๐”– Bobbio Scriptorium
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Gene therapy in lysosomal diseases

โœ 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 ## 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

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