2.Verdera HC, Kuranda K, Mingozzi F. AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer. Mol Ther. 2020 Mar 4;28(3):723-746. doi: 3.1016/j.ymthe.2019.12.010. Epub 2020 Jan 10. PMID: 31972133; PMCID...
[5] Martinez-Fernandez de la Camara C, McClements M E, MacLaren R E. Accurate Quantification of AAV Vector Genomes by Quantitative PCR[J]. Genes, 2021, 12(4): 601. [6] BLA Clinical Review Memorandum (Zolgensma). FDA. 2019. [...
The lack of pathogenicity and the ability to integrate site-specifically are very attractive attributes for a gene therapy vector. A further attractive attribute is its ability to infect a wide range of cell types in a wide range of species. For example, in vivo infections have revealed that ...
经验分享:基因治疗AAV载体生产过程中rcAAV的鉴定和消除简介 腺相关病毒载体(adeno-associated viral vector,AAV vector)是一种具有良好应用前景的基因治疗载体。但是,腺相关病毒(AAV)载体制剂经常被不同量的具有复制能力的AAV病毒(rcAAV)污染,这可能影响这些载体在培养细胞和动物中的行为。构建了不含显著同源性且缺乏野...
1.Verdera, Helena Costa, Klaudia Kuranda, and Federico Mingozzi. "AAV vector immunogenicity in humans: A long journey to successful gene transfer." Molecular Therapy 28.3 (2020): 723-746.2. Shirley, Jamie L., et al. "Type I IFN sensing by cDCs and CD4+ T cell help are both ...
[1] Landegger LD, Pan B, Askew C, Wassmer SJ, Gluck SD, Galvin A, Taylor R, Forge A, Stankovic KM, Holt JR, Vandenberghe LH. A synthetic AAV vector enables safe and efficient gene transfer to the mammalian inner ear. Nat Biotechnol. 2017 Mar;35(3):280-284. ...
PackGene provides end-to-end AAV vector design and construction solutions for AAV gene delivery. Our professional vector design team can work directly with you to provide efficient & effective AAV vector design service upon your request. Simply provide u
Accurate Quantification of AAV Vector Genomes by Quantitative PCR[J]. Genes, 2021, 12(4): 601. [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). [...
7. Bulcha, J.T., Wang, Y., Ma, H. et al. Viral vector platforms within the gene therapy landscape. Sig Transduct Target Ther (2021) 6, 53. 8. Leborgne C, Barbon E, Alexander JM, et al. IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV ...
In this eBook learn step-by-step strategies for mastering AAV vector development and enhancing your gene therapy applications. Download Now Tech Note: An Efficient and Reliable Approach to AAV Packaging While AAV is a powerful vehicle for gene delivery, unstable ITRs threaten the fidelity of rA...