Gene therapy/cell therapy for lysosomal storage disease
β Scribed by Y. Eto; T. Ohashi
- Book ID
- 110228308
- Publisher
- Springer
- Year
- 2000
- Tongue
- English
- Weight
- 64 KB
- Volume
- 23
- Category
- Article
- ISSN
- 0141-8955
No coin nor oath required. For personal study only.
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## Abstract In the last years, much progress has been achieved in the field of lysosomal storage disorders. In the past, no specific treatment was available for the affected patients; management mainly consisted of supportive care and treatment of complications. As orphan drug regulations, however,
## Abstract There are more than 40 different forms of inherited lysosomal storage diseases (LSDs) known to occur in humans and the aggregate incidence has been estimated to approach 1 in 7000 live births. Most LSDs are associated with high morbidity and mortality and represent a significant burden