Current Environment:

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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 Jan 25:S1934-5909(20)30599-3. doi: 10.1016/j.stem.2020.12.015. Epub ahead of print. PMID: 33513358.