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STABLE EXPRESSION OF AAV VECTORS IN JUVENILE SUBJECTS
STABLE EXPRESSION OF AAV VECTORS IN JUVENILE SUBJECTS
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机译:少年受试者AAV载体的稳定表达
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摘要
The invention relates to the use of adeno-associated virus (AAV) vectors to achieve long term expression of a transgene in the liver of a juvenile subject. The invention includes the stable long-term amelioration of disease symptoms of the subjection following a single administration of an AAV vector to a juvenile subject, wherein the AAV vector delivers the transgene to the subject's liver.
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