随着全球基因治疗市场的高速增长，腺相关病毒（AAV）因其能长期表达、毒性低、免疫原性低、组织特异性高等优势，已成为基因治疗领域最重要的基因载体之一。

Adeno-associated viral (AAV) vectors are widely used for gene therapy, providing treatment for diseases caused by absent or defective genes. Despite the success of gene therapy, AAV manufacturing is still challenging, with production yields being ...

2024年4月3日 · Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene therapy owing to its minimal pathogenicity and ability to establish long-term...

2025年1月16日 · Adeno-associated virus (AAV) is a versatile viral vector technology that can be engineered for specific functionality in vaccine and gene therapy applications. One of the major challenges in AAV production is the need for a GMP-ready platform-based approach to downstream processing, as this would lead to a standardized method for multiple products.

本文带大家了解肺脏特异性基因调控策略，主要从肺组织特性及组成、肺部的常见疾病、肺嗜性AAV血清型、肺特异性启动子和病毒递送到肺部的方法等五个方面展开分享。

已报道有多种病毒载体可用于体内基因治疗，包括腺相关病毒（AAV）、腺病毒（Ad）、逆转录病毒、慢病毒和单纯疱疹病毒（HSV）等，其中AAV载体已成为临床试验和FDA批准应用中的首选。

AAV 是属于细小病毒科的小型无囊膜 DNA 病毒，于 1965 年首次分离出来，作为猿猴腺病毒 (Ad) 制剂中的污染物。 这些病毒被发现无法有效地感染细胞，除非与辅助病毒（通常是 Ad 或任何类型的疱疹病毒）共同感染，因此它们被命名为“腺相关病毒”，并被分类为 ...

衣壳设计是为不同临床应用开发定制rAAV的主要策略。 它主要包括三种方法：鉴定自然存在的AAV变体、理性设计和定向进化（图1）。 最近，AI辅助进化已成为rAAV衣壳设计的新工具。

Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the treatment of a variety of human diseases. Recent advances in developing clinically desirable AAV capsids, optimizing genome designs and harnessing revolutionary biotechnologies have contributed substantially to t …

2023年5月16日 · Adeno-associated viruses (AAVs) are one of the most promising tools for gene therapy applications. These vectors are purified using affinity and ion exchange chromatography, typically using packed beds of resin adsorbents. This leads to diffusion and pressure drop limitations that affect process productivity.