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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Detalhes bibliográficos
Main Authors: 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
Idioma:Inglês
Publicado em: 2011
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Article
Acesso em linha: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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