Bicistronic lentiviral vector corrects β-hexosaminidase deficiency in transduced and cross-corrected human Sandhoff fibroblasts
Sandhoff disease is an autosomal recessive neurodegenerative disease characterized by a GM2 ganglioside intralysosomal accumulation. It is due to mutations in the β-hexosaminidases β-chain gene, resulting in a β-hexosaminidases A (αβ) and B (ββ) deficiency. Mono and bicistronic lentiviral vectors co...
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| 主要な著者: | , , , , , , , , , , |
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| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
Elsevier
2005-11-01
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| シリーズ: | Neurobiology of Disease |
| 主題: | |
| オンライン･アクセス: | http://www.sciencedirect.com/science/article/pii/S0969996105001336 |
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