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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. 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.
Obstacles and future of gene therapy for hemophilia.
Arruda VR, Samelson-Jones BJ. Arruda VR, et al. Expert Opin Orphan Drugs. 2015;3(9):997-1010. doi: 10.1517/21678707.2015.1069179. Epub 2015 Jul 18. Expert Opin Orphan Drugs. 2015. PMID: 26900534 Free PMC article.
Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy.
Samelson-Jones BJ, Arruda VR. Samelson-Jones BJ, et al. Mol Ther Methods Clin Dev. 2018 Dec 31;12:184-201. doi: 10.1016/j.omtm.2018.12.007. eCollection 2019 Mar 15. Mol Ther Methods Clin Dev. 2018. PMID: 30705923 Free PMC article. Review.
37 results