Research Summary

The over-arching goal of my research career has been to develop and expand curative therapies for hemoglobin disorders in particular and non-malignant hematopoietic disorders more broadly. I have focused on genome editing approaches most recently, which rely upon modification of autologous hematopoietic cells to elicit a curative effect. In collaboration with colleagues at UC Berkeley, BCHO and UCLA, we are pursuing genomic editing of the sickle mutation in hematopoietic stem cells using the CRSPR/Cas9 ribonucleoprotein system. I have a long track record of experience in cellular therapies for pediatric acquired and hereditary conditions and in bone marrow transplantation.

Research Funding

  • April 25, 2020 - March 31, 2022 - Addressing safety issues by quantify large deletions and chromosomal rearrangements in HBB gene editing , Co-Investigator . Sponsor: NIH, Sponsor Award ID: OT2HL154977
  • September 1, 2015 - July 31, 2021 - Hematopoietic Stem Cell Transplantation for Young Adults with Sickle Cell Disease - Clinical Coordinating Center , Co-Principal Investigator . Sponsor: NIH, Sponsor Award ID: U01HL128566
  • September 20, 2019 - June 30, 2021 - Clinical Microfluidic Assessment of Red Blood Cell Adhesion, Deformability, Cellular Hemoglobin Distribution, Cellular Density, and Blood Rheology for Curative Therapies in Sickle Cell Disease , Co-Investigator . Sponsor: NIH, Sponsor Award ID: OT2HL152643
  • June 12, 2019 - May 31, 2021 - Curing Sickle Cell Disease with CRISPR-Cas9 genome editing , Principal Investigator . Sponsor: NIH, Sponsor Award ID: OT2HL151319


2019 - Diversity, Equity, and Inclusion Champion Training, University of California
06/1991 - Pediatric Hem/Onc/BMT fellowship, Fred Hutch/Univ of Washington
06/1988 - Pediatric Residency, University of Washington
M.D., 06/1985 - Medicine, University of California, San Diego
A.B., 06/1981 - Genetics, University of California, Berkeley

Selected Publications

  1. Olson TS, Walters MC. Allogeneic haematopoietic stem-cell transplantation versus gene therapy for haemoglobinopathies. Lancet Haematol. 2023 Oct; 10(10):e798-e800.  View on PubMed
  2. Hall AG, Winestone LE, Sullivan EM, Wu Q, Lamble AJ, Walters MC, Aguayo-Hiraldo P, Baez Conde L, Coker TR, Dornsife D, Keating AK, Merino DM, Ramsey B, Park JR, Agrawal AK. Access to Chimeric Antigen Receptor T Cell Clinical Trials in Underrepresented Populations: A Multicenter Cohort Study of Pediatric and Young Adult Acute Lymphobastic Leukemia Patients. Transplant Cell Ther. 2023 06; 29(6):356.e1-356.e7.  View on PubMed
  3. Lin C, Schwarzbach A, Sanz J, Montesinos P, Stiff P, Parikh S, Brunstein C, Cutler C, Lindemans CA, Hanna R, Koh LP, Jagasia MH, Valcarcel D, Maziarz RT, Keating AK, Hwang WYK, Rezvani AR, Karras NA, Fernandes JF, Rocha V, Badell I, Ram R, Schiller GJ, Volodin L, Walters MC, Hamerschlak N, Cilloni D, Frankfurt O, McGuirk JP, Kurtzberg J, Sanz G, Simantov R, Horwitz ME. Multicenter Long-Term Follow-Up of Allogeneic Hematopoietic Cell Transplantation with Omidubicel: A Pooled Analysis of Five Prospective Clinical Trials. Transplant Cell Ther. 2023 05; 29(5):338.e1-338.e6.  View on PubMed
  4. Eapen M, Brazauskas R, Williams DA, Walters MC, St Martin A, Jacobs BL, Antin JH, Bona K, Chaudhury S, Coleman-Cowger VH, DiFronzo NL, Esrick EB, Field JJ, Fitzhugh CD, Kanter J, Kapoor N, Kohn DB, Krishnamurti L, London WB, Pulsipher MA, Talib S, Thompson AA, Waller EK, Wun T, Horowitz MM. Secondary Neoplasms After Hematopoietic Cell Transplant for Sickle Cell Disease. J Clin Oncol. 2023 04 20; 41(12):2227-2237.  View on PubMed
  5. Lawal RA, Walters MC, Fitzhugh CD. Allogeneic Transplant and Gene Therapy: Evolving Toward a Cure. Hematol Oncol Clin North Am. 2022 Dec; 36(6):1313-1335.  View on PubMed
  6. Krishnamurti L, Neuberg D, Sullivan KM, Smith S, Eapen M, Walters MC. Enrollment Lessons from a Biological Assignment Study of Marrow Transplantation versus Standard Care for Adolescents and Young Adults with Sickle Cell Disease: Considerations for Future Gene and Cellular Therapy Trials. Transplant Cell Ther. 2023 04; 29(4):217-221.  View on PubMed
  7. Kanter J, Thompson AA, Pierciey FJ, Hsieh M, Uchida N, Leboulch P, Schmidt M, Bonner M, Guo R, Miller A, Ribeil JA, Davidson D, Asmal M, Walters MC, Tisdale JF. Lovo-cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the HGB-206 study. Am J Hematol. 2023 01; 98(1):11-22.  View on PubMed
  8. Magis W, DeWitt MA, Wyman SK, Vu JT, Heo SJ, Shao SJ, Hennig F, Romero ZG, Campo-Fernandez B, Said S, McNeill MS, Rettig GR, Sun Y, Wang Y, Behlke MA, Kohn DB, Boffelli D, Walters MC, Corn JE, Martin DIK. High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation. iScience. 2022 Jun 17; 25(6):104374.  View on PubMed
  9. Kanter J, Walters MC, Krishnamurti L, Mapara MY, Kwiatkowski JL, Rifkin-Zenenberg S, Aygun B, Kasow KA, Pierciey FJ, Bonner M, Miller A, Zhang X, Lynch J, Kim D, Ribeil JA, Asmal M, Goyal S, Thompson AA, Tisdale JF. Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease. N Engl J Med. 2022 02 17; 386(7):617-628.  View on PubMed
  10. Locatelli F, Thompson AA, Kwiatkowski JL, Porter JB, Thrasher AJ, Hongeng S, Sauer MG, Thuret I, Lal A, Algeri M, Schneiderman J, Olson TS, Carpenter B, Amrolia PJ, Anurathapan U, Schambach A, Chabannon C, Schmidt M, Labik I, Elliot H, Guo R, Asmal M, Colvin RA, Walters MC. Betibeglogene Autotemcel Gene Therapy for Non-β0/β0 Genotype β-Thalassemia. N Engl J Med. 2022 02 03; 386(5):415-427.  View on PubMed
  11. Kanter J, Liem RI, Bernaudin F, Bolaños-Meade J, Fitzhugh CD, Hankins JS, Murad MH, Panepinto JA, Rondelli D, Shenoy S, Wagner J, Walters MC, Woolford T, Meerpohl JJ, Tisdale J. American Society of Hematology 2021 guidelines for sickle cell disease: stem cell transplantation. Blood Adv. 2021 09 28; 5(18):3668-3689.  View on PubMed
  12. Heslop HE, Stadtmauer EA, Levine JE, Ballen KK, Chen YB, DeZern AE, Eapen M, Hamadani M, Hamilton BK, Hari P, Jones RJ, Logan BR, Kean LS, Leifer ES, Locke FL, Maziarz RT, Nemecek ER, Pasquini M, Phelan R, Riches ML, Shaw BE, Walters MC, Foley A, Devine SM, Horowitz MM. Blood and Marrow Transplant Clinical Trials Network State of the Science Symposium 2021: Looking Forward as the Network Celebrates its 20th Year. Transplant Cell Ther. 2021 11; 27(11):885-907.  View on PubMed
  13. Friedman D, Dozor AJ, Milner J, D'Souza M, Talano JA, Moore TB, Shenoy S, Shi Q, Walters MC, Vichinsky E, Parsons SK, Braniecki S, Moorthy CR, Ayello J, Flower A, Morris E, Mahanti H, Fabricatore S, Klejmont L, van de Ven C, Baxter-Lowe LA, Cairo MS. Stable to improved cardiac and pulmonary function in children with high-risk sickle cell disease following haploidentical stem cell transplantation. Bone Marrow Transplant. 2021 09; 56(9):2221-2230.  View on PubMed
  14. Walters MC. Induction of Fetal Hemoglobin by Gene Therapy. N Engl J Med. 2021 01 21; 384(3):284-285.  View on PubMed
  15. Brazauskas R, Scigliuolo GM, Wang HL, Cappelli B, Ruggeri A, Fitzhugh CD, Hankins JS, Kanter J, Meerpohl JJ, Panepinto JA, Rondelli D, Shenoy S, Walters MC, Wagner JE, Tisdale JF, Gluckman E, Eapen M. Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease. Blood. 2020 07 30; 136(5):623-626.  View on PubMed
  16. Walters MC. Sickle marrow: double, double toil and trouble. Blood. 2020 06 04; 135(23):2017-2018.  View on PubMed
  17. Tisdale JF, Pierciey FJ, Bonner M, Thompson AA, Krishnamurti L, Mapara MY, Kwiatkowski JL, Shestopalov I, Ribeil JA, Huang W, Asmal M, Kanter J, Walters MC. Safety and feasibility of hematopoietic progenitor stem cell collection by mobilization with plerixafor followed by apheresis vs bone marrow harvest in patients with sickle cell disease in the multi-center HGB-206 trial. Am J Hematol. 2020 09; 95(9):E239-E242.  View on PubMed
  18. Hsieh MM, Bonner M, Pierciey FJ, Uchida N, Rottman J, Demopoulos L, Schmidt M, Kanter J, Walters MC, Thompson AA, Asmal M, Tisdale JF. Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease. Blood Adv. 2020 05 12; 4(9):2058-2063.  View on PubMed
  19. Farrell AT, Panepinto J, Desai AA, Kassim AA, Lebensburger J, Walters MC, Bauer DE, Blaylark RM, DiMichele DM, Gladwin MT, Green NS, Hassell K, Kato GJ, Klings ES, Kohn DB, Krishnamurti L, Little J, Makani J, Malik P, McGann PT, Minniti C, Morris CR, Odame I, Oneal PA, Setse R, Sharma P, Shenoy S. End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings. Blood Adv. 2019 12 10; 3(23):4002-4020.  View on PubMed
  20. Li C, Mathews V, Kim S, George B, Hebert K, Jiang H, Li C, Zhu Y, Keesler DA, Boelens JJ, Dvorak CC, Agarwal R, Auletta JJ, Goyal RK, Hanna R, Kasow K, Shenoy S, Smith AR, Walters MC, Eapen M. Related and unrelated donor transplantation for β-thalassemia major: results of an international survey. Blood Adv. 2019 09 10; 3(17):2562-2570.  View on PubMed

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