Gene transfer using adeno-associated viral (AAV) vectors has made tremendous progress in
the last decade and has achieved cures of debilitating diseases such as hemophilia A and …

The complement system is crucial for immune surveillance, providing the body's first line of
defence against pathogens. However, an imbalance in its regulators can lead to …

BACKGROUND Systemic administration of adeno-associated virus (AAV) can trigger life-
threatening inflammatory responses, including thrombotic microangiopathy (TMA), acute …

The complement alternative pathway (AP) is implicated in numerous diseases affecting
many organs, ranging from the rare hematological disease paroxysmal nocturnal …

The complement system is involved in the origin of autoimmunity and systemic lupus
erythematosus. Both genetic deficiency of complement components and excessive …

Aim: To evaluate the regulatory landscape underlying the active enhancer marked by
H3K27ac in high-fat diet (HFD)-induced nonalcoholic fatty liver disease (NAFLD) in rats …

C3 glomerulopathy (C3G) is associated with dysregulation of the alternative pathway (AP) of
complement and treatment options remain inadequate. Factor H (FH) is a potent regulator of …

Gene therapy using adeno-associated virus (AAV)-based vectors has become a realistic
therapeutic option for hemophilia. We examined the potential of a novel engineered liver …

Adeno-associated virus (AAV) vectors are proving to be clinically transformative tools in the
treatment of monogenic genetic disease. Rapid ongoing development of this technology …

Recombinant adeno-associated viruses (AAV) are amongst the most promising in vivo
delivery tools for genomic medicines and have opened the door to potentially …