Functional factor VIII made with von Willebrand factor at high levels in transgenic milk.

ABSTRACT:

Background

Current manufacturing methods for recombinant human factor VIII (rFVIII) within mammalian cell cultures are inefficient, hampering the production of sufficient amounts for affordable, worldwide treatment of hemophilia A. However, rFVIII has been expressed at very high levels by the transgenic mammary glands of mice, rabbits, sheep, and pigs. Unfortunately, it is secreted into milk with low specific activity, owing in part to the labile, heterodimeric structure that results from furin processing of its B domain.

Objectives

To express biologically active rFVIII in the milk of transgenic mice through targeted bioengineering.

Methods

Transgenic mice were made with a mammary-specific FVIII gene (226/N6) bioengineered for efficient expression and stability, encod

SUBMITTER: Pipe SW 

PROVIDER: S-EPMC3444248 | biostudies-literature | 2011 Nov

REPOSITORIES: biostudies-literature