The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy

Studies on gene therapy for hemophilia B (HB) using adeno-associated viral (AAV) vectors showed that the safety of a given strategy is directly related to the vector dose. To overcome this limitation, we sought to test the efficacy and the risk of immunogenicity of a novel factor IX (FIX) R338L asso...

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Bibliografiset tiedot
Päätekijät: Finn, Jonathan D., Nichols, Timothy C., Svoronos, Nikolaos, Merricks, Elizabeth P., Bellenger, Dwight A., Zhou, Shangshen, Simioni, Paolo, High, Katherine A., Arruda, Valder R.
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: American Society of Hematology 2012
Aiheet:
Gene Therapy
Linkit:https://ncbi.nlm.nih.gov/pmc/articles/PMC3512231/
https://ncbi.nlm.nih.gov/pubmed/22919027
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2012-06-440123
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