但Sf9和哺乳动物细胞制造平台产出的AAV表现出不同的化学和功能,例如不同的衣壳蛋白体翻译后修饰,BEV生产的AAV可能有更差的包装百分比。 在这篇review中,大佬带读者们回顾了科研人员为克服AAV作为基因传递系统的限制,在AAV衣壳改造、基因组设计以及病毒生产中的努力。总体来说,可行的手段还是依旧,目前有理论优势的方...
RichardJude Samulski在Molecular Therapy上刚发表了一篇关于AAV载体的review,AAV Vectors The Rubik’s Cube of Human Gene Therapy。题目就很有意思,魔方本身有多个平面,每个平面上又有不同的颜色,各种角块、棱块的位置让魔方有海量的花色组合。AAV也是这样,不同...
这篇文章来自微信公众号“细胞与基因治疗领域”，作者向阳屯铁匠分享了RichardJude Samulski在Molecular Therapy上发表的关于AAV载体的review文章，以“AAV载体：人类基因治疗的魔方”为题，将AAV的复杂性比喻为魔方的多样性。文章探讨了AAV的衣壳改造，包括天然血清型的特性、翻译后修饰的异质性、嵌合型衣壳的...
2. Allison M. Keeler and Terence R. Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here? Flotte Annual Review of Virology (2019) 6:601-621.3. Kuzmin DA, et al., The clinical landscape for AAV gen...
2、A multicenter, retrospective medical record review of X-linked myotubular myopathy: The RECENSUS study. Muscle Nerve. 2018;57(4):550–60. 3、Medical complications in long-term survivors with X-linked myotubular myopathy. ...
2.Papaioannou I, Owen J S, Yáñez‐Muñoz R J. Clinical applications of gene therapy for rare diseases: A review[J]. International Journal of Experimental Pathology, 2023. 3.https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/luxturna ...
3. Keeler, A. M., & Flotte, T. R. (2019). Recombinant adeno-associated virus gene therapy in light of Luxturna (and Zolgensma and Glybera): Where are we, and how did we get here? *Annual Review of Virology*, 6, 601-621....
[6] BLA Clinical Review Memorandum (Zolgensma). FDA. 2019. [7] European Medicines Agency. EMA General Principles to Address Virus and Vector Shedding (EMEA/CHMP/ICH/449035/2009). [8] US FDA, Center for biologics evaluation and research. Guidance for industry: Design and Analysis...
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In the current review, we discuss the strategies employed to achieve selective transduction of microglia, including the use of cell-specific promoters, engineered capsids, and microRNA (miRNA) strategies. Additionally, we address the challenges and future directions in the development of AAV-based ...