Cell-type-specific expression of molecular tools and sensors is critical to construct circuit
diagrams and to investigate the activity and function of neurons within the nervous system …

Gene therapy is at the cusp of a revolution for treating a large spectrum of CNS disorders by
providing a durable therapeutic protein via a single administration. Adeno-associated virus …

Several attempts have been made to discover the ideal vector for gene therapy in central
nervous system (CNS). Adeno-associated viruses (AAVs) are currently the preferred vehicle …

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 …

Background Neurodevelopmental disorders, as a class of diseases, have been particularly
difficult to treat even when the underlying cause (s), such as genetic alterations, are …

Neurological disorders–disorders of the brain, spine and associated nerves–are a leading
contributor to global disease burden with a shockingly large associated economic cost …

Some recombinant adeno-associated viruses (rAAVs) can cross the neonatal blood–brain
barrier (BBB) and efficiently transduce cells of the central nervous system (CNS). However …

For most lysosomal storage diseases (LSDs) affecting the CNS, there is currently no cure.
The BBB, which limits the bioavailability of drugs administered systemically, and the short …

Adeno-associated virus serotype 9 (AAV9)-mediated gene transfer has been reported in
central nervous system (CNS) and peripheral tissues. The current study compared the …

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 …