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Updated: Sep 12, 2025

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在AAV基因治疗中,细胞外矩阵的屏障功能

Yahya Cheema1, Devorah Cahn1,2, Sahana Kumar1

  • 1Fischell Department of Bioengineering, University of Maryland, College Park, MD 20742, USA.

bioRxiv : the preprint server for biology
|August 6, 2025
PubMed
概括

腺相关病毒 (AAV) 传输和基因传递可能会受到细胞外基质 (ECM) 的阻碍. 了解AAV-ECM相互作用对于有效的AAV基因疗法开发至关重要.

关键词:
腺相关病毒的病毒.细胞外矩阵是细胞外矩阵.多个粒子跟踪追踪.

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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...
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