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Role of the vector genome and underlying factor IX mutation in immune responses to AAV gene therapy for hemophilia B

BACKGROUND: Self-complementary adeno-associated virus (scAAV) vectors have become a desirable vector for therapeutic gene transfer due to their ability to produce greater levels of transgene than single-stranded AAV (ssAAV). However, recent reports have suggested that scAAV vectors are more immunoge...

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Detalles Bibliográficos
Autores principales: Rogers, Geoffrey L, Martino, Ashley T, Zolotukhin, Irene, Ertl, Hildegund CJ, Herzog, Roland W
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2014
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3904690/
https://www.ncbi.nlm.nih.gov/pubmed/24460861
http://dx.doi.org/10.1186/1479-5876-12-25