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 …

Immunogenicity has imposed a challenge to efficacy and safety evaluation of adeno-
associated virus (AAV) vector-based gene therapies. Mild to severe adverse events …

Mucopolysaccharidoses (MPS) are a group of lysosomal storage diseases caused by
defects in genes coding for proteins involved in degradation of glycosaminoglycans (GAGs) …

Recombinant adeno‐associated virus (AAV) is currently the most widely used platform for in
vivo gene therapy. Clinical pharmacology is a central field for AAV gene therapy …

Mucopolysaccharidoses (MPS) are a group of rare, heterogeneous, lysosomal storage
disorders. Patients show a broad spectrum of clinical features with a substantial unmet …

Background Due to the broad application of next-generation sequencing, the molecular
diagnosis of genetic disorders in pediatric neurology is no longer an unachievable goal …

Based on studies in experimental animals demonstrating that administration of adeno-
associated virus (AAV) vectors to the cerebrospinal fluid (CSF) is an effective route to …

CLN2 disease is a fatal, childhood autosomal recessive disorder caused by mutations in
ceroid lipofuscinosis type 2 (CLN2) gene, encoding tripeptidyl peptidase 1 (TPP-1). Loss of …

Mucopolysaccharidoses (MPSs) are complex lysosomal storage disorders that result in the
accumulation of glycosaminoglycans (GAGs) in urine, blood, and tissues. Lysosomal …