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Intracerebroventricular administration of a modified hexosaminidase ameliorates late-stage neurodege...

Intracerebroventricular administration of a modified hexosaminidase ameliorates late-stage neurodege...

https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_plos_journals_3151369441

Intracerebroventricular administration of a modified hexosaminidase ameliorates late-stage neurodegeneration in a GM2 mouse model

About this item

Full title

Intracerebroventricular administration of a modified hexosaminidase ameliorates late-stage neurodegeneration in a GM2 mouse model

Publisher

United States: Public Library of Science

Journal title

PloS one, 2025-01, Vol.20 (1), p.e0315005

Language

English

Formats

Publication information

Publisher

United States: Public Library of Science

More information

Scope and Contents

Contents

The GM2 gangliosidoses, Tay-Sachs disease and Sandhoff disease, are devastating neurodegenerative disorders caused by β-hexosaminidase A (HexA) deficiency. In the Sandhoff disease mouse model, rescue potential was severely reduced when HexA was introduced after disease onset. Here, we assess the effect of recombinant HexA and HexD3, a newly enginee...

Alternative Titles

Full title

Intracerebroventricular administration of a modified hexosaminidase ameliorates late-stage neurodegeneration in a GM2 mouse model

Identifiers

Primary Identifiers

Record Identifier

TN_cdi_plos_journals_3151369441

Permalink

https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_plos_journals_3151369441

Other Identifiers

ISSN

1932-6203

E-ISSN

1932-6203

DOI

10.1371/journal.pone.0315005

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