Stable Human FIX Expression After 0.9G Intrauterine Gene Transfer of Self-complementary Adeno-associated Viral Vector 5 and 8 in Macaques

Intrauterine gene transfer (IUGT) offers ontological advantages including immune naiveté mediating tolerance to the vector and transgenic products, and effecting a cure before development of irreversible pathology. Despite proof-of-principle in rodent models, expression efficacy with a therapeutic t...

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Detalles Bibliográficos
Autores principales: Mattar, Citra NZ, Nathwani, Amit C, Waddington, Simon N, Dighe, Niraja, Kaeppel, Christine, Nowrouzi, Ali, Mcintosh, Jenny, Johana, Nuryanti B, Ogden, Bryan, Fisk, Nicholas M, Davidoff, Andrew M, David, Anna, Peebles, Donald, Valentine, Marcus B, Appelt, Jens-Uwe, von Kalle, Christof, Schmidt, Manfred, Biswas, Arijit, Choolani, Mahesh, Chan, Jerry KY
Formato: Artigo
Lenguaje:Inglês
Publicado: Nature Publishing Group 2011
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Original Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3222517/
https://ncbi.nlm.nih.gov/pubmed/21629224
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2011.107
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