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Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models.
Uchida N, Li L, Nassehi T, Drysdale CM, Yapundich M, Gamer J, Haro-Mora JJ, Demirci S, Leonard A, Bonifacino AC, Krouse AE, Linde NS, Allen C, Peshwa MV, De Ravin SS, Donahue RE, Malech HL, Tisdale JF. Uchida N, et al. Among authors: bonifacino ac. Cell Rep Med. 2021 Apr 20;2(4):100247. doi: 10.1016/j.xcrm.2021.100247. eCollection 2021 Apr 20. Cell Rep Med. 2021. PMID: 33948577 Free PMC article.
Transient in vivo selection of transduced peripheral blood cells using antifolate drug selection in rhesus macaques that received transplants with hematopoietic stem cells expressing dihydrofolate reductase vectors.
Persons DA, Allay JA, Bonifacino A, Lu T, Agricola B, Metzger ME, Donahue RE, Dunbar CE, Sorrentino BP. Persons DA, et al. Blood. 2004 Feb 1;103(3):796-803. doi: 10.1182/blood-2003-05-1572. Epub 2003 Aug 14. Blood. 2004. PMID: 12920024 Free article.
Long-term clinical and molecular follow-up of large animals receiving retrovirally transduced stem and progenitor cells: no progression to clonal hematopoiesis or leukemia.
Kiem HP, Sellers S, Thomasson B, Morris JC, Tisdale JF, Horn PA, Hematti P, Adler R, Kuramoto K, Calmels B, Bonifacino A, Hu J, von Kalle C, Schmidt M, Sorrentino B, Nienhuis A, Blau CA, Andrews RG, Donahue RE, Dunbar CE. Kiem HP, et al. Mol Ther. 2004 Mar;9(3):389-95. doi: 10.1016/j.ymthe.2003.12.006. Mol Ther. 2004. PMID: 15006605 Free article.
52 results