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Prevalence of pre-existing neutralizing antibodies to AAV5 and AAV8 in patients with Wilson's disease威尔逊病患者中AAV5和AAV8预存中和抗体的流行率
Zheng, Zi-Wei; Liu, Hong; Guo, Ling-Yu; Fan, Lu-Na; Liu, Chuan; Li, Fu-Rong; Zhou, Ran; Qu, Lin; Wang, Rou-Min; Xu, Wan-Qing; Yang, Guo-Min; Dong, Biao; Dong, Yi; Wu, Zhi-Ying.png)
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收藏 Efficient and safe lung gene delivery using AAV6.2FF in neonatal pigs demonstrates pediatric translational potentialAAV6.2FF在新生猪中的高效安全肺基因递送展示了儿科转化潜力Zielinska, Nicole; Howard, Erin L.; Yang, Cici; Stevens, Brenna A. Y.; Goens, Melanie M.; Pei, Yanlong; Thompson, Brad; Caswell, Jeff L.; Thebaud, Bernard; Wey, Douglas; Valverde, Alexander; Arroyo, Luis G.; Wootton, Sarah K. 分享 收藏 Efficacy of anti-VEGF single-chain variable fragment AAV-based gene therapy in a laser-induced choroidal neovascularisation mouse modelAAV介导的抗VEGF单链可变片段基因疗法在激光诱导的脉络膜新生血管小鼠模型中的疗效Salman, Ahmed; Griffiths, Helen; Garba, Kamaluddeen; Lynn, Savannah A.; Cree, Angela J.; Szczepan, Manon; Jashal, Charnitkaur B.; Chozhavel, T. M. Rajanathan; Rao, Poornima; Modi, Kush; MacLaren, Robert E.; Yuen, Ho Ming; Lotery, Andrew J. 分享 收藏 Systemic clinical-grade recombinant decorin reduces disease burden and fibrosis in advanced recessive dystrophic epidermolysis bullosa系统性临床级重组 decorin 可减轻显性营养不良型大疱性表皮松解症的疾病负担和纤维化Gretzmeier, Christine; Hang, Bing; Sengle, Gerhard; Phillips, Christopher; Buer, Ben; Fletcher, Sarah; Belov, Arseniy; Bleck, Gregory; Collins, Ian J.; Brinckmann, Jurgen; de Souza, Mark P.; Landy, Hal; Nystrom, Alexander 分享 收藏 Clinical development of tacrolimus-resistant regulatory T cells to enable simultaneous immunosuppression and immune regulation他克莫司抵抗性调节性T细胞的临床开发以实现同时免疫抑制和免疫调节Zarrinrad, Ghazaleh; Burkhardt, Lisa-Marie; Beltran-Mestres, Claudia; Romero-Suarez, Silvina; Wagner, Dimitrios Laurin; Schlickeiser, Stephan; Stein, Maik; Wendering, Desiree Jacqueline; de la Cebosa, Ivan Juky Carrera Diaz; Ehlen, Lukas; Hamm, Frederik; Kurgan, Gavin L.; Durek, Pawel; Heinrich, Frederik; Giri, Anamika; Pu, Yaolin; Ou, Kristy; Hoffmann, Henrike; Muench, Sandra; Stuewe, Insa; Dolling, Sven; McCallion, Oliver; Kaeda, Jaspal; Kath, Jonas; Schulenberg, Sarah; Wiese, Niklas; Nazari, Abdolreza; Peter, Lena; Picht, Samira; Sanchez-Pena, Andrea; Usman, Olalekan; Brommel, Christian; Turk, Rolf; Rettig, Garrett; Sturgeon, Morgan; Osborne, Thomas L.; Jacobi, Ashley; Friedrich, Rebecca; Kaufmann, Masako Monika; Klermund, Julia; Fink, Simon; Templin, Markus F.; Roemhild, Andy; Kaiser, Daniel; Klein, Oliver; Cathomen, Toni; Mashreghi, Mir-Farzin; Issa, Fadi; Polansky, Julia K.; Volk, Hans-Dieter; Schmueck-Henneresse, Michael; Reinke, Petra; Amini, Leila 分享 收藏 Continuous production of recombinant adeno-associated virus in the insect cell/baculovirus expression vector system昆虫细胞/杆状病毒表达载体系统中重组腺相关病毒的分批生产Joseph, John; Destro, Francesco; Yuan, Arella; Antov, Daniel; Chen, Wenyu; Song, Sally; Theriault, Matthew; Yuan, Chengcheng; Sansom, Alexander; Lundin, Chiara; Burns, Tyler; Hamed, Hadeel Abubaker; Morales, Piero; Fan, Jessica Xinyuan; Rodriguez, Avner Romero; Sichuma, Sanyambe; McCracken, Courtney; Neufeld, Caleb; Wolfrum, Jacqueline M.; Srinivasan, Prasanna; Barone, Paul W.; Sinskey, Anthony J.; Braatz, Richard D.; Springs, Stacy L. 分享 收藏 A single-cell multi-omics assay for simultaneous measurement of vector copy number and protein expression in CAR T cells单细胞多组学方法用于CAR T细胞中载体拷贝数和蛋白质表达的同步测量Yang, Yilong; Parikh, Saurabh; Li, Chieh-Yuan; Murphy, Lindsey; Patel, Khushali; Ayaz, Qawer; Mohiuddin, Mahir; Elliott, John T.; He, Hua-Jun; He, Zhiyong; Maragh, Samantha; Schroeder, Benjamin; Fry, Terry J.; Winters, Amanda; Wang, Shu 分享 收藏 Engineering novel AAV capsids by broadly attenuated and subsequent muscle-specific tropism in mice and NHPs通过广泛减毒和后续肌肉特异性趋向性，在啮齿动物和非人灵长类动物中工程化新型AAV衣壳Pan, Yue; Zhong, Yujian; Chen, Huan; Zhang, Youwei; Dai, Zhiyong; Chen, Junlin; Tan, Keqin; Chen, Xiaoqu; Qiu, Danlan; Sheng, Longxiang; Tan, Xinping; Fan, Ying; Bu, Ye; Zhou, Zexin; Yang, Zhiming; Duan, Rui; Guan, Min; Gao, Guangping; Li, Huapeng 分享 收藏 Efficient kidney gene transfer and proximal tubule transduction using self-complementary AAV.cc47 vectors (vol 33, 101606, 2025)使用自互补AAV.cc47载体实现高效的肾脏基因转移和近端小管转导（vol 33, 101606, 2025）Peek, Jennifer L.; Rosales, Alan; Qi, Julie; Feleke, Lena; Menshikh, Anna; Welch, Richard C.; Woodard, Lauren E.; Asokan, Aravind; Wilson, Matthew H. 分享 收藏 
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