Article

Brain transplantation of genetically corrected Sanfilippo B Neural Stem Cells induces partial cross-correction of the disease

2022-07-02

Abstract excerpt

<h4>ABSTRACT</h4> Sanfilippo syndrome type B (Mucopolysaccharidosis type IIIB or MPS IIIB) is a recessive genetic disorder that severely affects the brain due to a deficiency in the enzyme α- N -acetylglucosaminidase (NAGLU), leading to intralysosomal accumulation of partially degraded heparan sulfate. There are no effective treatments for this disorder. In this project, we carried out an ex vivo lentiviral cor...

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Literature Corpus work: 2c42f16a-0241-534a-b817-c5dae29c4951
DOI: 10.1101/2022.06.30.498131

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