Mark Walters, MD
Professor
Pediatrics
School of Medicine

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.

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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.

Education & Training

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

Websites

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• UCSF alpha stem cell clinic (stemcellclinic.ucsf.edu)

Grants and Projects

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• Addressing safety issues by quantify large deletions and chromosomal rearrangements in HBB gene editing, NIH, 2020-2022
• Hematopoietic Stem Cell Transplantation for Young Adults with Sickle Cell Disease - Clinical Coordinating Center, NIH, 2015-2021
• Clinical Microfluidic Assessment of Red Blood Cell Adhesion, Deformability, Cellular Hemoglobin Distribution, Cellular Density, and Blood Rheology for Curative Therapies in Sickle Cell Disease, NIH, 2019-2021
• Curing Sickle Cell Disease with CRISPR-Cas9 genome editing, NIH, 2019-2021
• Hematopoietic Cell Therapy for Young Adults with Severe Sickle Cell Disease, NIH, 2011-2016
• Northern California Consortium for Sickle Cell Disease, NIH, 2006-2011
• Induction of Stable Chimerism for Sickle Cell Anemia, NIH, 2001-2007
• Mechanisms of Globin Gene Silencing, NIH, 1998-2002
• Globin Gene Regulation by Gata-1 and Chromatin, NIH, 1992-2001
• Mechanisms of Erythroid Gene Expression, NIH, 1994-1999

Publications (116)

Top publication keywords:
Bone Marrow TransplantationTransplantation ConditioningTransplantation, HomologousHematopoietic Stem Cell TransplantationUnrelated Donorsbeta-ThalassemiaGraft vs Host DiseaseAnemia, Sickle CellHistocompatibility TestingHemoglobinopathiesThalassemiaGenetic Therapybeta-GlobinsBone MarrowCord Blood Stem Cell Transplantation

• Hematopoietic cell transplant compared with standard care in adolescents and young adults with sickle cell disease.
  Blood advances 2025 Walters MC, Eapen M, Liu Y, El Rassi F, Waller EK, Levine JE, Strouse JJ, Antin JH, Parikh SH, Bakshi N, Dampier C, Jaroscak JJ, Bergmann S, Wong T, Kota V, Pace B, Lekakis LJ, Lulla P, Nickel RS, …
• Haploidentical Bone Marrow Transplantation for Sickle Cell Disease.
  NEJM evidence 2025 Kassim AA, Walters MC, Eapen M, Smith M, Logan BR, Solh M, McKinney C, Nieder M, Ross M, Kent M, Abusin GA, Mallhi K, Silva JG, Shaughnessy P, Kanter J, Haines H, Farah R, Khaled YA, Ritzau N, …
• Incremental eligibility criteria for the BMT CTN 1507 haploidentical trial for children with sickle cell disease.
  Blood advances 2024 John TD, Walters MC, Rangarajan HG, Rahim MQ, McKinney C, Bollard CM, Abusin G, Eapen M, Kassim AA, DeBaun MR
• Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial.
  Lancet (London, England) 2024 Kwiatkowski JL, Walters MC, Hongeng S, Yannaki E, Kulozik AE, Kunz JB, Sauer MG, Thrasher AJ, Thuret I, Lal A, Tao G, Ali S, Thakar HL, Elliot H, Lodaya A, Lee J, Colvin RA, Locatelli F, Thompson AA
• Zinc finger nuclease-mediated gene editing in hematopoietic stem cells results in reactivation of fetal hemoglobin in sickle cell disease.
  Scientific reports 2024 Lessard S, Rimmelé P, Ling H, Moran K, Vieira B, Lin YD, Rajani GM, Hong V, Reik A, Boismenu R, Hsu B, Chen M, Cockroft BM, Uchida N, Tisdale J, Alavi A, Krishnamurti L, Abedi M, Galeon I, Reiner D, …

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• A Cure for Sickle Cell Disease.
  Transplantation and cellular therapy 2024 Walters M
• Neurocognitive outcome in children with sickle cell disease after myeloimmunoablative conditioning and haploidentical hematopoietic stem cell transplantation: a non-randomized clinical trial.
  Frontiers in neurology 2024 Braniecki S, Vichinsky E, Walters MC, Shenoy S, Shi Q, Moore TB, Talano JA, Parsons SK, Flower A, Panarella A, Fabricatore S, Morris E, Mahanti H, Milner J, McKinstry RC, Duncan CN, van de Ven C, …
• Exagamglogene Autotemcel for Severe Sickle Cell Disease.
  The New England journal of medicine 2024 Frangoul H, Locatelli F, Sharma A, Bhatia M, Mapara M, Molinari L, Wall D, Liem RI, Telfer P, Shah AJ, Cavazzana M, Corbacioglu S, Rondelli D, Meisel R, Dedeken L, Lobitz S, de Montalembert M, …
• Long-term outcomes after unrelated donor transplantation for severe sickle cell disease on the BMT CTN 0601 trial.
  American journal of hematology 2024 Eapen M, Kou J, Andreansky M, Bhatia M, Brochstein J, Chaudhury S, Haight AE, Haines H, Jacobsohn D, Jaroscak J, Kasow KA, Krishnamurti L, Levine JE, Leung K, Margolis D, Yu LC, Horowitz MM, Kamani N,…
• Defining curative endpoints for transfusion-dependent β-thalassemia in the era of gene therapy and gene editing.
  American journal of hematology 2023 Corbacioglu S, Frangoul H, Locatelli F, Hobbs W, Walters M
• Defining curative endpoints for sickle cell disease in the era of gene therapy and gene editing.
  American journal of hematology 2023 Locatelli F, Corbacioglu S, Hobbs W, Frangoul H, Walters MC
• Allogeneic haematopoietic stem-cell transplantation versus gene therapy for haemoglobinopathies.
  The Lancet. Haematology 2023 Olson TS, Walters MC
• 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.
  Transplantation and cellular therapy 2023 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
• Multicenter Long-Term Follow-Up of Allogeneic Hematopoietic Cell Transplantation with Omidubicel: A Pooled Analysis of Five Prospective Clinical Trials.
  Transplantation and cellular therapy 2023 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, …
• Secondary Neoplasms After Hematopoietic Cell Transplant for Sickle Cell Disease.
  Journal of clinical oncology : official journal of the American Society of Clinical Oncology 2023 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, …
• Allogeneic Transplant and Gene Therapy: Evolving Toward a Cure.
  Hematology/oncology clinics of North America 2022 Lawal RA, Walters MC, Fitzhugh CD
• 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.
  Transplantation and cellular therapy 2022 Krishnamurti L, Neuberg D, Sullivan KM, Smith S, Eapen M, Walters MC
• Lovo-cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the HGB-206 study.
  American journal of hematology 2022 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
• High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation.
  iScience 2022 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 …
• Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease.
  The New England journal of medicine 2021 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 …
• Betibeglogene Autotemcel Gene Therapy for Non-β0/β0 Genotype β-Thalassemia.
  The New England journal of medicine 2021 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, …
• American Society of Hematology 2021 guidelines for sickle cell disease: stem cell transplantation.
  Blood advances 2021 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
• Blood and Marrow Transplant Clinical Trials Network State of the Science Symposium 2021: Looking Forward as the Network Celebrates its 20th Year.
  Transplantation and cellular therapy 2021 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, …
• Stable to improved cardiac and pulmonary function in children with high-risk sickle cell disease following haploidentical stem cell transplantation.
  Bone marrow transplantation 2021 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, …
• Induction of Fetal Hemoglobin by Gene Therapy.
  The New England journal of medicine 2021 Walters MC
• Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease.
  Blood 2020 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
• Sickle marrow: double, double toil and trouble.
  Blood 2020 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.
  American journal of hematology 2020 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
• Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease.
  Blood advances 2020 Hsieh MM, Bonner M, Pierciey FJ, Uchida N, Rottman J, Demopoulos L, Schmidt M, Kanter J, Walters MC, Thompson AA, Asmal M, Tisdale JF
• End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings.
  Blood advances 2019 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, …
• Related and unrelated donor transplantation for β-thalassemia major: results of an international survey.
  Blood advances 2019 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
• Effect of donor type and conditioning regimen intensity on allogeneic transplantation outcomes in patients with sickle cell disease: a retrospective multicentre, cohort study.
  The Lancet. Haematology 2019 Eapen M, Brazauskas R, Walters MC, Bernaudin F, Bo-Subait K, Fitzhugh CD, Hankins JS, Kanter J, Meerpohl JJ, Bolaños-Meade J, Panepinto JA, Rondelli D, Shenoy S, Williamson J, Woolford TL, Gluckman E,…
• Bone marrow transplantation for adolescents and young adults with sickle cell disease: Results of a prospective multicenter pilot study.
  American journal of hematology 2019 Krishnamurti L, Neuberg DS, Sullivan KM, Kamani NR, Abraham A, Campigotto F, Zhang W, Dahdoul T, De Castro L, Parikh S, Bakshi N, Haight A, Hassell KL, Loving R, Rosenthal J, Smith SL, Smith W, …
• CRISPR-Cas9 interrogation of a putative fetal globin repressor in human erythroid cells.
  PloS one 2019 Chung JE, Magis W, Vu J, Heo SJ, Wartiovaara K, Walters MC, Kurita R, Nakamura Y, Boffelli D, Martin DIK, Corn JE, DeWitt MA
• Promise of gene therapy to treat sickle cell disease.
  Expert opinion on biological therapy 2018 Romero Z, DeWitt M, Walters MC
• The safety and efficacy of clofarabine in combination with high-dose cytarabine and total body irradiation myeloablative conditioning and allogeneic stem cell transplantation in children, adolescents, and young adults (CAYA) with poor-risk acute leukemia.
  Bone marrow transplantation 2018 Hochberg J, Zahler S, Geyer MB, Chen N, Krajewski J, Harrison L, Militano O, Ozkaynak MF, Cheerva AC, Talano J, Moore TB, Gillio AP, Walters MC, Baxter-Lowe LA, Hamby C, Cairo MS
• CIRM Alpha Stem Cell Clinics: Collaboratively Addressing Regenerative Medicine Challenges.
  Cell stem cell 2018 Jamieson CHM, Millan MT, Creasey AA, Lomax G, Donohoe ME, Walters MC, Abedi M, Bota DA, Zaia JA, Adams JS
• Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia.
  The New England journal of medicine 2018 Thompson AA, Walters MC, Kwiatkowski J, Rasko JEJ, Ribeil JA, Hongeng S, Magrin E, Schiller GJ, Payen E, Semeraro M, Moshous D, Lefrere F, Puy H, Bourget P, Magnani A, Caccavelli L, Diana JS, Suarez F…
• Late Effects Screening Guidelines after Hematopoietic Cell Transplantation (HCT) for Hemoglobinopathy: Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric HCT.
  Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation 2018 Shenoy S, Gaziev J, Angelucci E, King A, Bhatia M, Smith A, Bresters D, Haight AE, Duncan CN, de la Fuente J, Dietz AC, Baker KS, Pulsipher MA, Walters MC
• Unrelated Donor Transplantation in Children with Thalassemia using Reduced-Intensity Conditioning: The URTH Trial.
  Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation 2018 Shenoy S, Walters MC, Ngwube A, Soni S, Jacobsohn D, Chaudhury S, Grimley M, Chan K, Haight A, Kasow KA, Parikh S, Andreansky M, Connelly J, Delgado D, Godder K, Hale G, Nieder M, Pulsipher MA, …
• The case for HLA-identical sibling hematopoietic stem cell transplantation in children with symptomatic sickle cell anemia.
  Blood advances 2017 Fitzhugh CD, Walters MC
• Relationship between Mixed Donor-Recipient Chimerism and Disease Recurrence after Hematopoietic Cell Transplantation for Sickle Cell Disease.
  Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation 2017 Abraham A, Hsieh M, Eapen M, Fitzhugh C, Carreras J, Keesler D, Guilcher G, Kamani N, Walters MC, Boelens JJ, Tisdale J, Shenoy S, National Institutes of Health, Center for International Blood and …
• Clinical risks and healthcare utilization of hematopoietic cell transplantation for sickle cell disease in the USA using merged databases.
  Haematologica 2017 Arnold SD, Brazauskas R, He N, Li Y, Aplenc R, Jin Z, Hall M, Atsuta Y, Dalal J, Hahn T, Khera N, Bonfim C, Majhail NS, Diaz MA, Freytes CO, Wood WA, Savani BN, Kamble RT, Parsons S, Ahmed I, Sullivan…
• Dose-adapted post-transplant cyclophosphamide for HLA-haploidentical transplantation in Fanconi anemia.
  Bone marrow transplantation 2017 Thakar MS, Bonfim C, Walters MC, Storb R, Pasquini R, Burroughs L, Sandmaier BM, Woolfrey A, Kiem HP
• Current Results and Future Research Priorities in Late Effects after Hematopoietic Stem Cell Transplantation for Children with Sickle Cell Disease and Thalassemia: A Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric Hematopoietic Stem Cell Transplantation.
  Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation 2017 Shenoy S, Angelucci E, Arnold SD, Baker KS, Bhatia M, Bresters D, Dietz AC, De La Fuente J, Duncan C, Gaziev J, King AA, Pulsipher MA, Smith AR, Walters MC
• Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation.
  Blood 2016 Gluckman E, Cappelli B, Bernaudin F, Labopin M, Volt F, Carreras J, Pinto Simões B, Ferster A, Dupont S, de la Fuente J, Dalle JH, Zecca M, Walters MC, Krishnamurti L, Bhatia M, Leung K, Yanik G, …
• Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells.
  Science translational medicine 2016 DeWitt MA, Magis W, Bray NL, Wang T, Berman JR, Urbinati F, Heo SJ, Mitros T, Muñoz DP, Boffelli D, Kohn DB, Walters MC, Carroll D, Martin DI, Corn JE
• The Second Pediatric Blood and Marrow Transplant Consortium International Consensus Conference on Late Effects after Pediatric Hematopoietic Cell Transplantation: Defining the Unique Late Effects of Children Undergoing Hematopoietic Cell Transplantation for Immune Deficiencies, Inherited Marrow Failure Disorders, and Hemoglobinopathies.
  Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation 2016 Dietz AC, Duncan CN, Alter BP, Bresters D, Cowan MJ, Notarangelo L, Rosenberg PS, Shenoy S, Skinner R, Walters MC, Wagner J, Baker KS, Pulsipher MA
• A trial of unrelated donor marrow transplantation for children with severe sickle cell disease.
  Blood 2016 Shenoy S, Eapen M, Panepinto JA, Logan BR, Wu J, Abraham A, Brochstein J, Chaudhury S, Godder K, Haight AE, Kasow KA, Leung K, Andreansky M, Bhatia M, Dalal J, Haines H, Jaroscak J, Lazarus HM, Levine…
• Ankyrin-B directs membrane tethering of periaxin and is required for maintenance of lens fiber cell hexagonal shape and mechanics.
  American journal of physiology. Cell physiology 2015 Maddala R, Walters M, Brophy PJ, Bennett V, Rao PV
• Indications and Results of HLA-Identical Sibling Hematopoietic Cell Transplantation for Sickle Cell Disease.
  Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation 2015 Walters MC, De Castro LM, Sullivan KM, Krishnamurti L, Kamani N, Bredeson C, Neuberg D, Hassell KL, Farnia S, Campbell A, Petersdorf E
• A booster shot to cure hemoglobinopathies.
  Blood 2015 Walters MC, MacKenzie TC
• Update of hematopoietic cell transplantation for sickle cell disease.
  Current opinion in hematology 2015 Walters MC
• In utero hematopoietic cell transplantation for hemoglobinopathies.
  Frontiers in pharmacology 2015 Derderian SC, Jeanty C, Walters MC, Vichinsky E, MacKenzie TC
• One-unit versus two-unit cord-blood transplantation for hematologic cancers.
  The New England journal of medicine 2014 Wagner JE, Eapen M, Carter S, Wang Y, Schultz KR, Wall DA, Bunin N, Delaney C, Haut P, Margolis D, Peres E, Verneris MR, Walters M, Horowitz MM, Kurtzberg J, Blood and Marrow Transplant Clinical …
• Phenotyping: targeting genotype's rich cousin for diagnosis.
  Journal of paediatrics and child health 2014 Baynam G, Walters M, Claes P, Kung S, LeSouef P, Dawkins H, Bellgard M, Girdea M, Brudno M, Robinson P, Zankl A, Groza T, Gillett D, Goldblatt J
• Combined umbilical cord blood and bone marrow from HLA-identical sibling donors for hematopoietic stem cell transplantation in children with hemoglobinopathies.
  Pediatric blood & cancer 2014 Soni S, Boulad F, Cowan MJ, Scaradavou A, Dahake J, Edwards S, Walters MC
• Evaluation of the Spectra Optia apheresis system for mononuclear cell (MNC) collection in G-CSF mobilized and nonmobilized healthy donors: results of a multicenter study.
  Journal of clinical apheresis 2014 Karafin MS, Graminske S, Erickson P, Walters MC, Scott EP, Carter S, Padmanabhan A
• Thiol/redox metabolomic profiling implicates GSH dysregulation in early experimental graft versus host disease (GVHD).
  PloS one 2014 Suh JH, Kanathezhath B, Shenvi S, Guo H, Zhou A, Tiwana A, Kuypers F, Ames BN, Walters MC
• β-globin gene transfer to human bone marrow for sickle cell disease.
  The Journal of clinical investigation 2013 Romero Z, Urbinati F, Geiger S, Cooper AR, Wherley J, Kaufman ML, Hollis RP, de Assin RR, Senadheera S, Sahagian A, Jin X, Gellis A, Wang X, Gjertson D, Deoliveira S, Kempert P, Shupien S, Abdel-Azim …
• Outcome of patients with hemoglobinopathies given either cord blood or bone marrow transplantation from an HLA-identical sibling.
  Blood 2013 Locatelli F, Kabbara N, Ruggeri A, Ghavamzadeh A, Roberts I, Li CK, Bernaudin F, Vermylen C, Dalle JH, Stein J, Wynn R, Cordonnier C, Pinto F, Angelucci E, Socié G, Gluckman E, Walters MC, Rocha V, …
• Unrelated donor cord blood transplantation for children with severe sickle cell disease: results of one cohort from the phase II study from the Blood and Marrow Transplant Clinical Trials Network (BMT CTN).
  Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation 2012 Kamani NR, Walters MC, Carter S, Aquino V, Brochstein JA, Chaudhury S, Eapen M, Freed BM, Grimley M, Levine JE, Logan B, Moore T, Panepinto J, Parikh S, Pulsipher MA, Sande J, Schultz KR, Spellman S, …
• Hematopoietic cell transplantation for nonmalignant disorders.
  Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation 2012 Tolar J, Mehta PA, Walters MC
• Improved engraftment with minimal graft-versus-host disease after major histocompatibility complex-mismatched cord blood transplantation with photochemically treated donor lymphocytes.
  Experimental biology and medicine (Maywood, N.J.) 2011 Kanathezhath B, Mizokami M, Stanislaus S, Hounshell C, Neumayr L, Guo H, Hearst JE, Walters MC, Kuypers FA
• Umbilical cord blood transplantation for children with thalassemia and sickle cell disease.
  Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation 2011 Ruggeri A, Eapen M, Scaravadou A, Cairo MS, Bhatia M, Kurtzberg J, Wingard JR, Fasth A, Lo Nigro L, Ayas M, Purtill D, Boudjedir K, Chaves W, Walters MC, Wagner J, Gluckman E, Rocha V, Eurocord …
• Umbilical cord blood transplantation for thalassemia major.
  Hematology/oncology clinics of North America 2010 Kanathezhath B, Walters MC
• HLA-matched sibling bone marrow transplantation for β-thalassemia major.
  Blood 2010 Sabloff M, Chandy M, Wang Z, Logan BR, Ghavamzadeh A, Li CK, Irfan SM, Bredeson CN, Cowan MJ, Gale RP, Hale GA, Horan J, Hongeng S, Eapen M, Walters MC
• Gene therapy and bone marrow transplantation for thalassemia: changing of the guard?
  Molecular therapy : the journal of the American Society of Gene Therapy 2010 Walters MC
• Stem-cell transplantation for sickle cell disease.
  The New England journal of medicine 2010 Walters MC, Sullivan KM
• Transplant outcomes in bone marrow failure syndromes and hemoglobinopathies.
  Seminars in hematology 2010 MacMillan ML, Walters MC, Gluckman E
• Pulmonary, gonadal, and central nervous system status after bone marrow transplantation for sickle cell disease.
  Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation 2009 Walters MC, Hardy K, Edwards S, Adamkiewicz T, Barkovich J, Bernaudin F, Buchanan GR, Bunin N, Dickerhoff R, Giller R, Haut PR, Horan J, Hsu LL, Kamani N, Levine JE, Margolis D, Ohene-Frempong K, …
• Translational stability for sickle gene therapy.
  Gene therapy 2009 Walters MC
• Human term placenta as a source of hematopoietic cells.
  Experimental biology and medicine (Maywood, N.J.) 2009 Serikov V, Hounshell C, Larkin S, Green W, Ikeda H, Walters MC, Kuypers FA
• Risk factors affecting outcome of second HLA-matched sibling donor transplantations for graft failure in severe acquired aplastic anemia.
  Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation 2009 Horan JT, Carreras J, Tarima S, Camitta BM, Gale RP, Hale GA, Hinterberger W, Marsh J, Passweg JR, Walters MC, Eapen M
• Newborn screening for hemoglobinopathies in California.
  Pediatric blood & cancer 2009 Michlitsch J, Azimi M, Hoppe C, Walters MC, Lubin B, Lorey F, Vichinsky E
• Recent advances in bone marrow transplantation in hemoglobinopathies.
  Current molecular medicine 2008 Michlitsch JG, Walters MC
• Transfusional iron burden and liver toxicity after bone marrow transplantation for acute myelogenous leukemia and hemoglobinopathies.
  Pediatric blood & cancer 2008 Jastaniah W, Harmatz P, Pakbaz Z, Fischer R, Vichinsky E, Walters MC
• Hematopoietic cell transplantation for thalassemia and sickle cell disease: past, present and future.
  Bone marrow transplantation 2007 Bhatia M, Walters MC
• Mixed haematopoietic chimerism for sickle cell disease prevents intravascular haemolysis.
  British journal of haematology 2007 Wu CJ, Gladwin M, Tisdale J, Hsieh M, Law T, Biernacki M, Rogers S, Wang X, Walters M, Zahrieh D, Antin JH, Ritz J, Krishnamurti L
• A prospective study of G-CSF primed bone marrow as a stem-cell source for allogeneic bone marrow transplantation in children: a Pediatric Blood and Marrow Transplant Consortium (PBMTC) study.
  Blood 2007 Frangoul H, Nemecek ER, Billheimer D, Pulsipher MA, Khan S, Woolfrey A, Manes B, Cole C, Walters MC, Ayas M, Ravindranath Y, Levine JE, Grupp SA
• Cord blood transplantation for sickle cell anemia: bust or boom?
  Pediatric transplantation 2007 Walters MC
• Matched-related donor transplantation for sickle cell disease: report from the Center for International Blood and Transplant Research.
  British journal of haematology 2007 Panepinto JA, Walters MC, Carreras J, Marsh J, Bredeson CN, Gale RP, Hale GA, Horan J, Hows JM, Klein JP, Pasquini R, Roberts I, Sullivan K, Eapen M, Ferster A, Non-Malignant Marrow Disorders Working …
• Effect of myeloablative bone marrow transplantation on growth in children with sickle cell anaemia: results of the multicenter study of haematopoietic cell transplantation for sickle cell anaemia.
  British journal of haematology 2007 Eggleston B, Patience M, Edwards S, Adamkiewicz T, Buchanan GR, Davies SC, Dickerhoff R, Donfield S, Feig SA, Giller RH, Haight A, Horan J, Hsu LL, Kamani N, Lane P, Levine JE, Margolis D, Moore TB, …
• Clinical hemoglobinopathies: iron, lungs and new blood.
  Current opinion in hematology 2006 Morris CR, Singer ST, Walters MC
• The prevention and management of stroke in sickle cell anaemia.
  Expert opinion on biological therapy 2006 Qureshi N, Lubin B, Walters MC
• Hematopoietic stem cell transplantation for multiply transfused patients with sickle cell disease and thalassemia after low-dose total body irradiation, fludarabine, and rabbit anti-thymocyte globulin.
  Bone marrow transplantation 2005 Horan JT, Liesveld JL, Fenton P, Blumberg N, Walters MC
• Outcomes of preimplantation genetic diagnosis therapy in treatment of beta-thalassemia: A retrospective analysis.
  Annals of the New York Academy of Sciences 2005 Qureshi N, Foote D, Walters MC, Singer ST, Quirolo K, Vichinsky EP
• Sibling donor cord blood transplantation for thalassemia major: Experience of the Sibling Donor Cord Blood Program.
  Annals of the New York Academy of Sciences 2005 Walters MC, Quirolo L, Trachtenberg ET, Edwards S, Hale L, Lee J, Morton-Wiley J, Quirolo K, Robertson S, Saba J, Lubin B
• Stem cell therapy for sickle cell disease: transplantation and gene therapy.
  Hematology. American Society of Hematology. Education Program 2005 Walters MC
• Stem cell transplantation with S-59 photochemically treated T-cell add-backs to establish allochimerism in murine thalassemia.
  Annals of the New York Academy of Sciences 2005 Kuypers FA, Watson G, Sage E, Walters MC, Hamrick J, Hearst JE
• Treatment of hepatitis C virus infection in thalassemia.
  Annals of the New York Academy of Sciences 2005 Butensky E, Pakbaz Z, Foote D, Walters MC, Vichinsky EP, Harmatz P
• Sickle cell anemia and hematopoietic cell transplantation: When is a pound of cure worth more than an ounce of prevention?
  Pediatric transplantation 2004 Walters MC
• Haematopoietic cell transplantation in the treatment of sickle cell disease.
  Expert opinion on biological therapy 2003 Atkins RC, Walters MC
• Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia.
  Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation 2003 Iannone R, Casella JF, Fuchs EJ, Chen AR, Jones RJ, Woolfrey A, Amylon M, Sullivan KM, Storb RF, Walters MC
• Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease.
  Blood 2002 Locatelli F, Rocha V, Reed W, Bernaudin F, Ertem M, Grafakos S, Brichard B, Li X, Nagler A, Giorgiani G, Haut PR, Brochstein JA, Nugent DJ, Blatt J, Woodard P, Kurtzberg J, Rubin CM, Miniero R, Lutz P…
• Comprehensive banking of sibling donor cord blood for children with malignant and nonmalignant disease.
  Blood 2002 Reed W, Smith R, Dekovic F, Lee JY, Saba JD, Trachtenberg E, Epstein J, Haaz S, Walters MC, Lubin BH
• Novel therapeutic approaches in sickle cell disease.
  Hematology. American Society of Hematology. Education Program 2002 Walters MC, Nienhuis AW, Vichinsky E
• Bone marrow transplantation in sickle cell anemia.
  Current opinion in oncology 2001 Hoppe CC, Walters MC
• Sibling donor cord blood banking for children with sickle cell disease.
  Pediatric pathology & molecular medicine 2001 Reed W, Walters M, Trachtenberg E, Smith R, Lubin BH
• Stable mixed hematopoietic chimerism after bone marrow transplantation for sickle cell anemia.
  Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation 2001 Walters MC, Patience M, Leisenring W, Rogers ZR, Aquino VM, Buchanan GR, Roberts IA, Yeager AM, Hsu L, Adamkiewicz T, Kurtzberg J, Vichinsky E, Storer B, Storb R, Sullivan KM, Multicenter …
• Stem cell transplantation for sickle cell disease: can we reduce the toxicity?
  Pediatric pathology & molecular medicine 2001 Fixler J, Vichinsky E, Walters MC
• Collection of sibling donor cord blood for children with thalassemia.
  Journal of pediatric hematology/oncology 2000 Reed W, Walters M, Lubin BH
• Impact of bone marrow transplantation for symptomatic sickle cell disease: an interim report. Multicenter investigation of bone marrow transplantation for sickle cell disease.
  Blood 2000 Walters MC, Storb R, Patience M, Leisenring W, Taylor T, Sanders JE, Buchanan GE, Rogers ZR, Dinndorf P, Davies SC, Roberts IA, Dickerhoff R, Yeager AM, Hsu L, Kurtzberg J, Ohene-Frempong K, Bunin N, …
• Bone Marrow Transplantation for Non-Malignant Disease.
  Hematology. American Society of Hematology. Education Program 2000 Sullivan KM, Parkman R, Walters MC
• Bone marrow transplantation for sickle cell disease: where do we go from here?
  Journal of pediatric hematology/oncology 1999 Walters MC
• A functional enhancer suppresses silencing of a transgene and prevents its localization close to centrometric heterochromatin.
  Cell 1999 Francastel C, Walters MC, Groudine M, Martin DI
• The chicken beta-globin 5'HS4 boundary element blocks enhancer-mediated suppression of silencing.
  Molecular and cellular biology 1999 Walters MC, Fiering S, Bouhassira EE, Scalzo D, Goeke S, Magis W, Garrick D, Whitelaw E, Martin DI
• Current and future preparative regimens for bone marrow transplantation in thalassemia.
  Annals of the New York Academy of Sciences 1998 Storb R, Yu C, Deeg HJ, Georges G, Kiem HP, Mcsweeney PA, Nash RA, Sandmaier BM, Sullivan KM, Wagner JL, Walters MC
• Unrelated and HLA-nonidentical related donor marrow transplantation for thalassemia and leukemia. A combined report from the Seattle Marrow Transplant Team and the International Bone Marrow Transplant Registry.
  Annals of the New York Academy of Sciences 1998 Sullivan KM, Anasetti C, Horowitz M, Rowlings PA, Petersdorf EW, Martin PJ, Clift RA, Walters MC, Gooley T, Sierra J, Anderson JE, Bjerke J, Siadak M, Flowers ME, Nash RA, Sanders JE, Appelbaum FR, …
• Collaborative multicenter investigation of marrow transplantation for sickle cell disease: current results and future directions.
  Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation 1997 Walters MC, Patience M, Leisenring W, Rogers ZR, Dinndorf P, Davies SC, Roberts IA, Yeager A, Kurtzberg J, Bunin N, Scott JP, Wall DA, Wayne AS, Wiley J, Darbyshire PJ, Mentzer WC, Smith FO, Sullivan …
• Bone marrow transplantation for sickle cell disease.
  The New England journal of medicine 1996 Walters MC, Patience M, Leisenring W, Eckman JR, Scott JP, Mentzer WC, Davies SC, Ohene-Frempong K, Bernaudin F, Matthews DC, Storb R, Sullivan KM
• Barriers to bone marrow transplantation for sickle cell anemia.
  Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation 1996 Walters MC, Patience M, Leisenring W, Eckman JR, Buchanan GR, Rogers ZR, Olivieri NE, Vichinsky E, Davies SC, Mentzer WC, Powars D, Scott JP, Bernaudin F, Ohene-Frempong K, Darbyshire PJ, Wayne A, …
• Transcriptional enhancers act in cis to suppress position-effect variegation.
  Genes & development 1996 Walters MC, Magis W, Fiering S, Eidemiller J, Scalzo D, Groudine M, Martin DI
• Enhancers increase the probability but not the level of gene expression.
  Proceedings of the National Academy of Sciences of the United States of America 1995 Walters MC, Fiering S, Eidemiller J, Magis W, Groudine M, Martin DI
• Neurologic complications after allogeneic marrow transplantation for sickle cell anemia.
  Blood 1995 Walters MC, Sullivan KM, Bernaudin F, Souillet G, Vannier JP, Johnson FL, Lenarsky C, Powars D, Bunin N, Ohene-Frempong K
• Bone marrow transplantation for thalassemia. The USA experience.
  The American journal of pediatric hematology/oncology 1994 Walters MC, Sullivan KM, O'Reilly RJ, Boulad F, Brockstein J, Blume K, Amylon M, Johnson FL, Klemperer M, Graham-Pole J