Mark Walters, MD

Title(s)Professor, Pediatrics
SchoolSchool of Medicine
Address35 Medical Center Way, #903D
San Francisco CA 94143
ORCID ORCID Icon0000-0002-6515-4559 Additional info
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    Collapse Biography 
    Collapse Education and Training
    University of California, Berkeley, Berkeley, CAA.B.06/1981Genetics
    University of California, San Diego, La Jolla, CAM.D.06/1985Medicine
    University of Washington, Seattle, WA06/1988Pediatric Residency
    Fred Hutch/Univ of Washington, Seattle, WA06/1991Pediatric Hem/Onc/BMT fellowship
    University of California, San Francisco, CA2019Diversity, Equity, and Inclusion Champion Training

    Collapse Overview 
    Collapse Overview
    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.

    Collapse Research 
    Collapse Research Activities and Funding
    Addressing safety issues by quantify large deletions and chromosomal rearrangements in HBB gene editing
    NIH OT2HL154977Apr 25, 2020 - Mar 31, 2022
    Role: Co-Investigator
    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 OT2HL152643Sep 20, 2019 - Jun 30, 2021
    Role: Co-Investigator
    Curing Sickle Cell Disease with CRISPR-Cas9 genome editing
    NIH OT2HL151319Jun 12, 2019 - May 31, 2021
    Role: Principal Investigator
    Hematopoietic Stem Cell Transplantation for Young Adults with Sickle Cell Disease - Clinical Coordinating Center
    NIH U01HL128566Sep 1, 2015 - Jul 31, 2021
    Role: Co-Principal Investigator
    Hematopoietic Cell Therapy for Young Adults with Severe Sickle Cell Disease
    NIH R34HL108761Sep 1, 2011 - Apr 30, 2016
    Role: Co-Principal Investigator
    Northern California Consortium for Sickle Cell Disease
    NIH U10HL083704Apr 18, 2006 - Mar 31, 2011
    Role: Principal Investigator
    Induction of Stable Chimerism for Sickle Cell Anemia
    NIH U01HL068091Aug 25, 2001 - Jul 31, 2007
    Role: Principal Investigator
    MECHANISMS OF GLOBIN GENE SILENCING
    NIH R29HL060927Jul 10, 1998 - Jun 30, 2002
    Role: Principal Investigator
    MECHANISMS OF ERYTHROID GENE EXPRESSION
    NIH K08HL003098Jul 1, 1994 - Jun 30, 1999
    Role: Principal Investigator
    GLOBIN GENE REGULATION BY GATA-1 AND CHROMATIN
    NIH R01HL048790Aug 1, 1992 - Jun 30, 2001
    Role: Co-Principal Investigator

    Collapse ORNG Applications 
    Collapse Clinical Trials

    Collapse Bibliographic 
    Collapse Publications
    Publications listed below are automatically derived from MEDLINE/PubMed and other sources, which might result in incorrect or missing publications. Researchers can login to make corrections and additions, or contact us for help. to make corrections and additions.
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    Altmetrics Details PMC Citations indicate the number of times the publication was cited by articles in PubMed Central, and the Altmetric score represents citations in news articles and social media. (Note that publications are often cited in additional ways that are not shown here.) Fields are based on how the National Library of Medicine (NLM) classifies the publication's journal and might not represent the specific topic of the publication. Translation tags are based on the publication type and the MeSH terms NLM assigns to the publication. Some publications (especially newer ones and publications not in PubMed) might not yet be assigned Field or Translation tags.) Click a Field or Translation tag to filter the publications.
    1. Long-term outcomes after unrelated donor transplantation for severe sickle cell disease on the BMT CTN 0601 trial. Am J Hematol. 2024 Apr; 99(4):785-788. 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. PMID: 38343182; PMCID: PMC10947844.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    2. Defining curative endpoints for transfusion-dependent β-thalassemia in the era of gene therapy and gene editing. Am J Hematol. 2024 Mar; 99(3):422-429. Corbacioglu S, Frangoul H, Locatelli F, Hobbs W, Walters M. PMID: 38100154.
      View in: PubMed   Mentions: 1     Fields:    Translation:Humans
    3. Defining curative endpoints for sickle cell disease in the era of gene therapy and gene editing. Am J Hematol. 2024 Mar; 99(3):430-438. Locatelli F, Corbacioglu S, Hobbs W, Frangoul H, Walters MC. PMID: 38010293.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    4. Allogeneic haematopoietic stem-cell transplantation versus gene therapy for haemoglobinopathies. Lancet Haematol. 2023 10; 10(10):e798-e800. Olson TS, Walters MC. PMID: 37793770.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    5. 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. 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. PMID: 36966871; PMCID: PMC10247402.
      View in: PubMed   Mentions: 2     Fields:    Translation:HumansCells
    6. 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. 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. PMID: 36775201; PMCID: PMC10149622.
      View in: PubMed   Mentions: 5     Fields:    Translation:Humans
    7. Secondary Neoplasms After Hematopoietic Cell Transplant for Sickle Cell Disease. J Clin Oncol. 2023 04 20; 41(12):2227-2237. 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. PMID: 36623245; PMCID: PMC10448940.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    8. Allogeneic Transplant and Gene Therapy: Evolving Toward a Cure. Hematol Oncol Clin North Am. 2022 12; 36(6):1313-1335. Lawal RA, Walters MC, Fitzhugh CD. PMID: 36400545; PMCID: PMC9681017.
      View in: PubMed   Mentions: 1     Fields:    Translation:Humans
    9. 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. Krishnamurti L, Neuberg D, Sullivan KM, Smith S, Eapen M, Walters MC. PMID: 36270432; PMCID: PMC10539686.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    10. 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. 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. PMID: 36161320; PMCID: PMC10092845.
      View in: PubMed   Mentions: 8     Fields:    Translation:HumansCells
    11. High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation. iScience. 2022 Jun 17; 25(6):104374. 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. PMID: 35633935; PMCID: PMC9130532.
      View in: PubMed   Mentions: 19  
    12. Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease. N Engl J Med. 2022 02 17; 386(7):617-628. 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. PMID: 34898139.
      View in: PubMed   Mentions: 63     Fields:    Translation:HumansCellsCTClinical Trials
    13. Betibeglogene Autotemcel Gene Therapy for Non-β0/β0 Genotype β-Thalassemia. N Engl J Med. 2022 02 03; 386(5):415-427. 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. PMID: 34891223.
      View in: PubMed   Mentions: 42     Fields:    Translation:HumansCellsCTClinical Trials
    14. American Society of Hematology 2021 guidelines for sickle cell disease: stem cell transplantation. Blood Adv. 2021 09 28; 5(18):3668-3689. 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. PMID: 34581773; PMCID: PMC8945587.
      View in: PubMed   Mentions: 11     Fields:    Translation:Humans
    15. 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. 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. PMID: 34461278; PMCID: PMC8556300.
      View in: PubMed   Mentions: 5     Fields:    Translation:Humans
    16. 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. 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. PMID: 33958740; PMCID: PMC8416746.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    17. Induction of Fetal Hemoglobin by Gene Therapy. N Engl J Med. 2021 01 21; 384(3):284-285. Walters MC. PMID: 33471981.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    18. Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease. Blood. 2020 07 30; 136(5):623-626. 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. PMID: 32518950; PMCID: PMC7393258.
      View in: PubMed   Mentions: 14     Fields:    Translation:Humans
    19. Sickle marrow: double, double toil and trouble. Blood. 2020 06 04; 135(23):2017-2018. Walters MC. PMID: 32497220.
      View in: PubMed   Mentions:    Fields:    Translation:Animals
    20. 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. 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. PMID: 32401372.
      View in: PubMed   Mentions: 7     Fields:    Translation:HumansCTClinical Trials
    21. 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. Hsieh MM, Bonner M, Pierciey FJ, Uchida N, Rottman J, Demopoulos L, Schmidt M, Kanter J, Walters MC, Thompson AA, Asmal M, Tisdale JF. PMID: 32396618; PMCID: PMC7218414.
      View in: PubMed   Mentions: 47     Fields:    Translation:HumansCells
    22. 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. 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. PMID: 31809537; PMCID: PMC6963248.
      View in: PubMed   Mentions: 10     Fields:    Translation:Humans
    23. Related and unrelated donor transplantation for β-thalassemia major: results of an international survey. Blood Adv. 2019 09 10; 3(17):2562-2570. 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. PMID: 31471325; PMCID: PMC6737407.
      View in: PubMed   Mentions: 22     Fields:    Translation:Humans
    24. Effect of donor type and conditioning regimen intensity on allogeneic transplantation outcomes in patients with sickle cell disease: a retrospective multicentre, cohort study. Lancet Haematol. 2019 Nov; 6(11):e585-e596. 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. PMID: 31495699; PMCID: PMC6813907.
      View in: PubMed   Mentions: 56     Fields:    Translation:Humans
    25. Bone marrow transplantation for adolescents and young adults with sickle cell disease: Results of a prospective multicenter pilot study. Am J Hematol. 2019 04; 94(4):446-454. 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. PMID: 30637784; PMCID: PMC6542639.
      View in: PubMed   Mentions: 24     Fields:    Translation:HumansCTClinical Trials
    26. CRISPR-Cas9 interrogation of a putative fetal globin repressor in human erythroid cells. PLoS One. 2019; 14(1):e0208237. Chung JE, Magis W, Vu J, Heo SJ, Wartiovaara K, Walters MC, Kurita R, Nakamura Y, Boffelli D, Martin DIK, Corn JE, DeWitt MA. PMID: 30645582; PMCID: PMC6333401.
      View in: PubMed   Mentions: 16     Fields:    Translation:HumansCells
    27. Promise of gene therapy to treat sickle cell disease. Expert Opin Biol Ther. 2018 11; 18(11):1123-1136. Romero Z, DeWitt M, Walters MC. PMID: 30324810.
      View in: PubMed   Mentions: 10     Fields:    Translation:HumansCells
    28. 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 Transplant. 2019 02; 54(2):226-235. 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. PMID: 29899571.
      View in: PubMed   Mentions: 3     Fields:    Translation:HumansCTClinical Trials
    29. CIRM Alpha Stem Cell Clinics: Collaboratively Addressing Regenerative Medicine Challenges. Cell Stem Cell. 2018 06 01; 22(6):801-805. Jamieson CHM, Millan MT, Creasey AA, Lomax G, Donohoe ME, Walters MC, Abedi M, Bota DA, Zaia JA, Adams JS. PMID: 29859170.
      View in: PubMed   Mentions: 2     Fields:    Translation:HumansCells
    30. Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia. N Engl J Med. 2018 04 19; 378(16):1479-1493. 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. PMID: 29669226.
      View in: PubMed   Mentions: 261     Fields:    Translation:HumansCellsCTClinical Trials
    31. 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. Biol Blood Marrow Transplant. 2018 07; 24(7):1313-1321. 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. PMID: 29653206.
      View in: PubMed   Mentions: 17     Fields:    Translation:HumansPHPublic Health
    32. Unrelated Donor Transplantation in Children with Thalassemia using Reduced-Intensity Conditioning: The URTH Trial. Biol Blood Marrow Transplant. 2018 06; 24(6):1216-1222. 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. PMID: 29374585; PMCID: PMC5993578.
      View in: PubMed   Mentions: 9     Fields:    Translation:HumansCTClinical Trials
    33. The case for HLA-identical sibling hematopoietic stem cell transplantation in children with symptomatic sickle cell anemia. Blood Adv. 2017 Dec 12; 1(26):2563-2567. Fitzhugh CD, Walters MC. PMID: 29296908; PMCID: PMC5728630.
      View in: PubMed   Mentions: 8     Fields:    
    34. Relationship between Mixed Donor-Recipient Chimerism and Disease Recurrence after Hematopoietic Cell Transplantation for Sickle Cell Disease. Biol Blood Marrow Transplant. 2017 Dec; 23(12):2178-2183. 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 Marrow Transplant Research. PMID: 28882446; PMCID: PMC5782809.
      View in: PubMed   Mentions: 36     Fields:    Translation:Humans
    35. Clinical risks and healthcare utilization of hematopoietic cell transplantation for sickle cell disease in the USA using merged databases. Haematologica. 2017 11; 102(11):1823-1832. 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. PMID: 28818869; PMCID: PMC5664386.
      View in: PubMed   Mentions: 16     Fields:    Translation:Humans
    36. Dose-adapted post-transplant cyclophosphamide for HLA-haploidentical transplantation in Fanconi anemia. Bone Marrow Transplant. 2017 Apr; 52(4):570-573. Thakar MS, Bonfim C, Walters MC, Storb R, Pasquini R, Burroughs L, Sandmaier BM, Woolfrey A, Kiem HP. PMID: 28067886; PMCID: PMC5382060.
      View in: PubMed   Mentions: 2     Fields:    Translation:HumansCells
    37. 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. Biol Blood Marrow Transplant. 2017 Apr; 23(4):552-561. 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. PMID: 28065838.
      View in: PubMed   Mentions: 31     Fields:    Translation:Humans
    38. Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation. Blood. 2017 03 16; 129(11):1548-1556. 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, Eurocord, the Pediatric Working Party of the European Society for Blood and Marrow Transplantation, . PMID: 27965196; PMCID: PMC5356458.
      View in: PubMed   Mentions: 148     Fields:    Translation:Humans
    39. Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells. Sci Transl Med. 2016 10 12; 8(360):360ra134. 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. PMID: 27733558; PMCID: PMC5500303.
      View in: PubMed   Mentions: 211     Fields:    Translation:HumansAnimalsCells
    40. 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. Biol Blood Marrow Transplant. 2017 01; 23(1):24-29. 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. PMID: 27737772; PMCID: PMC5267609.
      View in: PubMed   Mentions: 14     Fields:    Translation:HumansPHPublic Health
    41. A trial of unrelated donor marrow transplantation for children with severe sickle cell disease. Blood. 2016 11 24; 128(21):2561-2567. 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. PMID: 27625358; PMCID: PMC5123194.
      View in: PubMed   Mentions: 62     Fields:    Translation:HumansCTClinical Trials
    42. Ankyrin-B directs membrane tethering of periaxin and is required for maintenance of lens fiber cell hexagonal shape and mechanics. Am J Physiol Cell Physiol. 2016 Jan 15; 310(2):C115-26. Maddala R, Walters M, Brophy PJ, Bennett V, Rao PV. PMID: 26538089; PMCID: PMC4719034.
      View in: PubMed   Mentions: 15     Fields:    Translation:AnimalsCells
    43. Indications and Results of HLA-Identical Sibling Hematopoietic Cell Transplantation for Sickle Cell Disease. Biol Blood Marrow Transplant. 2016 Feb; 22(2):207-211. Walters MC, De Castro LM, Sullivan KM, Krishnamurti L, Kamani N, Bredeson C, Neuberg D, Hassell KL, Farnia S, Campbell A, Petersdorf E. PMID: 26500093; PMCID: PMC5031360.
      View in: PubMed   Mentions: 41     Fields:    Translation:Humans
    44. A booster shot to cure hemoglobinopathies. Blood. 2015 Sep 03; 126(10):1159-61. Walters MC, MacKenzie TC. PMID: 26337353.
      View in: PubMed   Mentions: 1     Fields:    Translation:Animals
    45. Update of hematopoietic cell transplantation for sickle cell disease. Curr Opin Hematol. 2015 May; 22(3):227-33. Walters MC. PMID: 25767957; PMCID: PMC5037959.
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    46. In utero hematopoietic cell transplantation for hemoglobinopathies. Front Pharmacol. 2014; 5:278. Derderian SC, Jeanty C, Walters MC, Vichinsky E, MacKenzie TC. PMID: 25628564; PMCID: PMC4290536.
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    47. One-unit versus two-unit cord-blood transplantation for hematologic cancers. N Engl J Med. 2014 Oct 30; 371(18):1685-94. 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 Trials Network. PMID: 25354103; PMCID: PMC4257059.
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    48. Phenotyping: targeting genotype's rich cousin for diagnosis. J Paediatr Child Health. 2015 Apr; 51(4):381-6. 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. PMID: 25109851.
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    49. Combined umbilical cord blood and bone marrow from HLA-identical sibling donors for hematopoietic stem cell transplantation in children with hemoglobinopathies. Pediatr Blood Cancer. 2014 Sep; 61(9):1690-4. Soni S, Boulad F, Cowan MJ, Scaradavou A, Dahake J, Edwards S, Walters MC. PMID: 24803091.
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    50. 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. J Clin Apher. 2014 Oct; 29(5):273-80. Karafin MS, Graminske S, Erickson P, Walters MC, Scott EP, Carter S, Padmanabhan A. PMID: 24677347.
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    51. Thiol/redox metabolomic profiling implicates GSH dysregulation in early experimental graft versus host disease (GVHD). PLoS One. 2014; 9(2):e88868. Suh JH, Kanathezhath B, Shenvi S, Guo H, Zhou A, Tiwana A, Kuypers F, Ames BN, Walters MC. PMID: 24558439; PMCID: PMC3928313.
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    52. β-globin gene transfer to human bone marrow for sickle cell disease. J Clin Invest. 2013 Jul 01. 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. PMID: 23863630; PMCID: PMC4011030.
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    53. Outcome of patients with hemoglobinopathies given either cord blood or bone marrow transplantation from an HLA-identical sibling. Blood. 2013 Aug 08; 122(6):1072-8. 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, Eurocord and European Blood and Marrow Transplantation (EBMT) group. PMID: 23692854.
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    54. 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). Biol Blood Marrow Transplant. 2012 Aug; 18(8):1265-72. 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, Shenoy S. PMID: 22343376; PMCID: PMC3618440.
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    55. Hematopoietic cell transplantation for nonmalignant disorders. Biol Blood Marrow Transplant. 2012 Jan; 18(1 Suppl):S166-71. Tolar J, Mehta PA, Walters MC. PMID: 22226101.
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    56. Improved engraftment with minimal graft-versus-host disease after major histocompatibility complex-mismatched cord blood transplantation with photochemically treated donor lymphocytes. Exp Biol Med (Maywood). 2011 Apr 01; 236(4):492-504. Kanathezhath B, Mizokami M, Stanislaus S, Hounshell C, Neumayr L, Guo H, Hearst JE, Walters MC, Kuypers FA. PMID: 21454375.
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    57. Umbilical cord blood transplantation for children with thalassemia and sickle cell disease. Biol Blood Marrow Transplant. 2011 Sep; 17(9):1375-82. 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 Registry, Center for International Blood and Marrow Transplant Research, New York Blood Center. PMID: 21277376; PMCID: PMC3395002.
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    58. Umbilical cord blood transplantation for thalassemia major. Hematol Oncol Clin North Am. 2010 Dec; 24(6):1165-77. Kanathezhath B, Walters MC. PMID: 21075286.
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    59. HLA-matched sibling bone marrow transplantation for β-thalassemia major. Blood. 2011 Feb 03; 117(5):1745-50. 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. PMID: 21119108; PMCID: PMC3056598.
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    60. Gene therapy and bone marrow transplantation for thalassemia: changing of the guard? Mol Ther. 2010 Sep; 18(9):1577. Walters MC. PMID: 20808320; PMCID: PMC2956918.
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    61. Stem-cell transplantation for sickle cell disease. N Engl J Med. 2010 Mar 11; 362(10):955-6; author reply 956. Walters MC, Sullivan KM. PMID: 20225347.
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    62. Transplant outcomes in bone marrow failure syndromes and hemoglobinopathies. Semin Hematol. 2010 Jan; 47(1):37-45. MacMillan ML, Walters MC, Gluckman E. PMID: 20109610.
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    63. Pulmonary, gonadal, and central nervous system status after bone marrow transplantation for sickle cell disease. Biol Blood Marrow Transplant. 2010 Feb; 16(2):263-72. 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, Multicenter Study of Bone Marrow Transplantation for Sickle Cell Disease. PMID: 19822218; PMCID: PMC2919571.
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    64. Translational stability for sickle gene therapy. Gene Ther. 2009 Aug; 16(8):943. Walters MC. PMID: 19554033.
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    65. Human term placenta as a source of hematopoietic cells. Exp Biol Med (Maywood). 2009 Jul; 234(7):813-23. Serikov V, Hounshell C, Larkin S, Green W, Ikeda H, Walters MC, Kuypers FA. PMID: 19429852; PMCID: PMC3389511.
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    66. Risk factors affecting outcome of second HLA-matched sibling donor transplantations for graft failure in severe acquired aplastic anemia. Biol Blood Marrow Transplant. 2009 May; 15(5):626-31. Horan JT, Carreras J, Tarima S, Camitta BM, Gale RP, Hale GA, Hinterberger W, Marsh J, Passweg JR, Walters MC, Eapen M. PMID: 19361755; PMCID: PMC2692140.
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    67. Newborn screening for hemoglobinopathies in California. Pediatr Blood Cancer. 2009 Apr; 52(4):486-90. Michlitsch J, Azimi M, Hoppe C, Walters MC, Lubin B, Lorey F, Vichinsky E. PMID: 19061217; PMCID: PMC4755934.
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    68. Recent advances in bone marrow transplantation in hemoglobinopathies. Curr Mol Med. 2008 Nov; 8(7):675-89. Michlitsch JG, Walters MC. PMID: 18991653.
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    69. Transfusional iron burden and liver toxicity after bone marrow transplantation for acute myelogenous leukemia and hemoglobinopathies. Pediatr Blood Cancer. 2008 Feb; 50(2):319-24. Jastaniah W, Harmatz P, Pakbaz Z, Fischer R, Vichinsky E, Walters MC. PMID: 17557314.
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    70. Hematopoietic cell transplantation for thalassemia and sickle cell disease: past, present and future. Bone Marrow Transplant. 2008 Jan; 41(2):109-17. Bhatia M, Walters MC. PMID: 18059330.
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    71. Mixed haematopoietic chimerism for sickle cell disease prevents intravascular haemolysis. Br J Haematol. 2007 Nov; 139(3):504-7. 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. PMID: 17910640; PMCID: PMC2742964.
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    72. 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 Dec 15; 110(13):4584-7. 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. PMID: 17827386.
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    73. Cord blood transplantation for sickle cell anemia: bust or boom? Pediatr Transplant. 2007 Sep; 11(6):582-3. Walters MC. PMID: 17663678.
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    74. Matched-related donor transplantation for sickle cell disease: report from the Center for International Blood and Transplant Research. Br J Haematol. 2007 Jun; 137(5):479-85. 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 Committee, Center for International Blood and Marrow Transpla. PMID: 17459050.
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    75. 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. Br J Haematol. 2007 Feb; 136(4):673-6. 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, Multicenter Study of HCT for SCA. PMID: 17223910.
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    76. Clinical hemoglobinopathies: iron, lungs and new blood. Curr Opin Hematol. 2006 Nov; 13(6):407-18. Morris CR, Singer ST, Walters MC. PMID: 17053452.
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    77. The prevention and management of stroke in sickle cell anaemia. Expert Opin Biol Ther. 2006 Nov; 6(11):1087-98. Qureshi N, Lubin B, Walters MC. PMID: 17049007.
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    78. 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 Transplant. 2005 Jan; 35(2):171-7. Horan JT, Liesveld JL, Fenton P, Blumberg N, Walters MC. PMID: 15531901.
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    79. Sibling donor cord blood transplantation for thalassemia major: Experience of the Sibling Donor Cord Blood Program. Ann N Y Acad Sci. 2005; 1054:206-13. Walters MC, Quirolo L, Trachtenberg ET, Edwards S, Hale L, Lee J, Morton-Wiley J, Quirolo K, Robertson S, Saba J, Lubin B. PMID: 16339667.
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    80. Stem cell transplantation with S-59 photochemically treated T-cell add-backs to establish allochimerism in murine thalassemia. Ann N Y Acad Sci. 2005; 1054:214-22. Kuypers FA, Watson G, Sage E, Walters MC, Hamrick J, Hearst JE. PMID: 16339668.
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    81. Outcomes of preimplantation genetic diagnosis therapy in treatment of beta-thalassemia: A retrospective analysis. Ann N Y Acad Sci. 2005; 1054:500-3. Qureshi N, Foote D, Walters MC, Singer ST, Quirolo K, Vichinsky EP. PMID: 16339706.
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    82. Stem cell therapy for sickle cell disease: transplantation and gene therapy. Hematology Am Soc Hematol Educ Program. 2005; 66-73. Walters MC. PMID: 16304361.
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    83. Treatment of hepatitis C virus infection in thalassemia. Ann N Y Acad Sci. 2005; 1054:290-9. Butensky E, Pakbaz Z, Foote D, Walters MC, Vichinsky EP, Harmatz P. PMID: 16339677.
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    84. Sickle cell anemia and hematopoietic cell transplantation: When is a pound of cure worth more than an ounce of prevention? Pediatr Transplant. 2004 Jun; 8 Suppl 5:33-8. Walters MC. PMID: 15125704.
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    85. Haematopoietic cell transplantation in the treatment of sickle cell disease. Expert Opin Biol Ther. 2003 Dec; 3(8):1215-24. Atkins RC, Walters MC. PMID: 14640947.
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    86. Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia. Biol Blood Marrow Transplant. 2003 Aug; 9(8):519-28. Iannone R, Casella JF, Fuchs EJ, Chen AR, Jones RJ, Woolfrey A, Amylon M, Sullivan KM, Storb RF, Walters MC. PMID: 12931121.
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    87. Comprehensive banking of sibling donor cord blood for children with malignant and nonmalignant disease. Blood. 2003 Jan 01; 101(1):351-7. Reed W, Smith R, Dekovic F, Lee JY, Saba JD, Trachtenberg E, Epstein J, Haaz S, Walters MC, Lubin BH. PMID: 12393579.
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    88. Sibling donor cord blood banking for children with sickle cell disease. Pediatr Pathol Mol Med. 2001 Mar-Apr; 20(2):167-74. Reed W, Walters M, Trachtenberg E, Smith R, Lubin BH. PMID: 12673840.
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    89. Bone marrow transplantation in sickle cell anemia. Curr Opin Oncol. 2001 Mar; 13(2):85-90. Hoppe CC, Walters MC. PMID: 11224704.
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    90. Stable mixed hematopoietic chimerism after bone marrow transplantation for sickle cell anemia. Biol Blood Marrow Transplant. 2001; 7(12):665-73. 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 Investigation of Bone Marrow Transplantation for Sickle Cell Disease. PMID: 11787529.
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    92. Collection of sibling donor cord blood for children with thalassemia. J Pediatr Hematol Oncol. 2000 Nov-Dec; 22(6):602-4. Reed W, Walters M, Lubin BH. PMID: 11132238.
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    93. 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 Mar 15; 95(6):1918-24. 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. PMID: 10706855.
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    94. Bone Marrow Transplantation for Non-Malignant Disease. Hematology Am Soc Hematol Educ Program. 2000; 319-338. Sullivan KM, Parkman R, Walters MC. PMID: 11701549.
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    96. A functional enhancer suppresses silencing of a transgene and prevents its localization close to centrometric heterochromatin. Cell. 1999 Oct 29; 99(3):259-69. Francastel C, Walters MC, Groudine M, Martin DI. PMID: 10555142.
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    97. The chicken beta-globin 5'HS4 boundary element blocks enhancer-mediated suppression of silencing. Mol Cell Biol. 1999 May; 19(5):3714-26. Walters MC, Fiering S, Bouhassira EE, Scalzo D, Goeke S, Magis W, Garrick D, Whitelaw E, Martin DI. PMID: 10207095; PMCID: PMC84188.
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    98. Current and future preparative regimens for bone marrow transplantation in thalassemia. Ann N Y Acad Sci. 1998 Jun 30; 850:276-87. Storb R, Yu C, Deeg HJ, Georges G, Kiem HP, Mcsweeney PA, Nash RA, Sandmaier BM, Sullivan KM, Wagner JL, Walters MC. PMID: 9668549.
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    99. 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. Ann N Y Acad Sci. 1998 Jun 30; 850:312-24. 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. PMID: 9668553.
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    103. Transcriptional enhancers act in cis to suppress position-effect variegation. Genes Dev. 1996 Jan 15; 10(2):185-95. Walters MC, Magis W, Fiering S, Eidemiller J, Scalzo D, Groudine M, Martin DI. PMID: 8566752.
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    104. Enhancers increase the probability but not the level of gene expression. Proc Natl Acad Sci U S A. 1995 Jul 18; 92(15):7125-9. Walters MC, Fiering S, Eidemiller J, Magis W, Groudine M, Martin DI. PMID: 7624382; PMCID: PMC41484.
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    105. Neurologic complications after allogeneic marrow transplantation for sickle cell anemia. Blood. 1995 Feb 15; 85(4):879-84. Walters MC, Sullivan KM, Bernaudin F, Souillet G, Vannier JP, Johnson FL, Lenarsky C, Powars D, Bunin N, Ohene-Frempong K, et al. PMID: 7849310.
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