INTRAVENTRICULAR ENZYME DELIVERY FOR LYSOSOMAL STORAGE DISEASES

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...

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Bibliographic Details
Main Authors SHIHABUDDIN, Lamya, DODGE, James, CHENG, Seng H, PASSINI, Marco A
Format Patent
LanguageEnglish
Published 21.03.2019
Subjects
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
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Summary: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.
Bibliography:Application Number: US201816111009