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Editorial
. 2025 Jan 8;33(1):1-2.
doi: 10.1016/j.ymthe.2024.11.026. Epub 2024 Nov 29.

Second gene therapy for hemophilia B approved: More answers or questions?

Radoslaw Kaczmarek  1 , Glenn F Pierce  2
Affiliations
Editorial

Second gene therapy for hemophilia B approved: More answers or questions?

Radoslaw Kaczmarek et al. Mol Ther. .
No abstract available

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References

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    1. Pipe S.W., Leebeek F.W.G., Recht M., Key N.S., Castaman G., Miesbach W., Lattimore S., Peerlinck K., Van der Valk P., Coppens M., et al. Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B. N. Engl. J. Med. 2023;388:706–718. doi: 10.1056/NEJMoa2113708. - DOI - PubMed
    1. Cuker A., Kavakli K., Frenzel L., Wang J.-D., Astermark J., Cerqueira M.H., Iorio A., Katsarou-Fasouli O., Klamroth R., Shapiro A.D., et al. Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B. N. Engl. J. Med. 2024;391:1108–1118. doi: 10.1056/NEJMoa2302982. - DOI - PubMed
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    1. Pierce G.F., Fong S., Long B.R., Kaczmarek R. Deciphering conundrums of AAV liver-directed gene therapy: focus on hemophilia. J. Thromb. Haemost. 2024;22:1263–1289. doi: 10.1016/J.JTHA.2023.12.005. - DOI - PubMed

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