Mark Walters, MD

Professor In Residence
M_PEDS-HEMATOLOGY

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

Haploidentical Bone Marrow Transplantation for Sickle Cell Disease.

NEJM evidence

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, Mendizabal A, Abraham A, Bollard C, Cooke K, de la Fuente J, Hanna R, Horowitz MM, Jordan LC, Bakshi N, Krishnamurti L, Leifer E, Mahadeo KM, Shenoy S, Jones RJ, DeBaun MR, Brodsky RA

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

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

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

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

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

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

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

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

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

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

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