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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Autors principals: 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
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group 2011
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Original Article
Accés en línia: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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