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2024 3

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Page 1
Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease.
Lattanzi A, Camarena J, Lahiri P, Segal H, Srifa W, Vakulskas CA, Frock RL, Kenrick J, Lee C, Talbott N, Skowronski J, Cromer MK, Charlesworth CT, Bak RO, Mantri S, Bao G, DiGiusto D, Tisdale J, Wright JF, Bhatia N, Roncarolo MG, Dever DP, Porteus MH. Lattanzi A, et al. Among authors: charlesworth ct. Sci Transl Med. 2021 Jun 16;13(598):eabf2444. doi: 10.1126/scitranslmed.abf2444. Sci Transl Med. 2021. PMID: 34135108 Free PMC article.
Immunological barriers to haematopoietic stem cell gene therapy.
Charlesworth CT, Hsu I, Wilkinson AC, Nakauchi H. Charlesworth CT, et al. Nat Rev Immunol. 2022 Dec;22(12):719-733. doi: 10.1038/s41577-022-00698-0. Epub 2022 Mar 17. Nat Rev Immunol. 2022. PMID: 35301483 Free PMC article. Review.
Genome engineering with Cas9 and AAV repair templates generates frequent concatemeric insertions of viral vectors.
Suchy FP, Karigane D, Nakauchi Y, Higuchi M, Zhang J, Pekrun K, Hsu I, Fan AC, Nishimura T, Charlesworth CT, Bhadury J, Nishimura T, Wilkinson AC, Kay MA, Majeti R, Nakauchi H. Suchy FP, et al. Among authors: charlesworth ct. Nat Biotechnol. 2024 Apr 8. doi: 10.1038/s41587-024-02171-w. Online ahead of print. Nat Biotechnol. 2024. PMID: 38589662
Investigation of Cas9 antibodies in the human eye.
Toral MA, Charlesworth CT, Ng B, Chemudupati T, Homma S, Nakauchi H, Bassuk AG, Porteus MH, Mahajan VB. Toral MA, et al. Among authors: charlesworth ct. Nat Commun. 2022 Feb 25;13(1):1053. doi: 10.1038/s41467-022-28674-1. Nat Commun. 2022. PMID: 35217666 Free PMC article.
Author Correction: Investigation of Cas9 antibodies in the human eye.
Toral MA, Charlesworth CT, Ng B, Chemudupati T, Homma S, Nakauchi H, Bassuk AG, Porteus MH, Mahajan VB. Toral MA, et al. Among authors: charlesworth ct. Nat Commun. 2022 Apr 12;13(1):2109. doi: 10.1038/s41467-022-29844-x. Nat Commun. 2022. PMID: 35414059 Free PMC article. No abstract available.
Identification of preexisting adaptive immunity to Cas9 proteins in humans.
Charlesworth CT, Deshpande PS, Dever DP, Camarena J, Lemgart VT, Cromer MK, Vakulskas CA, Collingwood MA, Zhang L, Bode NM, Behlke MA, Dejene B, Cieniewicz B, Romano R, Lesch BJ, Gomez-Ospina N, Mantri S, Pavel-Dinu M, Weinberg KI, Porteus MH. Charlesworth CT, et al. Nat Med. 2019 Feb;25(2):249-254. doi: 10.1038/s41591-018-0326-x. Epub 2019 Jan 28. Nat Med. 2019. PMID: 30692695 Free PMC article.
12 results