Viral vectors have been used for a broad spectrum of gene therapy for both acute and
chronic diseases. In the context of cancer gene therapy, viral vectors expressing anti-tumor …

Phenylketonuria (PKU) or hyperphenylalaninemia is considered a paradigm for an inherited
(metabolic) liver defect and is, based on murine models that replicate all human pathology …

Adeno-associated virus gene therapy has been the subject of intensive investigation for
monogenic disease gene addition therapy for more than 25 years, yet few therapies have …

The introduction of adeno-associated virus–mediated, liver-directed gene therapy into the
hemophilia treatment landscape brings not only great promise but also considerable …

Adeno-associated virus (AAV) vectors are used for correcting multiple genetic disorders.
Although the goal is to achieve lifelong correction with a single vector administration, the …

Gene therapy aims to add, replace or turn off genes to help treat disease. To date, the US
Food and Drug Administration (FDA) has approved 14 gene therapy products. With the …

Remarkably, it has been 40 years since the isolation of the two genes involved in
hemophilia A (HA) and hemophilia B (HB), encoding clotting factors VIII (FVIII) and IX (FIX) …

Primary liver cancer, which is scientifically referred to as hepatocellular carcinoma (HCC), is
a significant concern in the field of global health. It has been demonstrated that conventional …

De novo immune responses are considered major challenges in gene therapy. With the aim
to lower innate immune responses directly in cells targeted by adeno-associated virus (AAV) …

Congenital hemophilia A and B are caused by mutations in the genes encoding coagulation
factor (F) VIII and IX, respectively, resulting in lifelong bleeding disorders. After decades of …