In recent years, adeno-associated virus (AAV) has become the most important vector for
central nervous system (CNS) gene therapy. AAV has already shown promising results in …

Pompe disease is an inherited neuromuscular disorder caused by deficiency of the
lysosomal enzyme acid alpha-glucosidase (GAA). The most severe form is infantile-onset …

Pompe disease is caused by deficiency of acid α-glucosidase (GAA), resulting in glycogen
accumulation in various tissues, including cardiac and skeletal muscles and the central …

This experiment was conducted to investigate the impacts of dietary selenium yeast (SeY)
on the growth performance, fish body composition, metabolic ability, antioxidant capability …

Pompe disease is a rare genetic disorder caused by a deficiency of the enzyme acid alpha-
glucosidase (GAA). This enzyme is responsible for breaking down glycogen, leading to the …

Pompe disease (PD) is a neuromuscular disorder caused by acid α‐glucosidase (GAA)
deficiency. Reduced GAA activity leads to pathological glycogen accumulation in cardiac …

Advances in adeno-associated virus (AAV)-based gene therapy are transforming our ability
to treat rare genetic disorders and address other unmet medical needs. However, the natural …

The highly prevalent metabolic dysfunction-associated steatohepatitis (MASH) is associated
with liver steatosis, inflammation, and hepatocyte injury, which can lead to fibrosis and may …

Objective Pompe disease (PD) is caused by deficiency of the lysosomal enzyme acid α-
glucosidase (GAA), leading to progressive glycogen accumulation and severe myopathy …

Years of research into the structure, processing, and function of acid alpha-glucosidase led
to the development and 2006 approval of alglucosidase alfa (recombinant human acid …