Haematopoietic stem cell transplantation arrests the progression of neurodegenerative disease in late-onset Tay-Sachs disease
Tay-Sachs disease is a rare metabolic disease caused by a deficiency of hexosaminidase A that leads to accumulation of GM2 gangliosides predominantly in neural tissue. Late-onset Tay-Sachs disease variant is associated with a higher level of residual HexA activity. Treatment options are limited, and...
| Автори: | , , , , , , , , , |
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| Формат: | Journal article |
| Мова: | English |
| Опубліковано: |
Springer, Berlin, Heidelberg
2017
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