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In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs.

Hemophilia B is a bleeding disorder caused by mutations in the factor IX gene. The disorder is X-linked recessive with a prevalence of about 1 in 30,000 Caucasian males. Factor IX is naturally synthesized in the liver and secreted into blood. Here we report the construction of recombinant adenoviral...

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Publicado no:Proc Natl Acad Sci U S A
Principais autores: Kay, M A, Landen, C N, Rothenberg, S R, Taylor, L A, Leland, F, Wiehle, S, Fang, B, Bellinger, D, Finegold, M, Thompson, A R
Formato: Artigo
Idioma:Inglês
Publicado em: National Academy of Sciences 1994
Assuntos:
Research Article
Acesso em linha:https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC43369/
https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/8134398/
https://ncbi.nlm.nih.govhttps://doi.org/10.1073/pnas.91.6.2353
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