Lecture9DownSyndrome_000

Commercially produced factor concentrates such as

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Unformatted text preview: t 65 years today. Commercially produced factor concentrates such as "Advate", a recombinant Factor VIII produced by Baxter International, come as a white powder in a vial which must be mixed with sterile water prior to intravenous injection. Wikipedia Gene therapy for Hemophilia B The coding sequence for Factor IX was inserted into Adeno-associated viral vector (AAV). AAV’s are non-pathogenic human viruses, ssDNA genome of 4.7 kb AAV8 strain infects specifically liver cells. Factor IX was placed under liver-specific promoter 6 adult males with Hemophilia B were treated Reported levels of Factor IX 1-6% of the...
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This note was uploaded on 01/07/2013 for the course BIMM 100 taught by Professor Pasquinelli during the Spring '06 term at UCSD.

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