טוען...
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...
שמור ב:
| הוצא לאור ב: | Gene Ther |
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| Main Authors: | , , , , , , , , , , |
| פורמט: | Artigo |
| שפה: | Inglês |
| יצא לאור: |
2020
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| נושאים: | |
| גישה מקוונת: | 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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