Delivery of therapeutic compounds to the brain and other...

Details Delivery of therapeutic compounds to the brain and other... Delivery of therapeutic compounds to the brain and other...

2003-08-29

2008-10-28

Weber, Jon (Department: 1657)

Drug, bio-affecting and body treating compositions

Enzyme or coenzyme containing

Hydrolases

C435S018000, C435S195000

Reexamination Certificate

active

07442372

ABSTRACT:
The present invention relates to the intrathecal (IT) administration of recombinant enzyme to treat lysosomal storage disorders. In an exemplary embodiment, intrathecal administration of human α-L-iduronidase (rhIDU) injections in MPS I affected animals resulted in significant enzyme uptake, significant rh-iduronidase activity in brain and meninges and a decrease of glycosaminoglycan (GAG) storage in cells of MPS I subjects to that of normal subjects. Intrathecal administration proved more effective than intravenous treatment at alleviating MPS I symptoms, indicating it is a useful method of treating lysosomal storage disorders.

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