Gene Therapy
Reports from University College London, Institute of Child Health Highlight Recent Research in Gene Therapy
Published in Gene Therapy Weekly, February 24th, 2011
2011 FEB 24 -- Researchers detail in 'Codon optimization of human factor VIII cDNAs leads to high-level expression,' new data in Gene Therapy. According to recent research from London, the United Kingdom, "Gene therapy for hemophilia A would be facilitated by development of smaller expression cassettes encoding factor VIII (FVIII), which demonstrate improved biosynthesis and/or enhanced biologic properties. B domain deleted (BDD) FVIII retains full procoagulant function and is expressed at higher levels than wild-type FVIII."
"However, a partial BDD FVIII, leaving an N-terminal 226 amino acid stretch (N6), increases in vitro secretion of FVIII tenfold...
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Source: Gene Therapy Weekly (2011-02-24)
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