AAV1、6、8 和 9 是最常用心脏血清型。 其中，AAV9 被认为心脏基因递送的最有效血清型。 我们对 AAV6 和 AAV9 进行了心肌转导测试，效果如下图。 载体家测试 AAV6-CMV-EGFP 和 AAV9-CMV-EGFP 转导小鼠心肌细胞心脏切片。 尾静脉注射，注射量：1.51 x 10 vg/g。 载体家不仅提供广泛表达启动子，如 CMV、EF1A、EFS、CAG、CBh 等，还能提供 cTnT 和αMHC 两种常用的心肌特异性 启动子，帮助您高效构建 AAV 载体。 小鼠 Mybpc3 基因治疗有助于长期预防肥厚性 …

In the field of cardiovascular disease (CVD), compared with non-viral vectors, lentiviruses, poxviruses, and adenovirus vectors, AAV possesses several advantages, including high security, low immunogenicity, sustainable and stable exogenous gene expression etc., which makes AAV one of the most promising candidates for the treatment of many genet...

2021年1月26日 · For example, adeno-associated virus (AAV)-mediated gene therapy has been approved by the FDA for Leber congenital amaurosis 13 and spinal muscular atrophy 14. A lipid...

Cardiac gene transfer is an attractive tool for developing novel heart disease treatments. Adeno-associated viral (AAV) vectors are widely used to mediate transgene expression in animal models and are being evaluated for human gene therapy.

Cardiac gene therapy with adeno-associated virus (AAV)-based vectors is emerging as an entirely new platform to treat, or even cure, so far intractable cardiac disorders. This review describes our current knowledge of cardiac AAV gene therapy with a ...

2023年7月6日 · Here we describe extracellular vesicle–encapsulated AAVs (EV-AAVs), secreted naturally by AAV-producing cells, as a superior cardiac gene delivery vector that delivers more genes and offers higher NAb resistance. We developed a 2-step density-gradient ultracentrifugation method to isolate highly purified EV-AAVs.

2020年4月10日 · Objective: We characterized murine germline TAZ knockout mice (TAZ-KO) and cardiomyocyte-specific TAZ knockout mice models and tested the efficacy of adeno-associated virus (AAV)-mediated gene replacement therapy with human TAZ (hTAZ).

Cardiac gene therapy with adeno-associated virus (AAV) vectors is emerging as a potential modality for addressing this desperate clinical need. After showing initial promise in extensive preclinical studies and an early clinical trial, disappointing results of large-scale clinical trial derailed the progress of AAV-mediated cardiac gene therapy.

The use of vectors based on the small parvovirus adeno-associated virus has gained significant momentum during the past decade. Their high efficiency of transduction of postmitotic tissues in vivo, such as heart, brain, and retina, renders these vectors extremely attractive for several gene therapy applications affecting these organs.

2024年3月18日 · We show that a single dose of AAV9:PKP2 gene delivery prevents disease development before the onset of cardiomyopathy and attenuates disease progression after overt cardiomyopathy.