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Gene therapy for the central nervous system in the lysosomal storage disorders

✍ Scribed by Kaye, Edward M; Sena-Esteves, Miguel

Book ID: 123402718
Publisher: Elsevier Science
Year: 2002
Tongue: English
Weight: 546 KB
Volume: 20
Category: Article
ISSN: 0733-8619
DOI: 10.1016/S0733-8619(02)00010-5

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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