Mark Walters, MD

Professor In Residence
Pediatrics
[email protected]

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.

Publications: 

Incremental Eligibility Criteria for the BMT CTN 1507 Haploidentical Trial for Children with Sickle Cell Disease.

Blood advances

John TD, Walters MC, Rangarajan HG, Rahim MQ, McKinney CM, 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)

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

Hematopoietic Cell Transplant compared with Standard Care in Adolescents and Young Adults with Sickle Cell Disease.

Blood advances

Walters MC, Eapen M, Liu Y, El Rassi F, Waller EK, Levine JE, Strouse JJ, Antin JH, Parikh SH, Bakshi N, Dampier CD, Jaroscak JJ, Bergmann S, Wong TE, Kota VK, Pace BS, Lekakis LJ, Lulla PD, Nickel R, Kasow KA, Popat UR, Smith WR, Yu LC, DiFronzo NL, Geller NL, Kamani N, Klings ES, Hassell K, Mendizabal AM, Sullivan K, Neuberg DS, Krishnamurti L

Zinc finger nuclease-mediated gene editing in hematopoietic stem cells results in reactivation of fetal hemoglobin in sickle cell disease.

Scientific reports

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, Wang L, Ramezi A, Rendo P, Walters MC, Levasseur D, Peters R, Harris T, Hicks A

A Cure for Sickle Cell Disease.

Transplantation and cellular therapy

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

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, Cairo MS

Exagamglogene Autotemcel for Severe Sickle Cell Disease.

The New England journal of medicine

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, Steinberg MH, Walters MC, Eckrich MJ, Imren S, Bower L, Simard C, Zhou W, Xuan F, Morrow PK, Hobbs WE, Grupp SA, CLIMB SCD-121 Study Group

Long-term outcomes after unrelated donor transplantation for severe sickle cell disease on the BMT CTN 0601 trial.

American journal of hematology

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, Walters MC, Shenoy S

Defining curative endpoints for transfusion-dependent β-thalassemia in the era of gene therapy and gene editing.

American journal of hematology

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

Locatelli F, Corbacioglu S, Hobbs W, Frangoul H, Walters MC

Access to CAR T-cell Clinical Trials in Underrepresented Populations: a Multicenter Cohort Study of Pediatric and Young Adult ALL Patients.

Transplantation and cellular therapy

Hall AG, Winestone LE, Sullivan EM, Wu Q, Lamble AJ, Walters MC, Aguayo-Hiraldo P, Conde LB, 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

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

Secondary Neoplasms After Hematopoietic Cell Transplant for Sickle Cell Disease.

Journal of clinical oncology : official journal of the American Society of Clinical Oncology

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

Allogeneic Transplant and Gene Therapy: Evolving Toward a Cure.

Hematology/oncology clinics of North America

Lawal RA, Walters MC, Fitzhugh CD

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

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

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

Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease.

The New England journal of medicine

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

Betibeglogene Autotemcel Gene Therapy for Non-β0/β0 Genotype β-Thalassemia.

The New England journal of medicine

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

American Society of Hematology 2021 guidelines for sickle cell disease: stem cell transplantation.

Blood advances

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

BMT CTN State of the Science Symposium 2021: Looking Forward as the Network Celebrates its 20th Year.

Transplantation and cellular therapy

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

Stable to improved cardiac and pulmonary function in children with high-risk sickle cell disease following haploidentical stem cell transplantation.

Bone marrow transplantation

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

Induction of Fetal Hemoglobin by Gene Therapy.

The New England journal of medicine

Walters MC

Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease.

Blood

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

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

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

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

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

Related and unrelated donor transplantation for ß-thalassemia major: results of an international survey.

Blood advances

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

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, Wagner JE, Tisdale JF

Bone marrow transplantation for adolescents and young adults with sickle cell disease: Results of a prospective multicenter pilot study.

American journal of hematology

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, Spearman M, Stevenson K, Wu CJ, Wiedl C, Waller EK, Walters MC

CRISPR-Cas9 interrogation of a putative fetal globin repressor in human erythroid cells.

PloS one

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

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

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

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 ß-Thalassemia.

The New England journal of medicine

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, Monpoux F, Brousse V, Poirot C, Brouzes C, Meritet JF, Pondarré C, Beuzard Y, Chrétien S, Lefebvre T, Teachey DT, Anurathapan U, Ho PJ, von Kalle C, Kletzel M, Vichinsky E, Soni S, Veres G, Negre O, Ross RW, Davidson D, Petrusich A, Sandler L, Asmal M, Hermine O, De Montalembert M, Hacein-Bey-Abina S, Blanche S, Leboulch P, Cavazzana M

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

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

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, Trachtenberg F, Neufeld E, Kwiatkowski JL, Thompson AA

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

Abraham A, Hsieh M, Eapen M, Fitzhugh C, Carreras J, Keesler D, Guilcher G, Kamani N, Walters MC, Boelens JJ, Tisdale J, Shenoy S

Clinical risks and healthcare utilization of hematopoietic cell transplantation for sickle cell disease in the USA using merged databases.

Haematologica

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 K, Beattie S, Dandoy C, Munker R, Marino S, Bitan M, Abdel-Azim H, Aljurf M, Olsson RF, Joshi S, Buchbinder D, Eckrich MJ, Hashmi S, Lazarus H, Marks DI, Steinberg A, Saad A, Gergis U, Krishnamurti L, Abraham A, Rangarajan HG, Walters M, Lipscomb J, Saber W, Satwani P

Dose-adapted post-transplant cyclophosphamide for HLA-haploidentical transplantation in Fanconi anemia.

Bone marrow transplantation

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 Inte

Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation

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

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, Kurtzberg J, Dhedin N, Kuentz M, Michel G, Apperley J, Lutz P, Neven B, Bertrand Y, Vannier JP, Ayas M, Cavazzana M, Matthes-Martin S, Rocha V, Elayoubi H, Kenzey C, Bader P, Locatelli F, Ruggeri A, Eapen M

Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells.

Science translational medicine

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 f

Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation

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

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 JE, Krishnamurti L, Margolis D, Megason GC, Yu LC, Pulsipher MA, Gersten I, DiFronzo N, Horowitz MM, Walters MC, Kamani N

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

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

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

Walters MC, MacKenzie TC

In utero hematopoietic cell transplantation for hemoglobinopathies.

Frontiers in pharmacology

Derderian SC, Jeanty C, Walters MC, Vichinsky E, MacKenzie TC

Phenotyping: targeting genotype's rich cousin for diagnosis.

Journal of paediatrics and child health

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

ß-globin gene transfer to human bone marrow for sickle cell disease.

The Journal of clinical investigation

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 H, Walters MC, Meiselman HJ, Wenby RB, Gruber T, Marder V, Coates TD, Kohn DB

Hematopoietic cell transplantation for nonmalignant disorders.

Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation

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.)

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

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

Umbilical cord blood transplantation for thalassemia major.

Hematology/oncology clinics of North America

Kanathezhath B, Walters MC

HLA-matched sibling bone marrow transplantation for ß-thalassemia major.

Blood

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

Walters MC

Stem-cell transplantation for sickle cell disease.

The New England journal of medicine

Walters MC, Sullivan KM

Transplant outcomes in bone marrow failure syndromes and hemoglobinopathies.

Seminars in Hematology

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

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, Patience M, Redding-Lallinger R, Roberts IA, Rogers ZR, Sanders JE, Scott JP, Sullivan KM

Human term placenta as a source of hematopoietic cells.

Experimental biology and medicine (Maywood, N.J.)

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

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

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

Michlitsch JG, Walters MC

Transfusional iron burden and liver toxicity after bone marrow transplantation for acute myelogenous leukemia and hemoglobinopathies.

Pediatric blood & cancer

Jastaniah W, Harmatz P, Pakbaz Z, Fischer R, Vichinsky E, Walters MC

Mixed haematopoietic chimerism for sickle cell disease prevents intravascular haemolysis.

British journal of haematology

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

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

Matched-related donor transplantation for sickle cell disease: report from the Center for International Blood and Transplant Research.

British journal of haematology

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

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

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, Ohene-Frempong K, Redding-Lallinger R, Roberts IA, Rogers ZR, Sanders JE, Scott JP, Sleight B, Thompson AA, Sullivan KM, Walters MC

Clinical hemoglobinopathies: iron, lungs and new blood.

Current opinion in hematology

Morris CR, Singer ST, Walters MC

The prevention and management of stroke in sickle cell anaemia.

Expert opinion on biological therapy

Qureshi N, Lubin B, Walters MC

Outcomes of preimplantation genetic diagnosis therapy in treatment of beta-thalassemia: A retrospective analysis.

Annals of the New York Academy of Sciences

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

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

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

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

Butensky E, Pakbaz Z, Foote D, Walters MC, Vichinsky EP, Harmatz P

Haematopoietic cell transplantation in the treatment of sickle cell disease.

Expert opinion on biological therapy

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

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

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, Raja T, Roberts I, Will AM, Yaniv I, Vermylen C, Tannoia N, Garnier F, Ionescu I, Walters MC, Lubin BH, Gluckman E

Comprehensive banking of sibling donor cord blood for children with malignant and nonmalignant disease.

Blood

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

Walters MC, Nienhuis AW, Vichinsky E

Bone marrow transplantation in sickle cell anemia.

Current opinion in oncology

Hoppe CC, Walters MC

Sibling donor cord blood banking for children with sickle cell disease.

Pediatric pathology & molecular medicine

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

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

Stem cell transplantation for sickle cell disease: can we reduce the toxicity?

Pediatric pathology & molecular medicine

Fixler J, Vichinsky E, Walters MC

Collection of sibling donor cord blood for children with thalassemia.

Journal of pediatric hematology/oncology

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

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, Bernaudin F, Wong WY, Scott JP, Margolis D, Vichinsky E, Wall DA, Wayne AS, Pegelow C, Redding-Lallinger R, Wiley J, Klemperer M, Mentzer WC, Smith FO, Sullivan KM

Bone Marrow Transplantation for Non-Malignant Disease.

Hematology. American Society of Hematology. Education Program

Sullivan KM, Parkman R, Walters MC

Bone marrow transplantation for sickle cell disease: where do we go from here?

Journal of pediatric hematology/oncology

Walters MC

The chicken beta-globin 5'HS4 boundary element blocks enhancer-mediated suppression of silencing.

Molecular and cellular biology

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

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

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, Storb R, Hansen JA

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

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 KM

Bone marrow transplantation for sickle cell disease.

The New England journal of medicine

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

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, Roberts IA, Dinndorf P, Brandalise S, Sanders JE, Matthews DC, Appelbaum FR, Storb R, Sullivan KM

Transcriptional enhancers act in cis to suppress position-effect variegation.

Genes & development

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

Walters MC, Fiering S, Eidemiller J, Magis W, Groudine M, Martin DI

Neurologic complications after allogeneic marrow transplantation for sickle cell anemia.

Blood

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

Walters MC, Sullivan KM, O'Reilly RJ, Boulad F, Brockstein J, Blume K, Amylon M, Johnson FL, Klemperer M, Graham-Pole J