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 …

Since the discovery of the lysosome in 1955, advances have been made in understanding
the key roles and functions of this organelle. The concept of lysosomal storage diseases …

Background Mucopolysaccharidosis type IIIB syndrome (also known as Sanfilippo type B
syndrome) is a lysosomal storage disease resulting in progressive deterioration of cognitive …

Mucopolysaccharidosis type IIIA is a severe degenerative disease caused by an autosomal
recessive defect of a gene encoding a lysosomal heparan-N-sulfamidase, the N …

Convection-enhanced delivery (CED) is a bulk flow–driven process. Its properties permit
direct, homogeneous, targeted perfusion of CNS regions with putative therapeutics while …

Late infantile neuronal ceroid lipofuscinosis (LINCL) is an autosomal recessive,
neurodegenerative lysosomal storage disease affecting the CNS and is fatal by age 8 to 12 …

Recombinant adeno-associated virus (rAAV) vectors possess a number of properties that
may make them suitable for clinical gene therapy, including being based upon a virus for …

Lysosomal storage diseases (LSDs) comprise a diverse group of monogenetic disorders
with complex clinical phenotypes that include both systemic and central nervous system …

Nerve growth factor (NGF) gene therapy rescues and stimulates cholinergic neurons, which
degenerate in Alzheimer's disease (AD). In a recent clinical trial for AD, intraparenchymal …

Background Canavan disease is a rare leukodystrophy with no current treatment. rAAV‐
ASPA has been developed for gene delivery to the central nervous system (CNS) for …