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Minimal Essential Human Factor VIII Alterations Enhance Secretion and Gene Therapy Efficiency

23 Oct 2020

Abstract excerpt

One important limitation for achieving therapeutic expression of human factor VIII (FVIII) in hemophilia A gene therapy is inefficient secretion of the FVIII protein. Substitution of five amino acids in the A1 domain of human FVIII with the corresponding porcine FVIII residues generated a secretion-enhanced human FVIII variant termed B-domain-deleted (BDD)-FVIII-X5 that resulted in 8-fold higher FVIII activity...

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PMID: 33313336
DOI: 10.1016/j.omtm.2020.10.013

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