## Abstract ## Background In some lysosomal storage diseases, considerable alterations of the central nervous system (CNS) occur prior to birth and neurodegeneration progresses rapidly soon after birth causing early death in patients. No effective treatment is available after birth. Treatment may
โฆ LIBER โฆ
Gene therapy in lysosomal diseases
โ Scribed by C Caillaud; L Poenaru
- Book ID
- 117575088
- Publisher
- Elsevier Science
- Year
- 2000
- Tongue
- English
- Weight
- 53 KB
- Volume
- 54
- Category
- Article
- ISSN
- 0753-3322
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## 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