Bioengineering

Re-engineered coagulation factor will improve hemophilia A gene therapy

Published in Gene Therapy Weekly, July 29th, 2004

Re-engineered coagulation factor will improve hemophilia A gene therapy.

"Factor VIII (FVIII) functions as a cofactor within the intrinsic pathway of blood coagulation. Quantitative or qualitative deficiencies of FVIII result in the inherited bleeding disorder hemophilia A. Expression of FVIII (domain structure A1-A2-B-A3-C1-C2) in heterologous mammalian systems is 2 to 3 orders of magnitude less efficient compared with other proteins of similar size compromising recombinant FVIII production and gene therapy strategies," investigators in the United States report.

"FVIII expression is limited by unstable mRNA, interaction with endoplasmic reticulum...

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Source: Gene Therapy Weekly (2004-07-29)

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