INTRAVENTRICULAR ENZYME DELIVERY FOR LYSOSOMAL STORAGE DISEASES
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.
申请公布号
WO2007084737(A3)
申请公布日期
2008.10.23
申请号
WO2007US01566
申请日期
2007.01.22
申请人
GENZYME CORPORATION;DODGE, JAMES;PASSINI, MARCO;SHIHABUDDIN, LAMYA;CHENG, SENG
发明人
DODGE, JAMES;PASSINI, MARCO;SHIHABUDDIN, LAMYA;CHENG, SENG