Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

BACKGROUND: Hemophilia B, an X-linked disorder, is ideally suited for gene therapy. We investigated the use of a new gene therapy in patients with the disorder. METHODS: We infused a single dose of a serotype-8–pseudotyped, self-complementary adenovirus-associated virus (AAV) vector expressing a cod...

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Autores principales: Nathwani, Amit C., Tuddenham, Edward G.D., Rangarajan, Savita, Rosales, Cecilia, McIntosh, Jenny, Linch, David C., Chowdary, Pratima, Riddell, Anne, Pie, Arnulfo Jaquilmac, Harrington, Chris, O’Beirne, James, Smith, Keith, Pasi, John, Glader, Bertil, Rustagi, Pradip, Ng, Catherine Y.C., Kay, Mark A., Zhou, Junfang, Spence, Yunyu, Morton, Christopher L., Allay, James, Coleman, John, Sleep, Susan, Cunningham, John M., Srivastava, Deokumar, Basner-Tschakarjan, Etiena, Mingozzi, Federico, High, Katherine A., Gray, John T., Reiss, Ulrike M., Nienhuis, Arthur W., Davidoff, Andrew M.
Formato: Artigo
Lenguaje:Inglês
Publicado: 2011
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Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3265081/
https://ncbi.nlm.nih.gov/pubmed/22149959
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1056/NEJMoa1108046
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