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Article

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

9 Nov 1999

Abstract excerpt

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 transfer at day 15 of gestation in a murine model of hemophilia A. Normal, carrier (X(H)X), and FVIII-deficient (X(H)Y...

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PMID: 10557319
DOI: 10.1073/pnas.96.23.13324

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