Publications
Frigault MJ, Graham CE, Berger TR, Ritchey J, Horick NK, El-Jawahri A, Scarfò I, Schmidts A, Haradhvala NJ, Wehrli M, Lee WH, Parker AL, Wiggin HR, Bouffard A, Dey A, Leick MB, Katsis K, Elder EL, Dolaher MA, Cook DT, Chekmasova AA, Huang L, Nikiforow S, Daley H, Ritz J, Armant M, Preffer F, DiPersio JF, Nardi V, Chen YB, Gallagher KME, Maus MV. Phase 1 study of CAR-37 T cells in patients with relapsed or refractory CD37+ lymphoid malignancies. Blood. 2024 Sep 12;144(11):1153-1167. doi: 10.1182/blood.2024024104. PMID: 38781564.
Spencer Chapman M, Cull AH, Ciuculescu MF, Esrick EB, Mitchell E, Jung H, O'Neill L, Roberts K, Fabre MA, Williams N, Nangalia J, Quinton J, Fox JM, Pellin D, Makani J, Armant M, Williams DA, Campbell PJ, Kent DG. Clonal selection of hematopoietic stem cells after gene therapy for sickle cell disease. Nat Med. 2023 Dec;29(12):3175-3183. doi: 10.1038/s41591-023-02636-6. Epub 2023 Nov 16. PMID: 37973947; PMCID: PMC10719109.
De Souza DC, Hebert N, Esrick EB, Ciuculescu MF, Archer NM, Armant M, Audureau É, Brendel C, Di Caprio G, Galactéros F, Liu D, McCabe A, Morris E, Schonbrun E, Williams D, Wood DK, Williams DA, Bartolucci P, Higgins JM. Genetic reversal of the globin switch concurrently modulates both fetal and sickle hemoglobin and reduces red cell sickling. Nat Commun. 2023 Sep 20;14(1):5850. doi: 10.1038/s41467-023-40923-5. PMID: 37730674; PMCID: PMC10511721.
Jurkunas UV, Yin J, Johns LK, Li S, Negre H, Shaw KL, Samarakoon L, Ayala AR, Kheirkhah A, Katikireddy K, Gauthier A, Ong Tone S, Kaufman AR, Ellender S, Hernandez Rodriguez DE, Daley H, Dana R, Armant M, Ritz J. Cultivated autologous limbal epithelial cell (CALEC) transplantation: Development of manufacturing process and clinical evaluation of feasibility and safety. Sci Adv. 2023 Aug 18;9(33):eadg6470. doi: 10.1126/sciadv.adg6470. Epub 2023 Aug 18. PMID: 37595035; PMCID: PMC10438443.
Labrosse R, Chu JI, Armant MA, Everett JK, Pellin D, Kareddy N, Frelinger AL, Henderson LA, O'Connell AE, Biswas A, Coenen-van der Spek J, Miggelbrink A, Fiorini C, Adhikari H, Berry CC, Cantu VA, Fong J, Jaroslavsky J, Karadeniz DF, Li QZ, Reddy S, Roche AM, Zhu C, Whangbo JS, Dansereau C, Mackinnon B, Morris E, Koo SM, London WB, Baris S, Ozen A, Karakoc-Aydiner E, Despotovic JM, Forbes Satter LR, Saitoh A, Aizawa Y, King A, Nguyen MAT, Vu VDU, Snapper SB, Galy A, Notarangelo LD, Bushman FD, Williams DA, Pai SY. Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome. Blood. 2023 Oct 12;142(15):1281-1296. doi: 10.1182/blood.2022019117. PMID: 37478401; PMCID: PMC10731922.
Zeng J, Nguyen MA, Liu P, Ferreira da Silva L, Lin LY, Justus DG, Petri K, Clement K, Porter SN, Verma A, Neri NR, Rosanwo T, Ciuculescu MF, Abriss D, Mintzer E, Maitland SA, Demirci S, Tisdale JF, Williams DA, Zhu LJ, Pruett- Miller SM, Pinello L, Joung JK, Pattanayak V, Manis JP, Armant M, Pellin D, Brendel C, Wolfe SA, Bauer DE. Gene editing without <i>ex vivo</i> culture evades genotoxicity in human hematopoietic stem cells. bioRxiv [Preprint]. 2023 May 27:2023.05.27.542323. doi: 10.1101/2023.05.27.542323. PMID: 37292647; PMCID: PMC10245949.
Campbell K, Groshen S, Evans AC, Wilson S, Sebastian A, Loots GG, Marachelian A, Armant M, Pal S, Haas-Kogan DA, Park JR, Granger M, Matthay KK, Coleman MA, DuBois SG. Modulation of Radiation Biomarkers in a Randomized Phase II Study of <sup>131</sup>I-MIBG With or Without Radiation Sensitizers for Relapsed or Refractory Neuroblastoma. Int J Radiat Oncol Biol Phys. 2023 Apr 1;115(5):1115-1128. doi: 10.1016/j.ijrobp.2022.11.045. Epub 2022 Dec 13. PMID: 36526235.
Cancellieri S, Zeng J, Lin LY, Tognon M, Nguyen MA, Lin J, Bombieri N, Maitland SA, Ciuculescu MF, Katta V, Tsai SQ, Armant M, Wolfe SA, Giugno R, Bauer DE, Pinello L. Human genetic diversity alters off-target outcomes of therapeutic gene editing. Nat Genet. 2023 Jan;55(1):34-43. doi: 10.1038/s41588-022-01257-y. Epub 2022 Dec 15. PMID: 36522432; PMCID: PMC10272994.
Pollard JA, Furutani E, Liu S, Esrick E, Cohen LE, Bledsoe J, Liu CW, Lu K, de Haro MJR, Surrallés J, Malsch M, Kuniholm A, Galvin A, Armant M, Kim AS, Ballotti K, Moreau L, Zhou Y, Babushok D, Boulad F, Carroll C, Hartung H, Hont A, Nakano T, Olson T, Sze SG, Thompson AA, Wlodarski MW, Gu X, Libermann TA, D'Andrea A, Grompe M, Weller E, Shimamura A. Metformin for treatment of cytopenias in children and young adults with Fanconi anemia. Blood Adv. 2022 Jun 28;6(12):3803-3811. doi: 10.1182/bloodadvances.2021006490. PMID: 35500223; PMCID: PMC9631552.
Jurkunas U, Johns L, Armant M. Cultivated Autologous Limbal Epithelial Cell Transplantation: New Frontier in the Treatment of Limbal Stem Cell Deficiency. Am J Ophthalmol. 2022 Jul;239:244-268. doi: 10.1016/j.ajo.2022.03.015. Epub 2022 Mar 18. PMID: 35314191; PMCID: PMC9638843.
Chou J, Platt CD, Habiballah S, Nguyen AA, Elkins M, Weeks S, Peters Z, Day-Lewis M, Novak T, Armant M, Williams L, Rockowitz S, Sliz P, Williams DA, Randolph AG, Geha RS; Taking on COVID-19 Together Study Investigators. Mechanisms underlying genetic susceptibility to multisystem inflammatory syndrome in children (MIS-C). J Allergy Clin Immunol. 2021 Sep;148(3):732-738.e1. doi: 10.1016/j.jaci.2021.06.024. Epub 2021 Jul 2. PMID: 34224783; PMCID: PMC8252701.
Rao S, Yao Y, Soares de Brito J, Yao Q, Shen AH, Watkinson RE, Kennedy AL, Coyne S, Ren C, Zeng J, Serbin AV, Studer S, Ballotti K, Harris CE, Luk K, Stevens CS, Armant M, Pinello L, Wolfe SA, Chiarle R, Shimamura A, Lee B, Newburger PE, Bauer DE. Dissecting ELANE neutropenia pathogenicity by human HSC gene editing. Cell Stem Cell. 2021 May 6;28(5):833-845.e5. doi: 10.1016/j.stem.2020.12.015. Epub 2021 Jan 28. PMID: 33513358; PMCID: PMC8106646.
Esrick EB, Lehmann LE, Biffi A, Achebe M, Brendel C, Ciuculescu MF, Daley H, MacKinnon B, Morris E, Federico A, Abriss D, Boardman K, Khelladi R, Shaw K, Negre H, Negre O, Nikiforow S, Ritz J, Pai SY, London WB, Dansereau C, Heeney MM, Armant M, Manis JP, Williams DA. Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease. N Engl J Med. 2021 Jan 21;384(3):205-215. doi: 10.1056/NEJMoa2029392. Epub 2020 Dec 5. PMID: 33283990; PMCID: PMC7962145.
Brendel C, Negre O, Rothe M, Guda S, Parsons G, Harris C, McGuinness M, Abriss D, Tsytsykova A, Klatt D, Bentler M, Pellin D, Christiansen L, Schambach A, Manis J, Trebeden-Negre H, Bonner M, Esrick E, Veres G, Armant M, Williams DA. Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy. Mol Ther Methods Clin Dev. 2020 Mar 17;17:589-600. doi: 10.1016/j.omtm.2020.03.015. PMID: 32300607; PMCID: PMC7150438.
Kohn DB, Booth C, Kang EM, Pai SY, Shaw KL, Santilli G, Armant M, Buckland KF, Choi U, De Ravin SS, Dorsey MJ, Kuo CY, Leon-Rico D, Rivat C, Izotova N, Gilmour K, Snell K, Dip JX, Darwish J, Morris EC, Terrazas D, Wang LD, Bauser CA, Paprotka T, Kuhns DB, Gregg J, Raymond HE, Everett JK, Honnet G, Biasco L, Newburger PE, Bushman FD, Grez M, Gaspar HB, Williams DA, Malech HL, Galy A, Thrasher AJ; Net4CGD consortium. Lentiviral gene therapy for X-linked chronic granulomatous disease. Nat Med. 2020 Feb;26(2):200-206. doi: 10.1038/s41591-019-0735-5. Epub 2020 Jan 27. PMID: 31988463; PMCID: PMC7115833.
Kim J, Hu C, Moufawad El Achkar C, Black LE, Douville J, Larson A, Pendergast MK, Goldkind SF, Lee EA, Kuniholm A, Soucy A, Vaze J, Belur NR, Fredriksen K, Stojkovska I, Tsytsykova A, Armant M, DiDonato RL, Choi J, Cornelissen L, Pereira LM, Augustine EF, Genetti CA, Dies K, Barton B, Williams L, Goodlett BD, Riley BL, Pasternak A, Berry ER, Pflock KA, Chu S, Reed C, Tyndall K, Agrawal PB, Beggs AH, Grant PE, Urion DK, Snyder RO, Waisbren SE, Poduri A, Park PJ, Patterson A, Biffi A, Mazzulli JR, Bodamer O, Berde CB, Yu TW. Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease. N Engl J Med. 2019 Oct 24;381(17):1644-1652. doi: 10.1056/NEJMoa1813279. Epub 2019 Oct 9. PMID: 31597037; PMCID: PMC6961983.
Schott JW, León-Rico D, Ferreira CB, Buckland KF, Santilli G, Armant MA, Schambach A, Cavazza A, Thrasher AJ. Enhancing Lentiviral and Alpharetroviral Transduction of Human Hematopoietic Stem Cells for Clinical Application. Mol Ther Methods Clin Dev. 2019 Jun 7;14:134-147. doi: 10.1016/j.omtm.2019.05.015. PMID: 31338385; PMCID: PMC6629974.
Ellison SM, Liao A, Wood S, Taylor J, Youshani AS, Rowlston S, Parker H, Armant M, Biffi A, Chan L, Farzaneh F, Wynn R, Jones SA, Heal P, Gaspar HB, Bigger BW. Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA. Mol Ther Methods Clin Dev. 2019 Apr 6;13:399-413. doi: 10.1016/j.omtm.2019.04.001. PMID: 31044143; PMCID: PMC6479204.
Esrick EB, Manis JP, Daley H, Baricordi C, Trébéden-Negre H, Pierciey FJ, Armant M, Nikiforow S, Heeney MM, London WB, Biasco L, Asmal M, Williams DA, Biffi A. Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients. Blood Adv. 2018 Oct 9;2(19):2505-2512. doi: 10.1182/bloodadvances.2018016725. PMID: 30282642; PMCID: PMC6177648.
Clarke EL, Connell AJ, Six E, Kadry NA, Abbas AA, Hwang Y, Everett JK, Hofstaedter CE, Marsh R, Armant M, Kelsen J, Notarangelo LD, Collman RG, Hacein- Bey-Abina S, Kohn DB, Cavazzana M, Fischer A, Williams DA, Pai SY, Bushman FD. T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency. Genome Med. 2018 Sep 28;10(1):70. doi: 10.1186/s13073-018-0580-z. PMID: 30261899; PMCID: PMC6161392.
Eichler F, Duncan C, Musolino PL, Orchard PJ, De Oliveira S, Thrasher AJ, Armant M, Dansereau C, Lund TC, Miller WP, Raymond GV, Sankar R, Shah AJ, Sevin C, Gaspar HB, Gissen P, Amartino H, Bratkovic D, Smith NJC, Paker AM, Shamir E, O'Meara T, Davidson D, Aubourg P, Williams DA. Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2017 Oct 26;377(17):1630-1638. doi: 10.1056/NEJMoa1700554. Epub 2017 Oct 4. PMID: 28976817; PMCID: PMC5708849.
Liu S, de Castro LF, Jin P, Civini S, Ren J, Reems JA, Cancelas J, Nayak R, Shaw G, O'Brien T, McKenna DH, Armant M, Silberstein L, Gee AP, Hei DJ, Hematti P, Kuznetsov SA, Robey PG, Stroncek DF. Manufacturing Differences Affect Human Bone Marrow Stromal Cell Characteristics and Function: Comparison of Production Methods and Products from Multiple Centers. Sci Rep. 2017 Apr 27;7:46731. doi: 10.1038/srep46731. PMID: 28447618; PMCID: PMC5406832.
Ouseph S, Tappitake D, Armant M, Wesselschmidt R, Derecho I, Draxler R, Wood D, Centanni JM. Cellular Therapies Clinical Research Roadmap: lessons learned on how to move a cellular therapy into a clinical trial. Cytotherapy. 2015 Apr;17(4):339-43. doi: 10.1016/j.jcyt.2014.10.008. Epub 2014 Dec 4. PMID: 25484311; PMCID: PMC4357554.
Lindblad RW, Ibenana L, Wagner JE, McKenna DH Jr, Hei DJ, Hematti P, Couture LA, Silberstein LE, Armant M, Rooney CM, Gee AP, Welniak LA, Heath Mondoro T, Wood DA, Styers D. Cell therapy product administration and safety: data capture and analysis from the Production Assistance for Cellular Therapies (PACT) program. Transfusion. 2015 Mar;55(3):674-9. doi: 10.1111/trf.12881. Epub 2014 Oct 14. PMID: 25315143; PMCID: PMC4357544.
Hacein-Bey-Abina S, Pai SY, Gaspar HB, Armant M, Berry CC, Blanche S, Bleesing J, Blondeau J, de Boer H, Buckland KF, Caccavelli L, Cros G, De Oliveira S, Fernández KS, Guo D, Harris CE, Hopkins G, Lehmann LE, Lim A, London WB, van der Loo JC, Malani N, Male F, Malik P, Marinovic MA, McNicol AM, Moshous D, Neven B, Oleastro M, Picard C, Ritz J, Rivat C, Schambach A, Shaw KL, Sherman EA, Silberstein LE, Six E, Touzot F, Tsytsykova A, Xu-Bayford J, Baum C, Bushman FD, Fischer A, Kohn DB, Filipovich AH, Notarangelo LD, Cavazzana M, Williams DA, Thrasher AJ. A modified γ-retrovirus vector for X-linked severe combined immunodeficiency. N Engl J Med. 2014 Oct 9;371(15):1407-17. doi: 10.1056/NEJMoa1404588. PMID: 25295500; PMCID: PMC4274995.
Wood D, Wesselschmidt R, Hematti P, Gee AP, Rooney C, Silberstein L, Armant M, Couture L, Wagner JE, McKenna DH Jr, Hei D, Mondoro TH, Welniak L, Lindblad R. An update from the United States National Heart, Lung, and Blood Institute- funded Production Assistance for Cellular Therapies (PACT) program: a decade of cell therapy. Clin Transl Sci. 2014 Apr;7(2):93-9. doi: 10.1111/cts.12148. Epub 2014 Mar 21. PMID: 24655892; PMCID: PMC4006308.
Eaker S, Armant M, Brandwein H, Burger S, Campbell A, Carpenito C, Clarke D, Fong T, Karnieli O, Niss K, Van't Hof W, Wagey R. Concise review: guidance in developing commercializable autologous/patient-specific cell therapy manufacturing. Stem Cells Transl Med. 2013 Nov;2(11):871-83. doi: 10.5966/sctm.2013-0050. Epub 2013 Oct 7. PMID: 24101671; PMCID: PMC3808202.
Cutler C, Multani P, Robbins D, Kim HT, Le T, Hoggatt J, Pelus LM, Desponts C, Chen YB, Rezner B, Armand P, Koreth J, Glotzbecker B, Ho VT, Alyea E, Isom M, Kao G, Armant M, Silberstein L, Hu P, Soiffer RJ, Scadden DT, Ritz J, Goessling W, North TE, Mendlein J, Ballen K, Zon LI, Antin JH, Shoemaker DD. Prostaglandin-modulated umbilical cord blood hematopoietic stem cell transplantation. Blood. 2013 Oct 24;122(17):3074-81. doi: 10.1182/blood-2013-05-503177. Epub 2013 Aug 30. PMID: 23996087; PMCID: PMC3811179.
Sharma SM, Fuchsluger T, Ahmad S, Katikireddy KR, Armant M, Dana R, Jurkunas UV. Comparative analysis of human-derived feeder layers with 3T3 fibroblasts for the ex vivo expansion of human limbal and oral epithelium. Stem Cell Rev Rep. 2012 Sep;8(3):696-705. doi: 10.1007/s12015-011-9319-6. PMID: 21964568.
Goessling W, Allen RS, Guan X, Jin P, Uchida N, Dovey M, Harris JM, Metzger ME, Bonifacino AC, Stroncek D, Stegner J, Armant M, Schlaeger T, Tisdale JF, Zon LI, Donahue RE, North TE. Prostaglandin E2 enhances human cord blood stem cell xenotransplants and shows long-term safety in preclinical nonhuman primate transplant models. Cell Stem Cell. 2011 Apr 8;8(4):445-58. doi: 10.1016/j.stem.2011.02.003. PMID: 21474107; PMCID: PMC3148081.
Turner CG, Klein JD, Steigman SA, Armant M, Nicksa GA, Zurakowski D, Ritz J, Fauza DO. Preclinical regulatory validation of an engineered diaphragmatic tendon made with amniotic mesenchymal stem cells. J Pediatr Surg. 2011 Jan;46(1):57-61. doi: 10.1016/j.jpedsurg.2010.09.063. PMID: 21238640.
Steigman SA, Armant M, Bayer-Zwirello L, Kao GS, Silberstein L, Ritz J, Fauza DO. Preclinical regulatory validation of a 3-stage amniotic mesenchymal stem cell manufacturing protocol. J Pediatr Surg. 2008 Jun;43(6):1164-9. doi: 10.1016/j.jpedsurg.2008.02.052. PMID: 18558201; PMCID: PMC2478751.
Kunisaki SM, Armant M, Kao GS, Stevenson K, Kim H, Fauza DO. Tissue engineering from human mesenchymal amniocytes: a prelude to clinical trials. J Pediatr Surg. 2007 Jun;42(6):974-9; discussion 979-80. doi: 10.1016/j.jpedsurg.2007.01.031. PMID: 17560205.