Methods and Systems for Treatment of Neurological Diseases of the Central Nervous System
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Abstract
The present invention is directed to methods and systems for the treatment of inborn genetic errors or other defects that cause deficiencies of active enzymes or proteins within the cells of the central nervous system. Such methods and systems generally comprise an implantable catheter system designed for the chronic delivery of specially formulated proteins to intrathecal, intracerebroventricular, and/or intraparenchymal regions of the central nervous system. The invention has application in the neuropathic aspects of the broad category of lysosomal storage diseases. These genetic based diseases are the result of insufficient enzyme activity to catabolize specific substances, which thereby accumulate in the cellular lysosomes.
115 Citations
103 Claims
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1-76. -76. (canceled)
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77. A method comprising the step of:
physically delivering a therapeutic protein formulation selected to treat a neurological disease of the central nervous system via an implantable catheter system across the blood brain barrier of a patient with the neurological disease, wherein the therapeutic protein formulation comprises proteins that have been modified for enhanced cellular uptake. - View Dependent Claims (78, 79, 80, 81, 82, 83, 84, 85, 86, 87, 88, 89, 90, 91, 92, 93, 94, 95, 96, 97, 98, 99, 100, 101, 102)
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103-128. -128. (canceled)
Specification