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Page 1
Human genetic diversity alters off-target outcomes of therapeutic gene editing.
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. Cancellieri S, et al. Among authors: armant m. Nat Genet. 2023 Jan;55(1):34-43. doi: 10.1038/s41588-022-01257-y. Epub 2022 Dec 15. Nat Genet. 2023. PMID: 36522432 Free PMC article.
Gene editing without ex vivo culture evades genotoxicity in human hematopoietic stem cells.
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. Zeng J, et al. Among authors: armant m. bioRxiv [Preprint]. 2023 May 27:2023.05.27.542323. doi: 10.1101/2023.05.27.542323. bioRxiv. 2023. Update in: Cell Stem Cell. 2024 Dec 11:S1934-5909(24)00400-4. doi: 10.1016/j.stem.2024.11.001 PMID: 37292647 Free PMC article. Updated. Preprint.
Genetic reversal of the globin switch concurrently modulates both fetal and sickle hemoglobin and reduces red cell sickling.
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. De Souza DC, et al. Among authors: armant m. Nat Commun. 2023 Sep 20;14(1):5850. doi: 10.1038/s41467-023-40923-5. Nat Commun. 2023. PMID: 37730674 Free PMC article.
Gene editing without ex vivo culture evades genotoxicity in human hematopoietic stem cells.
Zeng J, Nguyen MA, Liu P, da Silva LF, Levesque S, 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, Cha HJ, Orkin SH, 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. Zeng J, et al. Among authors: armant m. Cell Stem Cell. 2024 Dec 11:S1934-5909(24)00400-4. doi: 10.1016/j.stem.2024.11.001. Online ahead of print. Cell Stem Cell. 2024. PMID: 39672163
Clonal selection of hematopoietic stem cells after gene therapy for sickle cell disease.
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. Spencer Chapman M, et al. Among authors: armant m. Nat Med. 2023 Dec;29(12):3175-3183. doi: 10.1038/s41591-023-02636-6. Epub 2023 Nov 16. Nat Med. 2023. PMID: 37973947 Free PMC article.
Regulated GATA1 expression as a universal gene therapy for Diamond-Blackfan anemia.
Voit RA, Liao X, Caulier A, Antoszewski M, Cohen B, Armant M, Lu HY, Fleming TJ, Kamal E, Wahlster L, Roche AM, Everett JK, Petrichenko A, Huang MM, Clarke W, Myers KC, Forester C, Perez-Atayde A, Bushman FD, Pellin D, Shimamura A, Williams DA, Sankaran VG. Voit RA, et al. Among authors: armant m. Cell Stem Cell. 2024 Nov 8:S1934-5909(24)00374-6. doi: 10.1016/j.stem.2024.10.012. Online ahead of print. Cell Stem Cell. 2024. PMID: 39532107 Free article.
Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome.
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. Labrosse R, et al. Among authors: armant ma. Blood. 2023 Oct 12;142(15):1281-1296. doi: 10.1182/blood.2022019117. Blood. 2023. PMID: 37478401 Free PMC article.
Phase 1 study of CAR-37 T cells in patients with relapsed or refractory CD37+ lymphoid malignancies.
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. Frigault MJ, et al. Among authors: armant m. Blood. 2024 Sep 12;144(11):1153-1167. doi: 10.1182/blood.2024024104. Blood. 2024. PMID: 38781564 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: armant m. 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.
Modulation of Radiation Biomarkers in a Randomized Phase II Study of 131I-MIBG With or Without Radiation Sensitizers for Relapsed or Refractory Neuroblastoma.
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. Campbell K, et al. Among authors: armant m. 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. Int J Radiat Oncol Biol Phys. 2023. PMID: 36526235 Clinical Trial.
52 results