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Page 1
Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease.
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. Esrick EB, et al. Among authors: shaw k. N Engl J Med. 2021 Jan 21;384(3):205-215. doi: 10.1056/NEJMoa2029392. Epub 2020 Dec 5. N Engl J Med. 2021. PMID: 33283990 Free PMC article. Clinical Trial.
A modified γ-retrovirus vector for X-linked severe combined immunodeficiency.
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. Hacein-Bey-Abina S, et al. N Engl J Med. 2014 Oct 9;371(15):1407-17. doi: 10.1056/NEJMoa1404588. N Engl J Med. 2014. PMID: 25295500 Free PMC article. Clinical Trial.
Lentiviral gene therapy for X-linked chronic granulomatous disease.
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. Kohn DB, et al. Among authors: shaw kl. Nat Med. 2020 Feb;26(2):200-206. doi: 10.1038/s41591-019-0735-5. Epub 2020 Jan 27. Nat Med. 2020. PMID: 31988463 Free PMC article. Clinical Trial.
Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.
Kohn DB, Booth C, Shaw KL, Xu-Bayford J, Garabedian E, Trevisan V, Carbonaro-Sarracino DA, Soni K, Terrazas D, Snell K, Ikeda A, Leon-Rico D, Moore TB, Buckland KF, Shah AJ, Gilmour KC, De Oliveira S, Rivat C, Crooks GM, Izotova N, Tse J, Adams S, Shupien S, Ricketts H, Davila A, Uzowuru C, Icreverzi A, Barman P, Campo Fernandez B, Hollis RP, Coronel M, Yu A, Chun KM, Casas CE, Zhang R, Arduini S, Lynn F, Kudari M, Spezzi A, Zahn M, Heimke R, Labik I, Parrott R, Buckley RH, Reeves L, Cornetta K, Sokolic R, Hershfield M, Schmidt M, Candotti F, Malech HL, Thrasher AJ, Gaspar HB. Kohn DB, et al. Among authors: shaw kl. N Engl J Med. 2021 May 27;384(21):2002-2013. doi: 10.1056/NEJMoa2027675. Epub 2021 May 11. N Engl J Med. 2021. PMID: 33974366 Free PMC article. Clinical Trial.
Phase 1 study of CART-ddBCMA for the treatment of subjects with relapsed and refractory multiple myeloma.
Frigault MJ, Bishop MR, Rosenblatt J, O'Donnell EK, Raje N, Cook D, Yee AJ, Logan E, Avigan DE, Jakubowiak A, Shaw K, Daley H, Nikiforow S, Griffin F, Cornwell C, Shen A, Heery C, Maus MV. Frigault MJ, et al. Among authors: shaw k. Blood Adv. 2023 Mar 14;7(5):768-777. doi: 10.1182/bloodadvances.2022007210. Blood Adv. 2023. PMID: 35468618 Free PMC article. Clinical Trial.
Cultivated autologous limbal epithelial cell (CALEC) transplantation: Development of manufacturing process and clinical evaluation of feasibility and safety.
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. Jurkunas UV, et al. Among authors: shaw kl. Sci Adv. 2023 Aug 18;9(33):eadg6470. doi: 10.1126/sciadv.adg6470. Epub 2023 Aug 18. Sci Adv. 2023. PMID: 37595035 Free PMC article.
A phase 1/2 clinical trial of invariant natural killer T cell therapy in moderate-severe acute respiratory distress syndrome.
Hammond TC, Purbhoo MA, Kadel S, Ritz J, Nikiforow S, Daley H, Shaw K, van Besien K, Gomez-Arteaga A, Stevens D, Ortuzar W, Michelet X, Smith R, Moskowitz D, Masakayan R, Yigit B, Boi S, Soh KT, Chamberland J, Song X, Qin Y, Mishchenko I, Kirby M, Nasonenko V, Buffa A, Buell JS, Chand D, van Dijk M, Stebbing J, Exley MA. Hammond TC, et al. Among authors: shaw k. Nat Commun. 2024 Feb 6;15(1):974. doi: 10.1038/s41467-024-44905-z. Nat Commun. 2024. PMID: 38321023 Free PMC article. Clinical Trial.
Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency.
Reinhardt B, Habib O, Shaw KL, Garabedian E, Carbonaro-Sarracino DA, Terrazas D, Fernandez BC, De Oliveira S, Moore TB, Ikeda AK, Engel BC, Podsakoff GM, Hollis RP, Fernandes A, Jackson C, Shupien S, Mishra S, Davila A, Mottahedeh J, Vitomirov A, Meng W, Rosenfeld AM, Roche AM, Hokama P, Reddy S, Everett J, Wang X, Luning Prak ET, Cornetta K, Hershfield MS, Sokolic R, De Ravin SS, Malech HL, Bushman FD, Candotti F, Kohn DB. Reinhardt B, et al. Among authors: shaw kl. Blood. 2021 Oct 14;138(15):1304-1316. doi: 10.1182/blood.2020010260. Blood. 2021. PMID: 33974038 Free PMC article. Clinical Trial.
Clinical efficacy of gene-modified stem cells in adenosine deaminase-deficient immunodeficiency.
Shaw KL, Garabedian E, Mishra S, Barman P, Davila A, Carbonaro D, Shupien S, Silvin C, Geiger S, Nowicki B, Smogorzewska EM, Brown B, Wang X, de Oliveira S, Choi Y, Ikeda A, Terrazas D, Fu PY, Yu A, Fernandez BC, Cooper AR, Engel B, Podsakoff G, Balamurugan A, Anderson S, Muul L, Jagadeesh GJ, Kapoor N, Tse J, Moore TB, Purdy K, Rishi R, Mohan K, Skoda-Smith S, Buchbinder D, Abraham RS, Scharenberg A, Yang OO, Cornetta K, Gjertson D, Hershfield M, Sokolic R, Candotti F, Kohn DB. Shaw KL, et al. J Clin Invest. 2017 May 1;127(5):1689-1699. doi: 10.1172/JCI90367. Epub 2017 Mar 27. J Clin Invest. 2017. PMID: 28346229 Free PMC article. Clinical Trial.
2,009 results