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...

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Autori principali: Brunetti-Pierri, Nicola, Grove, Nathan C., Zuo, Yu, Edwards, Rachel, Palmer, Donna, Cerullo, Vincenzo, Teruya, Jun, Ng, Philip
Natura: Artigo
Lingua:Inglês
Pubblicazione: Mary Ann Liebert, Inc. 2009
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Scientific Articles
Accesso online: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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