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Short-term correction of factor VIII deficiency in a murine model of hemophilia A after delivery of adenovirus murine factor VIII in utero

Development of in utero gene transfer approaches may provide therapies for genetic disorders with perinatal morbidity. In hemophilia A, prenatal and postnatal bleeding may be catastrophic, and modest increments in factor VIII (FVIII) activity are therapeutic. We performed transuterine i.p. gene tran...

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Huvudupphovsmän: Lipshutz, G. S., Sarkar, R., Flebbe-Rehwaldt, L., Kazazian, H., Gaensler, K. M. L.
Materialtyp: Artigo
Språk:Inglês
Publicerad: The National Academy of Sciences 1999
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Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC23946/
https://ncbi.nlm.nih.gov/pubmed/10557319
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