Year: 2006
Type: article
Abstract: Protein replacement therapy for hemophilia A is very costly and has a potential side effect of developing inhibitors. Gene therapy, on the other hand, can potentially prevent these limitations of current treatments. Although recombinant adeno_associated virus (rAAV) vectors are promising for delivering factor VIII gene, applying AAV vector technology to Hemophilia A lagged behind other genetic diseases because of this size constraint (limited to ∼5kb).
Source: Molecular Therapy
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $4,500
OpenAlex ID: W2054470202