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Dosing and Re-Administration of Lentiviral Vector for In Vivo Gene Therapy in Rhesus Monkeys and ADA-Deficient Mice.
Carbonaro-Sarracino DA, Tarantal AF, Lee CCI, Kaufman ML, Wandro S, Jin X, Martinez M, Clark DN, Chun K, Koziol C, Hardee CL, Wang X, Kohn DB. Carbonaro-Sarracino DA, et al. Among authors: wang x. Mol Ther Methods Clin Dev. 2019 Nov 16;16:78-93. doi: 10.1016/j.omtm.2019.11.004. eCollection 2020 Mar 13. Mol Ther Methods Clin Dev. 2019. PMID: 31871959 Free PMC article.
Allelic exclusion and peripheral reconstitution by TCR transgenic T cells arising from transduced human hematopoietic stem/progenitor cells.
Giannoni F, Hardee CL, Wherley J, Gschweng E, Senadheera S, Kaufman ML, Chan R, Bahner I, Gersuk V, Wang X, Gjertson D, Baltimore D, Witte ON, Economou JS, Ribas A, Kohn DB. Giannoni F, et al. Among authors: wang x. Mol Ther. 2013 May;21(5):1044-54. doi: 10.1038/mt.2013.8. Epub 2013 Feb 5. Mol Ther. 2013. PMID: 23380815 Free PMC article.
Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency.
Carbonaro DA, Zhang L, Jin X, Montiel-Equihua C, Geiger S, Carmo M, Cooper A, Fairbanks L, Kaufman ML, Sebire NJ, Hollis RP, Blundell MP, Senadheera S, Fu PY, Sahaghian A, Chan RY, Wang X, Cornetta K, Thrasher AJ, Kohn DB, Gaspar HB. Carbonaro DA, et al. Among authors: wang x. Mol Ther. 2014 Mar;22(3):607-622. doi: 10.1038/mt.2013.265. Epub 2013 Nov 20. Mol Ther. 2014. PMID: 24256635 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: wang x. 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.
Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency.
Reinhardt B, Habib O, Shaw KL, Garabedian E, Carbonaro-Sarracino DA, Terrazas D, Fernandez BC, De Oliveira S, Moore TB, Ikeda AK, Engel BC, Podsakoff GM, Hollis RP, Fernandes A, Jackson C, Shupien S, Mishra S, Davila A, Mottahedeh J, Vitomirov A, Meng W, Rosenfeld AM, Roche AM, Hokama P, Reddy S, Everett J, Wang X, Luning Prak ET, Cornetta K, Hershfield MS, Sokolic R, De Ravin SS, Malech HL, Bushman FD, Candotti F, Kohn DB. Reinhardt B, et al. Among authors: wang x. Blood. 2021 Oct 14;138(15):1304-1316. doi: 10.1182/blood.2020010260. Blood. 2021. PMID: 33974038 Free PMC article. Clinical Trial.
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