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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: nguyen ma. 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.
Base Editing of Human Hematopoietic Stem Cells.
Zeng J, Casirati G, Nguyen MA, Genovese P, Bauer DE. Zeng J, et al. Among authors: nguyen ma. Methods Mol Biol. 2023;2606:43-62. doi: 10.1007/978-1-0716-2879-9_5. Methods Mol Biol. 2023. PMID: 36592307 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: nguyen ma. 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.
Direct delivery of stabilized Cas-embedded base editors achieves efficient and accurate editing of clinically relevant targets.
Lee JM, Zeng J, Liu P, Nguyen MA, Loustaunau DS, Bauer DE, Yilmaz NK, Wolfe SA, Schiffer CA. Lee JM, et al. Among authors: nguyen ma. bioRxiv [Preprint]. 2024 Feb 8:2024.02.08.579528. doi: 10.1101/2024.02.08.579528. bioRxiv. 2024. Update in: Nucleic Acids Res. 2024 Dec 16:gkae1217. doi: 10.1093/nar/gkae1217 PMID: 38370706 Free PMC article. Updated. Preprint.
Scalable assessment of genome editing off-targets associated with genetic variants.
Lin J, Nguyen MA, Lin LY, Zeng J, Verma A, Neri NR, da Silva LF, Mucci A, Wolfe S, Shaw KL, Clement K, Brendel C, Pinello L, Pellin D, Bauer DE. Lin J, et al. Among authors: nguyen ma. bioRxiv [Preprint]. 2024 Jul 25:2024.07.24.605019. doi: 10.1101/2024.07.24.605019. bioRxiv. 2024. PMID: 39211178 Free PMC article. Preprint.
BCL11A +58/+55 enhancer-editing facilitates HSPC engraftment and HbF induction in rhesus macaques conditioned with a CD45 antibody-drug conjugate.
Demirci S, Zeng J, Palchaudhuri R, Wu C, Abraham DM, Hayal TB, Essawi K, Nguyen MA, Stasula U, Chu R, Leonard A, Porter SN, Khan MBN, Hinojosa G, Uchida N, Hong S, Lazzarotto CR, Neri NR, da Silva LF, Pellin D, Verma A, Lanieri L, Bhat A, Hammond K, Tate T, Maitland SA, Sheikhsaran F, Bonifacino AC, Krouse AE, Linde NS, Engels T, Golomb J, Tsai SQ, Pruett-Miller SM, Scadden DT, Dunbar CE, Wolfe SA, Donahue RE, Olson LM, Bauer DE, Tisdale JF. Demirci S, et al. Among authors: nguyen ma. Cell Stem Cell. 2024 Dec 5:S1934-5909(24)00376-X. doi: 10.1016/j.stem.2024.10.014. Online ahead of print. Cell Stem Cell. 2024. PMID: 39642886
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: nguyen ma. 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
Corrigendum: An orally available small molecule that targets soluble TNF to deliver anti-TNF biologic-like efficacy in rheumatoid arthritis.
Vugler A, O'connell J, Nguyen MA, Weitz D, Leeuw T, Hickford E, Verbitsky A, Ying X, Rehberg M, Carrington B, Merriman M, Moss A, Nicolas JM, Stanley P, Wright S, Bourne T, Foricher Y, Zhu Z, Brookings D, Horsley H, Heer J, Schio L, Herrmann M, Rao S, Kohlmann M, Florian P. Vugler A, et al. Among authors: nguyen ma. Front Pharmacol. 2024 Oct 16;15:1479557. doi: 10.3389/fphar.2024.1479557. eCollection 2024. Front Pharmacol. 2024. PMID: 39478966 Free PMC article.
172 results