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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Main Authors: 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
格式: Artigo
語言:Inglês
出版: Nature Publishing Group 2011
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Original Article
在線閱讀: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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