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Intraventricular enzyme administration in lysosomal deposition disorders
Intraventricular enzyme administration in lysosomal deposition disorders
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机译:溶酶体沉积障碍的脑室内酶管理
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摘要
Lysosomal storage diseases can be successfully treated using intraventricular delivery of the enzyme which is etiologically deficient in the disease. The administration can be performed slowly to achieve maximum effect. Surprisingly, effects are seen on both sides of the blood-brain barrier, making this an ideal delivery means for lysosomal storage diseases which affect both brain and visceral organs.
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