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Lentiviral vectors for precise expression to treat X-linked lymphoproliferative disease.
Ayoub PG, Gensheimer J, Lathrop L, Juett C, Quintos J, Tam K, Reid J, Ma F, Tam C, McAuley GE, Brown D, Wu X, Zhang R, Bradford K, Hollis RP, Crooks GM, Kohn DB. Ayoub PG, et al. Among authors: zhang r. Mol Ther Methods Clin Dev. 2024 Aug 20;32(4):101323. doi: 10.1016/j.omtm.2024.101323. eCollection 2024 Dec 12. Mol Ther Methods Clin Dev. 2024. PMID: 39309261 Free PMC article.
Editing the Sickle Cell Disease Mutation in Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates.
Romero Z, Lomova A, Said S, Miggelbrink A, Kuo CY, Campo-Fernandez B, Hoban MD, Masiuk KE, Clark DN, Long J, Sanchez JM, Velez M, Miyahira E, Zhang R, Brown D, Wang X, Kurmangaliyev YZ, Hollis RP, Kohn DB. Romero Z, et al. Among authors: zhang r. Mol Ther. 2019 Aug 7;27(8):1389-1406. doi: 10.1016/j.ymthe.2019.05.014. Epub 2019 May 24. Mol Ther. 2019. PMID: 31178391 Free PMC article.
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: zhang r. 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.
IND-Enabling Studies for a Clinical Trial to Genetically Program a Persistent Cancer-Targeted Immune System.
Puig-Saus C, Parisi G, Garcia-Diaz A, Krystofinski PE, Sandoval S, Zhang R, Champhekar AS, McCabe J, Cheung-Lau GC, Truong NA, Vega-Crespo A, Komenan MDS, Pang J, Macabali MH, Saco JD, Goodwin JL, Bolon B, Seet CS, Montel-Hagen A, Crooks GM, Hollis RP, Campo-Fernandez B, Bischof D, Cornetta K, Gschweng EH, Adelson C, Nguyen A, Yang L, Witte ON, Baltimore D, Comin-Anduix B, Kohn DB, Wang X, Cabrera P, Kaplan-Lefko PJ, Berent-Maoz B, Ribas A. Puig-Saus C, et al. Among authors: zhang r. Clin Cancer Res. 2019 Feb 1;25(3):1000-1011. doi: 10.1158/1078-0432.CCR-18-0963. Epub 2018 Nov 8. Clin Cancer Res. 2019. PMID: 30409823 Free PMC article.
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