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Page 1
High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation.
Magis W, DeWitt MA, Wyman SK, Vu JT, Heo SJ, Shao SJ, Hennig F, Romero ZG, Campo-Fernandez B, Said S, McNeill MS, Rettig GR, Sun Y, Wang Y, Behlke MA, Kohn DB, Boffelli D, Walters MC, Corn JE, Martin DIK. Magis W, et al. Among authors: behlke ma. iScience. 2022 May 10;25(6):104374. doi: 10.1016/j.isci.2022.104374. eCollection 2022 Jun 17. iScience. 2022. PMID: 35633935 Free PMC article.
Fine Tuning of Phosphorothioate Inclusion in 2'-O-Methyl Oligonucleotides Contributes to Specific Cell Targeting for Splice-Switching Modulation.
Aoki Y, Rocha CSJ, Lehto T, Miyatake S, Johansson H, Hashimoto Y, Nordin JZ, Mager I, Aoki M, Graham M, Sathyaprakash C, Roberts TC, Wood MJA, Behlke MA, Andaloussi SE. Aoki Y, et al. Among authors: behlke ma. Front Physiol. 2021 Oct 13;12:689179. doi: 10.3389/fphys.2021.689179. eCollection 2021. Front Physiol. 2021. PMID: 34721051 Free PMC article.
Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency.
Fañanas-Baquero S, Quintana-Bustamante O, Dever DP, Alberquilla O, Sanchez-Dominguez R, Camarena J, Ojeda-Perez I, Dessy-Rodriguez M, Turk R, Schubert MS, Lattanzi A, Xu L, Lopez-Lorenzo JL, Bianchi P, Bueren JA, Behlke MA, Porteus M, Segovia JC. Fañanas-Baquero S, et al. Among authors: behlke ma. Mol Ther Methods Clin Dev. 2021 May 14;22:237-248. doi: 10.1016/j.omtm.2021.05.001. eCollection 2021 Sep 10. Mol Ther Methods Clin Dev. 2021. PMID: 34485608 Free PMC article.
Author Correction: AsCas12a ultra nuclease facilitates the rapid generation of therapeutic cell medicines.
Zhang L, Zuris JA, Viswanathan R, Edelstein JN, Turk R, Thommandru B, Rube HT, Glenn SE, Collingwood MA, Bode NM, Beaudoin SF, Lele S, Scott SN, Wasko KM, Sexton S, Borges CM, Schubert MS, Kurgan GL, McNeill MS, Fernandez CA, Myer VE, Morgan RA, Behlke MA, Vakulskas CA. Zhang L, et al. Among authors: behlke ma. Nat Commun. 2021 Jul 19;12(1):4500. doi: 10.1038/s41467-021-24770-w. Nat Commun. 2021. PMID: 34282150 Free PMC article. No abstract available.
AsCas12a ultra nuclease facilitates the rapid generation of therapeutic cell medicines.
Zhang L, Zuris JA, Viswanathan R, Edelstein JN, Turk R, Thommandru B, Rube HT, Glenn SE, Collingwood MA, Bode NM, Beaudoin SF, Lele S, Scott SN, Wasko KM, Sexton S, Borges CM, Schubert MS, Kurgan GL, McNeill MS, Fernandez CA, Myer VE, Morgan RA, Behlke MA, Vakulskas CA. Zhang L, et al. Among authors: behlke ma. Nat Commun. 2021 Jun 23;12(1):3908. doi: 10.1038/s41467-021-24017-8. Nat Commun. 2021. PMID: 34162850 Free PMC article.
Sequence-dependent inhibition of cGAS and TLR9 DNA sensing by 2'-O-methyl gapmer oligonucleotides.
Valentin R, Wong C, Alharbi AS, Pradeloux S, Morros MP, Lennox KA, Ellyard JI, Garcin AJ, Ullah TR, Kusuma GD, Pépin G, Li HM, Pearson JS, Ferrand J, Lim R, Veedu RN, Morand EF, Vinuesa CG, Behlke MA, Gantier MP. Valentin R, et al. Among authors: behlke ma. Nucleic Acids Res. 2021 Jun 21;49(11):6082-6099. doi: 10.1093/nar/gkab451. Nucleic Acids Res. 2021. PMID: 34057477 Free PMC article.
CRISPAltRations: a validated cloud-based approach for interrogation of double-strand break repair mediated by CRISPR genome editing.
Kurgan G, Turk R, Li H, Roberts N, Rettig GR, Jacobi AM, Tso L, Sturgeon M, Mertens M, Noten R, Florus K, Behlke MA, Wang Y, McNeill MS. Kurgan G, et al. Among authors: behlke ma. Mol Ther Methods Clin Dev. 2021 Apr 2;21:478-491. doi: 10.1016/j.omtm.2021.03.024. eCollection 2021 Jun 11. Mol Ther Methods Clin Dev. 2021. PMID: 33981780 Free PMC article.
Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells.
Cromer MK, Camarena J, Martin RM, Lesch BJ, Vakulskas CA, Bode NM, Kurgan G, Collingwood MA, Rettig GR, Behlke MA, Lemgart VT, Zhang Y, Goyal A, Zhao F, Ponce E, Srifa W, Bak RO, Uchida N, Majeti R, Sheehan VA, Tisdale JF, Dever DP, Porteus MH. Cromer MK, et al. Among authors: behlke ma. Nat Med. 2021 Apr;27(4):677-687. doi: 10.1038/s41591-021-01284-y. Epub 2021 Mar 18. Nat Med. 2021. PMID: 33737751 Free PMC article.
147 results