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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: kaufman ml. 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.
Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells.
Hoban MD, Cost GJ, Mendel MC, Romero Z, Kaufman ML, Joglekar AV, Ho M, Lumaquin D, Gray D, Lill GR, Cooper AR, Urbinati F, Senadheera S, Zhu A, Liu PQ, Paschon DE, Zhang L, Rebar EJ, Wilber A, Wang X, Gregory PD, Holmes MC, Reik A, Hollis RP, Kohn DB. Hoban MD, et al. Among authors: kaufman ml. Blood. 2015 Apr 23;125(17):2597-604. doi: 10.1182/blood-2014-12-615948. Epub 2015 Mar 2. Blood. 2015. PMID: 25733580 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: kaufman ml. 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 studies for a phase 1 clinical trial of autologous hematopoietic stem cell gene therapy for sickle cell disease.
Urbinati F, Wherley J, Geiger S, Fernandez BC, Kaufman ML, Cooper A, Romero Z, Marchioni F, Reeves L, Read E, Nowicki B, Grassman E, Viswanathan S, Wang X, Hollis RP, Kohn DB. Urbinati F, et al. Among authors: kaufman ml. Cytotherapy. 2017 Sep;19(9):1096-1112. doi: 10.1016/j.jcyt.2017.06.002. Epub 2017 Jul 18. Cytotherapy. 2017. PMID: 28733131
Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study.
Urbinati F, Campo Fernandez B, Masiuk KE, Poletti V, Hollis RP, Koziol C, Kaufman ML, Brown D, Mavilio F, Kohn DB. Urbinati F, et al. Among authors: kaufman ml. Hum Gene Ther. 2018 Oct;29(10):1153-1166. doi: 10.1089/hum.2018.061. Hum Gene Ther. 2018. PMID: 30198339
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: kaufman ml. 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.
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