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ADENO-ASSOCIATED VIRAL VECTORS FOR TREATMENT OF NIEMANN-PICK DISEASE TYPE C
ADENO-ASSOCIATED VIRAL VECTORS FOR TREATMENT OF NIEMANN-PICK DISEASE TYPE C
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机译:腺相关病毒载体,用于治疗Niemann-pick疾病C型
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摘要
Provided herein are gene therapy compositions and methods for treating, preventing, and/or curing NPC1. More specifically, the disclosure provides Adeno-associated virus (AAV) vectors for delivery of nucleic acids and nucleic acids (including AAV transfer cassettes) for treating, preventing, and/or curing NPC1.
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