Lysosomal storage diseases (LSDs) are a group of over 70 diseases that are characterized
by lysosomal dysfunction, most of which are inherited as autosomal recessive traits. These …

Abstract Lysosomal Storage Disorders (LSDs) are a diverse group of inherited, monogenic
diseases caused by functional defects in specific lysosomal proteins. The lysosome is a …

Sanfilippo syndrome or mucopolysaccharidosis III is a lysosomal storage disorder caused by
mutations in genes responsible for the degradation of heparan sulfate, a glycosaminoglycan …

Delivery of adeno-associated viral vectors (AAVs) to cerebrospinal fluid (CSF) has emerged
as a promising approach to achieve widespread transduction of the central nervous system …

Sanfilippo disease is one of mucopolysaccharidoses (MPS), a group of lysosomal storage
diseases characterized by accumulation of partially degraded glycosaminoglycans (GAGs) …

The development of gene therapies for central nervous system disorders is challenging
because it is difficult to translate preclinical data from current in vitro and in vivo models to …

Abstract Mucopolysaccharidoses III (MPS III, Sanfilippo syndrome) is a subtype of the
Mucopolysaccharidoses (MPS), a group of inherited lysosomal disorders caused by a …

The invention provides compositions and methods for the preparation, manufacture and
therapeutic use of viral vectors, such as adeno-associated virus (AAV) particles having viral …

Parvoviridae family viruses are small non-enveloped icosahedral capsid viruses
characterized by a single stranded DNA genome. Parvoviridae family viruses consist of two …

Abstract Mucopolysaccharidosis type IIIB (MPS IIIB) is an autosomal recessive lysosomal
storage disease caused by mutations in the gene that encodes the protein N-acetyl …