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Article

Expression of Human Factor VIII by Splicing between Dimerized AAV Vectors

1 Jun 2002

Abstract excerpt

Adeno-associated virus (AAV) is a useful vector for hemophilia gene therapy, but the limited effective packaging capacity of AAV (5 kb) appears to be incompatible with factor VIII (gene symbol F8) cDNA (7 kb). Although we previously demonstrated efficient packaging and expression of B-domain-deleted human F8 (BDD-F8) using a single AAV vector, the packaging limit still excludes the use of large/strong regulatory...

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Identifiers

PMID: 12027555
DOI: 10.1006/mthe.2002.0607

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