Gene therapy is an emerging and powerful therapeutic tool to deliver functional genetic
material to cells in order to correct a defective gene. During the past decades, several …

Choroid plexus (ChP) epithelial cells are crucial for the function of the blood-cerebrospinal
fluid barrier (BCSFB) in the developing and mature brain. The ChP is considered the primary …

Objective Dominant loss‐of‐function mutations in the gene encoding the lysosomal protein,
progranulin, cause 5‐10% of frontotemporal dementia cases. As progranulin undergoes …

Menkes disease is a lethal infantile neurodegenerative disorder of copper metabolism
caused by mutations in a P-type ATPase, ATP7A. Currently available treatment (daily …

Sixteen years after Graham and coworkers described the most used system for generating
helper-dependent adenovirus (HDAd) vectors, production systems have evolved …

Enzyme replacement via the cerebrospinal fluid (CSF) has been shown to ameliorate
neurological symptoms in model animals with neuropathic metabolic disorders. Gene …

Ependymal cells are epithelial support cells that line the central canal and ventricular
cavities of the central nervous system, providing the interface between the cerebrospinal …

The development, function, and pathology of the brain depend on interactions of neurons
and different types of glial cells, namely astrocytes, oligodendrocytes, microglia, and …

Ependymal cells are arranged along the inner surfaces of the ventricles and the central
canal of the spinal cord, providing anatomical, physiological and immunological barriers that …

Canine adenovirus type 2 vectors (CAV-2) are promising tools to treat global central nervous
system (CNS) disorders because of their preferential transduction of neurons and efficient …