Disease correction in mucopolysaccharidosis type IIIB mice by intraparenchymal or cisternal delivery of a capsid modified AAV8 codon-optimized NAGLU vector
CJ Rouse, K Hawkins, N Kabbej… - Human Molecular …, 2023 - academic.oup.com
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 …
storage disease caused by mutations in the gene that encodes the protein N-acetyl …
[PDF][PDF] Mucopolysaccharidoses: An overview and new treatment modalities
P Shivakumar - researchgate.net
Mucopolysaccharidoses are a group of inherited lysosomal storage disorders, caused due
to deficiency of enzymes required for the breakdown of Mucopolysaccharides. These …
to deficiency of enzymes required for the breakdown of Mucopolysaccharides. These …