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Methods of treating neurodegenerative diseases using gene therapy to delay disease initiation and progression while providing cognitive protection
Methods of treating neurodegenerative diseases using gene therapy to delay disease initiation and progression while providing cognitive protection
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机译：使用基因疗法治疗神经退行性疾病的方法可延缓疾病的发生和发展，同时提供认知保护
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摘要
The disclosure provides a method of treating a lysosomal accumulation disorder in a mammal, the method comprising direct administration of the AAV particle encoding the polypeptide with at least two immunosuppressant doses directly to the central nervous system of the mammal. AAV particles can be delivered by direct injection into the brain, spinal cord, cerebrospinal fluid or a portion thereof.
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