Gene therapy for hemophilia B using CB 2679d-GT: a novel factor IX variant with higher potency than factor IX Padua

Sustained expression of therapeutic factor IX (FIX) levels has been achieved after adeno-associated viral (AAV) vector-based gene therapy in patients with hemophilia B. Nevertheless, patients are still at risk of vector dose-limiting toxicity, particularly liver inflammation, justifying the need for...

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Bibliografiska uppgifter
I publikationen:Blood
Huvudupphovsmän: Nair, Nisha, De Wolf, Dries, Nguyen, Phuong Anh, Pham, Quang Hong, Samara-Kuko, Ermira, Landau, Jeff, Blouse, Grant E., Chuah, Marinee K., VandenDriessche, Thierry
Materialtyp: Artigo
Språk:Inglês
Publicerad: American Society of Hematology 2021
Ämnen:
Gene Therapy
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC8191862/
https://ncbi.nlm.nih.gov/pubmed/33735915
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood.2020006005
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