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Bioengineered Factor IX Molecules with Increased Catalytic Activity Improve the Therapeutic Index of Gene Therapy Vectors for Hemophilia B

Although the desire to develop gene therapy for hemophilia B is high, safety remains a concern. Therefore, improving the therapeutic index of gene therapy vectors is an important goal. Thus, we evaluated the use of three bioengineered factor IX (FIX) variants with improved catalytic activity in the...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Hauptverfasser: Brunetti-Pierri, Nicola, Grove, Nathan C., Zuo, Yu, Edwards, Rachel, Palmer, Donna, Cerullo, Vincenzo, Teruya, Jun, Ng, Philip
Format: Artigo
Sprache:Inglês
Veröffentlicht: Mary Ann Liebert, Inc. 2009
Schlagworte:
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2861950/
https://ncbi.nlm.nih.gov/pubmed/19196177
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2008.084
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