Pharmacology & ToxicologyMolecular TherapyPeer-reviewed
A Precision Gene Engineered B Cell Medicine Producing Sustained Levels of Active Factor IX for Hemophilia B Therapy
Hanlan Liu, Swati Singh, Timothy Mullen…·Richard A. Morgan
This paper describes a gene-engineered B Cell Medicine (BE-101) that produces sustained active factor IX for hemophilia B therapy, showing durable and redosable effects in mice.
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