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Treatment of neurodegenerative disease through intracranial delivery of short interfering rna (sirna)
Treatment of neurodegenerative disease through intracranial delivery of short interfering rna (sirna)
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机译:通过颅内短干扰RNA(Sirna)的颅内治疗来治疗神经退行性疾病
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摘要
The present invention provides devices, small interfering RNA, and methods for treating a neurodegenerative disorder comprising the steps of surgically implanting a catheter so that a discharge portion of the catheter lies adjacent to a predetermined infusion site in a brain, and discharging through the discharge portion of the catheter a predetermined dosage of at least one substance capable of inhibiting production of at least one neurodegenerative protein. The present invention also provides valuable small interfering RNA vectors, and methods for treating neurodegenerative disorders such as Alzheimer's disease, Parkinson's disease, Huntington's disease, Spinocerebellar Ataxia Type 1, Type 2, Type 3, and/or dentatorubral-pallidoluysian atrophy.
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