Mucopolysaccharidosis type IIIB: a current review and exploration of the AAV therapy landscape

Mucopolysaccharidosis type IIIB: a current review and exploration of the AAV therapy landscape

Mucopolysaccharidoses type IIIB is a rare genetic disorder caused by mutations in the gene that encodes for N-acetyl-alpha-glucosaminidase. This results in the aggregation of heparan sulfate polysaccharides within cell lysosomes that leads to progressive and severe debilitating neurological dysfunct...

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Bibliographic Details
Main Authors: Courtney J Rouse, Victoria N Jensen, Coy D Heldermon
Format: Article
Language:English
Published: Wolters Kluwer Medknow Publications 2024-01-01
Series:Neural Regeneration Research
Subjects:
adeno-associated virus; central nervous system; gene therapy; heparan sulfate; immune response; mucopolysaccharidoses type iiib; n-acetyl-alpha-glucosaminidase; newborn screening
Online Access:http://www.nrronline.org/article.asp?issn=1673-5374;year=2024;volume=19;issue=2;spage=355;epage=359;aulast=

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