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Updated: Jun 29, 2026

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微生理系统加速和降低AAV基因疗法开发风险

Patrizia Tornabene1, Estelle Berreur2, Adrian Roth3

  • 1Center for Stem Cell and Organoid Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45219, USA.

Advanced drug delivery reviews
|March 7, 2026
PubMed
概括

微生理系统 (MPS) 提供了一个与人类相关的平台来评估腺相关病毒 (AAV) 载体,提高基因治疗的安全性和有效性. 这些先进的模型有助于预测临床结果和降低AAV载体发展风险.

关键词:
在AAV基因疗法中,AAV基因疗法免疫性 免疫性 免疫性微生理系统是微生理系统.器官类动物 器官类动物器官在芯片上的器官临床前测试 临床前测试有毒性 有毒性

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Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...