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Page 1
Long-term safety and efficacy of factor IX gene therapy in hemophilia B.
Nathwani AC, Reiss UM, Tuddenham EG, Rosales C, Chowdary P, McIntosh J, Della Peruta M, Lheriteau E, Patel N, Raj D, Riddell A, Pie J, Rangarajan S, Bevan D, Recht M, Shen YM, Halka KG, Basner-Tschakarjan E, Mingozzi F, High KA, Allay J, Kay MA, Ng CY, Zhou J, Cancio M, Morton CL, Gray JT, Srivastava D, Nienhuis AW, Davidoff AM. Nathwani AC, et al. Among authors: cancio m. N Engl J Med. 2014 Nov 20;371(21):1994-2004. doi: 10.1056/NEJMoa1407309. N Engl J Med. 2014. PMID: 25409372 Free PMC article. Clinical Trial.
Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant.
McIntosh J, Lenting PJ, Rosales C, Lee D, Rabbanian S, Raj D, Patel N, Tuddenham EG, Christophe OD, McVey JH, Waddington S, Nienhuis AW, Gray JT, Fagone P, Mingozzi F, Zhou SZ, High KA, Cancio M, Ng CY, Zhou J, Morton CL, Davidoff AM, Nathwani AC. McIntosh J, et al. Among authors: cancio m. Blood. 2013 Apr 25;121(17):3335-44. doi: 10.1182/blood-2012-10-462200. Epub 2013 Feb 20. Blood. 2013. PMID: 23426947 Free PMC article.
CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease.
Sharma A, Boelens JJ, Cancio M, Hankins JS, Bhad P, Azizy M, Lewandowski A, Zhao X, Chitnis S, Peddinti R, Zheng Y, Kapoor N, Ciceri F, Maclachlan T, Yang Y, Liu Y, Yuan J, Naumann U, Yu VWC, Stevenson SC, De Vita S, LaBelle JL. Sharma A, et al. Among authors: cancio m. N Engl J Med. 2023 Aug 31;389(9):820-832. doi: 10.1056/NEJMoa2215643. N Engl J Med. 2023. PMID: 37646679 Free PMC article. Clinical Trial.
CD4+ T-cell reconstitution predicts survival outcomes after acute graft-versus-host-disease: a dual-center validation.
de Koning C, Prockop S, van Roessel I, Kernan N, Klein E, Langenhorst J, Szanto C, Belderbos M, Bierings M, Boulad F, Bresters D, Cancio M, Curran K, Kollen W, O'Reilly R, Scaradavou A, Spitzer B, Versluijs B, Huitema A, Lindemans C, Nierkens S, Boelens JJ. de Koning C, et al. Among authors: cancio m. Blood. 2021 Feb 11;137(6):848-855. doi: 10.1182/blood.2020007905. Blood. 2021. PMID: 33150379 Free PMC article.
A pragmatic multi-institutional approach to understanding transplant-associated thrombotic microangiopathy after stem cell transplant.
Dandoy CE, Rotz S, Alonso PB, Klunk A, Desmond C, Huber J, Ingraham H, Higham C, Dvorak CC, Duncan C, Schoettler M, Lehmann L, Cancio M, Killinger J, Davila B, Phelan R, Mahadeo KM, Khazal S, Lalefar N, Vissa M, Myers K, Wallace G, Nelson A, Khandelwal P, Bhatla D, Gloude N, Anderson E, Huo J, Roehrs P, Auletta JJ, Chima R, Lane A, Davies SM, Jodele S. Dandoy CE, et al. Among authors: cancio m. Blood Adv. 2021 Jan 12;5(1):1-11. doi: 10.1182/bloodadvances.2020003455. Blood Adv. 2021. PMID: 33570619 Free PMC article.
Antithymocyte globulin exposure in CD34+ T-cell-depleted allogeneic hematopoietic cell transplantation.
Lakkaraja M, Scordo M, Mauguen A, Cho C, Devlin S, Ruiz JD, Klein E, Avecilla ST, Boulad F, Cancio MI, Curran KJ, Jakubowski AA, Kernan NA, Kung AL, O'Reilly RJ, Papadopoulos EB, Prockop S, van Roessel I, Scaradavou A, Shaffer BC, Shah G, Spitzer B, Tamari R, Giralt SA, Perales MA, Boelens JJ. Lakkaraja M, et al. Among authors: cancio mi. Blood Adv. 2022 Feb 8;6(3):1054-1063. doi: 10.1182/bloodadvances.2021005584. Blood Adv. 2022. PMID: 34788361 Free PMC article.
Addressing challenges of clinical trials in acute pain: The Pain Management of Vaso-occlusive Crisis in Children and Young Adults with Sickle Cell Disease Study.
Nottage KA, Hankins JS, Faughnan LG, James DM, Richardson J, Christensen R, Kang G, Smeltzer M, Cancio MI, Wang WC, Anghelescu DL. Nottage KA, et al. Among authors: cancio mi. Clin Trials. 2016 Aug;13(4):409-16. doi: 10.1177/1740774516636573. Epub 2016 Mar 21. Clin Trials. 2016. PMID: 27000103 Clinical Trial.
Toxicity and response after CD19-specific CAR T-cell therapy in pediatric/young adult relapsed/refractory B-ALL.
Curran KJ, Margossian SP, Kernan NA, Silverman LB, Williams DA, Shukla N, Kobos R, Forlenza CJ, Steinherz P, Prockop S, Boulad F, Spitzer B, Cancio MI, Boelens JJ, Kung AL, Khakoo Y, Szenes V, Park JH, Sauter CS, Heller G, Wang X, Senechal B, O'Reilly RJ, Riviere I, Sadelain M, Brentjens RJ. Curran KJ, et al. Among authors: cancio mi. Blood. 2019 Dec 26;134(26):2361-2368. doi: 10.1182/blood.2019001641. Blood. 2019. PMID: 31650176 Free PMC article. Clinical Trial.
The clinical severity of hemoglobin S/Black (A γδβ)0 -thalassemia.
Cancio MI, Aygun B, Chui DHK, Rothman JA, Scott JP, Estepp JH, Hankins JS. Cancio MI, et al. Pediatr Blood Cancer. 2017 Nov;64(11):10.1002/pbc.26596. doi: 10.1002/pbc.26596. Epub 2017 Apr 28. Pediatr Blood Cancer. 2017. PMID: 28453928 Free PMC article.
82 results