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Comment
. 2018 Jan 3;26(1):14-16.
doi: 10.1016/j.ymthe.2017.12.007. Epub 2017 Dec 21.

Hyperactive Factor IX Padua: A Game-Changer for Hemophilia Gene Therapy

Thierry VandenDriessche  1 , Marinee K Chuah  2
Affiliations
Comment

Hyperactive Factor IX Padua: A Game-Changer for Hemophilia Gene Therapy

Thierry VandenDriessche et al. Mol Ther. .
No abstract available

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Comment on

  • 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.

References

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    1. Lillicrap D., VandenDriessche T., High K. Cellular and genetic therapies for haemophilia. Haemophilia. 2006;12(Suppl 3):36–41. - PubMed
    1. Nathwani A.C., Reiss U.M., Tuddenham E.G., Rosales C., Chowdary P., McIntosh J., Della Peruta M., Lheriteau E., Patel N., Raj D. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N. Engl. J. Med. 2014;371:1994–2004. - PMC - PubMed
    1. George L.A., Sullivan S.K., Giermasz A., Rasko J.E.J., Samelson-Jones B.J., Ducore J., Cuker A., Sullivan L.M., Majumdar S., Teitel J. Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant. N. Engl. J. Med. 2017;377:2215–2227. - PMC - PubMed
    1. Simioni P., Tormene D., Tognin G., Gavasso S., Bulato C., Iacobelli N.P., Finn J.D., Spiezia L., Radu C., Arruda V.R. X-linked thrombophilia with a mutant factor IX (factor IX Padua) N. Engl. J. Med. 2009;361:1671–1675. - PubMed

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