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Delivery of therapeutic compounds to the brain and other tissues
专利权人:
Emil D. Kakkis
发明人:
Emil D. Kakkis
申请号:
US11630446
公开号:
US09044473B2
申请日:
2004.08.30
申请国别(地区):
US
年份:
2015
代理人:
摘要:
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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