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Updated: Jun 5, 2026

Engineering and Evolution of Synthetic Adeno-Associated Virus AAV Gene Therapy Vectors via DNA Family Shuffling
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Published on: April 2, 2012

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基于机器学习的图书馆设计中最佳的权衡控制,适用于基因治疗的腺相关病毒 (AAV)

Danqing Zhu1, David H Brookes2, Akosua Busia3

  • 1California Institute for Quantitative Biosciences, University of California, Berkeley, Berkeley, CA 94720, USA.

Science advances
|January 24, 2024
PubMed
概括

机器学习增强了用于基因治疗的腺相关病毒 (AAV) 插入库. 这种新方法提高了包装效率,并确定了大脑组织输送的有希望的质细胞特异变体.

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相关概念视频

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...
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...