Recombinant adeno-associated viruses (AAVs) are commonly used gene delivery vehicles
for neuroscience research. They have two engineerable features: the capsid (outer protein …

Background Slowed clearance of amyloid β (Aβ) is believed to underlie the development of
Aβ plaques that characterize Alzheimer's disease (AD). Aβ is cleared in part by the …

Recent work has stimulated interest in the use of exosomes as nanocarriers for delivery of
small drugs, RNAs, and proteins to the central nervous system (CNS). To overcome the …

We recently developed adeno-associated virus (AAV) capsids to facilitate efficient and
noninvasive gene transfer to the central and peripheral nervous systems. However, a …

Neuroinflammation is a key contributor to the pathology of Alzheimer's disease (AD). CD33
(Siglec-3) is a transmembrane sialic acid-binding receptor on the surface of microglial cells …

The liver is the metabolic center of the body and an ideal target for gene therapy of inherited
metabolic disorders (IMDs). Adeno‐associated viral (AAV) vectors can deliver transgenes to …

Adeno-associated virus (AAV) vectors are showing promise in gene therapy trials and have
proven to be extremely efficient biological tools in basic neuroscience research. One major …

Developing therapies for central nervous system (CNS) diseases is exceedingly difficult
because of the blood–brain barrier (BBB). Notably, emerging technologies may provide …

Recently adeno-associated virus (AAV) became the first clinically approved gene therapy
product in the western world. To develop AAV for future clinical application in a widespread …

Hemophilia B (HB) is a life-threatening inherited disease caused by mutations in the FIX
gene, leading to reduced protein function and abnormal blood clotting. Due to its monogenic …