Introduction: Hemophilia A (HA) is the most common genetic bleeding disorder caused by
mutations in the F8 gene encoding coagulation factor VIII (FVIII). As the second predominant …

Hemophilia A is an X-linked genetic disorder caused by mutations in the F8 gene, which
encodes the blood coagulation factor VIII. Almost half of all severe hemophilia A cases result …

Given that the cDNA of F8 is too large to be packaged into adeno-associated virus (AAV)
capsids, gene transfer of some versions of B-domain-deleted F8 (BDD-F8) for hemophilia A …

Nearly half of severe Hemophilia A (HA) cases are caused by F8 intron 22 inversion (Inv22).
This 0.6-Mb inversion splits the 186-kb F8 into two parts with opposite transcription …

The importance of genetic diagnosis for patients with hemophilia has been recently
demonstrated. However, the pathological variant cannot be identified in some patients …

Hemophilia A (HA), a common bleeding disorder caused by a deficiency of coagulation
factor VIII (FVIII), has long been considered an attractive target for gene therapy studies …

Background Hemophilia A is caused by a deficiency of coagulation factor VIII in the body
due to a defect in the F8 gene. The emergence of CRISPR/Cas9 gene editing technology …

Target-specific genome editing, using engineered nucleases zinc finger nuclease (ZFN),
transcription activator-like effector nuclease (TALEN), and type II clustered regularly …

Abstract Background Factor V (FV) deficiency is a monogenic inherited coagulation disorder
considered to be an ideal indication for gene therapy. To investigate the possibility of …

Hemophilia A (HA) is caused by genetic mutations in the blood coagulation factor VIII (FVIII)
gene. Genome-editing approaches can be used to target the mutated site itself in patient …