<p id="p001">Leber's congenital amaurosis (LCA) is a group of inherited blinding diseases
with onset during childhood. One form of the disease, LCA2, is caused by mutations in the …

Background Gene therapy has the potential to reverse disease or prevent further deterioration
of vision in patients with incurable inherited retinal degeneration. We therefore did a …

Background The prevention of bleeding with adequately sustained levels of clotting factor,
after a single therapeutic intervention and without the need for further medical intervention, …

Sodium-dependent transport into astrocytes is critical for maintaining the extracellular
concentrations of glutamate below toxic levels in the central nervous system. In this study, the …

We evaluated the safety and efficacy of an optimized adeno-associated virus (AAV; AAV2.RPE65)
in animal models of the RPE65 form of Leber congenital amaurosis (LCA). Protein …

The glial glutamate transporter GLT-1 may be the predominant Na + -dependent glutamate
transporter in forebrain. Expression of GLT-1 correlates with astrocyte maturation in vivo and …

A recombinant adeno-associated virus serotype 2 Reference Standard Material (rAAV2 RSM)
has been produced and characterized with the purpose of providing a reference standard …

In the nervous system, astrocytes express different ratios of the two glial glutamate
transporters, glutamate transporter subtype 1 (GLT‐1) and glutamate/aspartate transporter (GLAST), …

In a gene therapy clinical trial for hemophilia B, adeno-associated virus 2 (AAV2) capsid–specific
CD8 + T cells were previously implicated in the elimination of vector-transduced …

The hepatitis C virus (HCV) chronically infects 2% of the world population and effective
treatment is limited by long duration and significant side-effects. Here, we describe a novel drug, …