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Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.
George LA, Sullivan SK, Giermasz A, Rasko JEJ, Samelson-Jones BJ, Ducore J, Cuker A, Sullivan LM, Majumdar S, Teitel J, McGuinn CE, Ragni MV, Luk AY, Hui D, Wright JF, Chen Y, Liu Y, Wachtel K, Winters A, Tiefenbacher S, Arruda VR, van der Loo JCM, Zelenaia O, Takefman D, Carr ME, Couto LB, Anguela XM, High KA. George LA, et al. Among authors: sullivan sk, sullivan lm. N Engl J Med. 2017 Dec 7;377(23):2215-2227. doi: 10.1056/NEJMoa1708538. N Engl J Med. 2017. PMID: 29211678 Free PMC article. Clinical Trial.
High-level transgene expression in induced pluripotent stem cell-derived megakaryocytes: correction of Glanzmann thrombasthenia.
Sullivan SK, Mills JA, Koukouritaki SB, Vo KK, Lyde RB, Paluru P, Zhao G, Zhai L, Sullivan LM, Wang Y, Kishore S, Gharaibeh EZ, Lambert MP, Wilcox DA, French DL, Poncz M, Gadue P. Sullivan SK, et al. Among authors: sullivan lm. Blood. 2014 Jan 30;123(5):753-7. doi: 10.1182/blood-2013-10-530725. Epub 2013 Dec 13. Blood. 2014. PMID: 24335497 Free PMC article.
Clonal genetic and hematopoietic heterogeneity among human-induced pluripotent stem cell lines.
Mills JA, Wang K, Paluru P, Ying L, Lu L, Galvão AM, Xu D, Yao Y, Sullivan SK, Sullivan LM, Mac H, Omari A, Jean JC, Shen S, Gower A, Spira A, Mostoslavsky G, Kotton DN, French DL, Weiss MJ, Gadue P. Mills JA, et al. Among authors: sullivan sk, sullivan lm. Blood. 2013 Sep 19;122(12):2047-51. doi: 10.1182/blood-2013-02-484444. Epub 2013 Aug 12. Blood. 2013. PMID: 23940280 Free PMC article.
AAV-mediated gene therapy for choroideremia: preclinical studies in personalized models.
Vasireddy V, Mills JA, Gaddameedi R, Basner-Tschakarjan E, Kohnke M, Black AD, Alexandrov K, Zhou S, Maguire AM, Chung DC, Mac H, Sullivan L, Gadue P, Bennicelli JL, French DL, Bennett J. Vasireddy V, et al. PLoS One. 2013 May 7;8(5):e61396. doi: 10.1371/journal.pone.0061396. Print 2013. PLoS One. 2013. PMID: 23667438 Free PMC article.
255 results