Literature summary for 3.2.1.50 extracted from

Fu, H.; DiRosario, J.; Kang, L.; Muenzer, J.; McCarty, D.M.
Restoration of central nervous system alpha-N-acetylglucosaminidase activity and therapeutic benefits in mucopolysaccharidosis IIIB mice by a single intracisternal recombinant adeno-associated viral type 2 vector delivery (2010), J. Gene Med., 12, 624-633.

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Application

Application	Comment	Organism
medicine	expression of human NaGlu in the central nervous system of mucopolysaccharidosis IIIB mice by an i.c. injection approach. The treatment slows the disease progression by mediating widespread recombinant NaGlu expression in the central nervous system, resulting in the reduction of brain lysosomal storage pathology, significantly improved cognitive function and prolonged survival. The therapeutic benefit of i.c. recombinant adeno-associated viral vector delivery is dose-dependent and can be attribute solely to the central nervous system transduction because the procedure does not lead to detectable transduction in somatic tissues	Homo sapiens

Protein Variants

Protein Variants	Comment	Organism
additional information	expression of human NaGlu in the central nervous system of mucopolysaccharidosis IIIB mice by an i.c. injection approach	Homo sapiens

Organism

Organism	UniProt	Comment	Textmining
Homo sapiens	P54802	-	-

Source Tissue

Source Tissue	Comment	Organism	Textmining

Synonyms

Synonyms	Comment	Organism
NAGLU	-	Homo sapiens

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Release 2023.1 (January 2023)