Comparison of Factor VIII Transgenes Bioengineered for Improved Expression in Gene Therapy of Hemophilia A

Successful gene therapy of hemophilia A depends on the sustained expression of therapeutic levels of factor VIII (fVIII). Because of mRNA instability, interactions with resident endoplasmic reticulum (ER) chaperones, and the requirement for carbohydrate-facilitated transport from the ER to the Golgi...

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Bibliografische gegevens
Hoofdauteurs: Dooriss, Kerry L., Denning, Gabriela, Gangadharan, Bagirath, Javazon, Elisabeth H., McCarty, David A., Spencer, H. Trent, Doering, Christopher B.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Mary Ann Liebert, Inc. 2009
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Scientific Articles
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2828624/
https://ncbi.nlm.nih.gov/pubmed/19222367
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2008.150
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