Article

Engineered coagulation factor VIII with enhanced secretion and coagulation potential for hemophilia A gene therapy

2024-12-09

Abstract excerpt

The major challenges of gene therapy for hemophilia A using adeno-associated virus (AAV) vectors are reducing vector doses and the long-term maintenance of stable factor VIII (FVIII). Here, we developed engineered human B-domain-deleted FVIIIs (FVIIISQs) with enhanced secretion and coagulation potential. Intracellular accumulation was markedly reduced in some engineered FVIIISQs, resulting in reduced unfolded prot...

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Literature Corpus work: 1dc1d6e2-91ac-5e1c-9819-57ebf8639a7c
DOI: 10.1101/2024.12.05.626963

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