cas9 proteins in humans.
    Charlesworth CT, Deshpande PS, Dever DP, Camarena J, Lemgart VT, Cromer MK, Vakulskas CA, Collingwood MA, Zhang L, Bode NM, Behlke MA, Dejene B, Cieniewicz B, Romano R, Lesch BJ, Gomez-Ospina N, Mantri S, Pavel-Dinu M, Weinberg KI, Porteus MH
    Nat Med. 2019 02; 25(2):249-254.; 
    PubMed