Promise of gene therapy for congenital neurologic disease due to GPI deficiency

A recent study by Murakami et al. describes a novel genetic treatment for inherited glycophosphatidylinositol (GPI) deficiency (IGD) using adeno-associated virus (AAV)-based
gene therapy. PIGA is an X-linked gene required for the first step of GPI anchor biosynthesis, a multistep process requiring at least 27 different genes. Disruption of PIGA or other genes in the GPI biosynthesis process leads to the loss of approximately 150 different GPI-anchored surface proteins (GPI-APs) in humans. […]