Therapeutic benefit after intracranial gene therapy delivered during the symptomatic stage in a feline model of Sandhoff disease

Sandhoff disease (SD) is an autosomal recessive lysosomal storage disease caused by defects in the β-subunit of β-N-acetylhexosaminidase (Hex), the enzyme that catabolizes GM2 ganglioside (GM2). Hex deficiency causes neuronal storage of GM2 and related glycoconjugates, resulting in progressive neuro...

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Vydáno v:Gene Ther
Hlavní autoři: McCurdy, Victoria J., Johnson, Aime K., Gray-Edwards, Heather L., Randle, Ashley N., Bradbury, Allison M., Morrison, Nancy E., Hwang, Misako, Baker, Henry J., Cox, Nancy R., Sena-Esteves, Miguel, Martin, Douglas R.
Médium: Artigo
Jazyk:Inglês
Vydáno: 2020
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Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7925702/
https://ncbi.nlm.nih.gov/pubmed/32884151
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41434-020-00190-1
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