First Hemophilia B Gene Therapy Approved: More Than Two Decades in the Making

Overview

Journal Mol Ther

Publisher Cell Press

Specialties Molecular Biology
Pharmacology

Date 2022 Dec 17

PMID 36528029

Authors

Roland W Herzog

Thierry VandenDriessche

Margareth C Ozelo

Affiliations

Soon will be listed here.

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References

High K, Anguela X . Adeno-associated viral vectors for the treatment of hemophilia. Hum Mol Genet. 2015; 25(R1):R36-41. PMC: 4802375. DOI: 10.1093/hmg/ddv475. View

Samelson-Jones B, George L . Adeno-Associated Virus Gene Therapy for Hemophilia. Annu Rev Med. 2022; 74:231-247. PMC: 9892335. DOI: 10.1146/annurev-med-043021-033013. View

VandenDriessche T, Pipe S, Pierce G, Kaczmarek R . First conditional marketing authorization approval in the European Union for hemophilia "A" gene therapy. Mol Ther. 2022; 30(11):3335-3336. PMC: 9637642. DOI: 10.1016/j.ymthe.2022.09.020. View

Simioni P, Tormene D, Tognin G, Gavasso S, Bulato C, Iacobelli N . X-linked thrombophilia with a mutant factor IX (factor IX Padua). N Engl J Med. 2009; 361(17):1671-5. DOI: 10.1056/NEJMoa0904377. View

Samelson-Jones B . Worldwide use of factor IX Padua for hemophilia B gene therapy. Mol Ther. 2022; 30(7):2394-2396. PMC: 9263299. DOI: 10.1016/j.ymthe.2022.06.002. View