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Gene Therapy 封面图

Gene Therapy

IF4.5
论文数286
被引数6730
期刊论文 286
发表时间
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Renal CLCN5 gene therapy for Dent disease type 1: local rapamycin immune modulation and age-dependent outcomes with lentiviral and AAV9 vectors肾CLCN5基因治疗I型 Dent病：局部雷帕霉素免疫调节及与慢病毒和AAV9载体的年龄依赖性结局
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IF4.5
delete2026-11-02
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deletePin Lyu; Aykut Bostanci; Cuili Jiang; Kyung What Yoo; Rhyston Broadhurst; Anthony Atala; Baisong Lu
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AAV8-PDE6B with 3’UTR provides rescue of photoreceptor structure and function in the rd10 mouse model of retinitis pigmentosaAAV8-PDE6B 带有 3’UTR 在rd10视网膜色素变性小鼠模型中挽救视杆细胞的结构和功能
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IF4.5
delete2026-11-02
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deleteSteven S. Eamegdool; To Ha Loi; Amin Sabri; Fidelle Chahine Karam; Anson Cheng; Leszek Lisowski; John R. Grigg; Ian Alexander; Robyn V. Jamieson
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In vivo frataxin expression of a triplex purified minicircle三重纯化微环体在体内的脆性X相关蛋白表达
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IF4.5
delete2026-10-31
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deleteNegin Mozafari; Salomé Milagres; Raul Cuellar; Karin E. Lundin; Maureen Fredriks; Tina-Thien Ho; MD Golam Azam; Jill S. Napierala; Marek Napierala; Jesper Wengel; C. I. Edvard Smith; Pontus Blomberg; Rula Zain
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AAV-mediated delivery of leptin but not adiponectin improves metabolic health in a pre-clinical mouse model of congenital generalised lipodystrophyAAV介导的瘦素而非脂联素递送可改善先天性全身性脂营养不良预临床试验小鼠模型的代谢健康
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IF4.5
delete2026-10-23
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deleteNadine Sommer; Ahlima Roumane; Mansi Tiwari; Weiping Han; Lora K. Heisler; George D. Mcilroy; Justin J. Rochford
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Chronic lung infection does not impair lentiviral vector gene transfer in a cystic fibrosis rat model慢性肺部感染不会损害在囊性纤维化大鼠模型中的慢病毒载体基因转移
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IF4.5
delete2026-10-19
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deleteAlexandra McCarron; Nicole Reyne; Bernadette Boog; Patricia Cmielewski; Martin Donnelley
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Statistical correlation between strength and mini-dystrophin expression in skeletal muscle after an rAAV9-mini-dystrophin gene therapy candidate in DMDmdx ratsDMDmdx大鼠经rAAV9-mini- dystrophin基因治疗候选方案后，骨骼肌中强度与mini- dystrophin表达之间的统计相关性
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IF4.5
delete2026-10-17
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PREAI
deleteJ. M. Walsh; A. McIntosh; J. Owens; C. Le Guiner; A. Lafoux; C. Huchet; M. Binks; S. Neelakantan; V. Farrokhi; H. Neubert
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Correction: CRISPR/Cas9-mediated gene correction of Wilson disease H1069Q point mutation in patient-specific induced pluripotent stem cells修正：利用CRISPR/Cas9介导的患者特异性诱导多能干细胞中威尔逊病H1069Q点突变的基因修复
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IF4.5
delete2026-10-02
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deleteViktoria Iwan; Oksana Nadzemova; Matthias Weiand; Andree Zibert; Hartmut H. Schmidt; Phil-Robin Tepasse; Robert Schierwagen; Jonel Trebicka; Vanessa Sandfort
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Local targeted suppression of neocortical spreading depolarization amplitude by viral expression of KCa3.1 channels通过病毒表达KCa3.1通道对局部新皮层扩散性去极化幅度的靶向抑制
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IF4.5
delete2026-09-29
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PREAI
deleteIlya A. Oblasov; Maria P. Smirnova; Anastasia A. Borodinova; Alena B. Zuzina; Ivan A. Smirnov; Pavel M. Balaban; Lyudmila V. Vinogradova; Evgeny S. Nikitin
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Rational selection of SpCas9 and variant-derived adenine base editors for human genome editing理性选择SpCas9及其变体衍生的腺嘌呤碱基编辑器用于人类基因组编辑
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IF4.5
delete2026-09-27
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PREAI
deleteJiahua Wang; Leben He; Lin Li; Yujing Zhang; Yuanyuan Wang; Yuejun Yang; Zongming Song; Yuanbo Liang; Jin Yang; Feng Gu
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Patient, consumer and societal values, perceptions and preferences on high-cost gene therapies患者、消费者和社会对高价基因治疗的价值、认知与偏好
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IF4.5
delete2026-09-17
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deleteAmanda Rush; Maria Gomez; Kristine Pierce; De Abreu Lourenco Richard; Kylie Mason; Kirsten Howard; Garry Lynch; Pippy Walker; Rosalie Viney; Jo Watson
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Intratumor delivery of a novel plasmid combination induces high levels of metastatic melanoma regression新型质粒组合的瘤内递送可诱导高水平的转移性黑色素瘤消退
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IF4.5
delete2026-09-16
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deleteLoree C. Heller; Julie S. Singh; Jody C. Synowiec; Mark J. Jaroszeski; Alex Otten; Richard Heller
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Culture media alter retinal organoid physiology promoting AAV transduction and retinal ganglion cell survival培养基改变视网膜类器官生理特性，促进AAV转导和视网膜神经节细胞存活
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IF4.5
delete2026-09-05
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deleteMichelle O’Hara-Wright; Benjamin Y. Lim; Melissa M. Mangala; Vivienne Kaiser; Emilie Wong; Deborah Aubin; Victoria Nemeruck; Hallur Reynisson; Fatemeh Doroudian; Otilia P. Y. Chan; Nader Aryamanesh; Joao A. Paulo; Stefano Palomba; Mehdi Mirzaei; Samantha L. Ginn; Anai Gonzalez-Cordero
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Optogenetic vision restoration: translational barriers and emerging therapeutic strategies光遗传学视觉恢复：转化障碍和新兴治疗策略
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IF4.5
delete2026-09-02
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PREAI
deleteNaoyuki Nakada
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Mapping the global trends and hotspots of research on gene therapy and hair cell regeneration for hearing loss: a comprehensive data-mining-based study绘制基因治疗和毛细胞再生治疗听力损失的研究全球趋势与热点：一项基于综合数据挖掘的研究
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IF4.5
delete2026-08-26
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PREAI
deleteYunsheng Liu; Bing Hu; Jinfang Zhang; Xianlin Wu; Qinghui Lu; Yanyan Jia; Zengwei Kou
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An engineered helper plasmid generates differential E4orf6 and L4-22/33K gene expression increasing AAV vector production工程化辅助质粒通过差异化表达E4orf6和L4-22/33K基因提高AAV载体产量
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IF4.5
delete2026-08-07
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deleteLaura van Lieshout; Katrina Costa-Grant; Dimpal Lata; Alejo Zacarias; Stacy Ota; Annie Adusei; Devin Schroeder; Diane Golebiowski; Ifeyinwa Iwuchukwu
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A gene therapy approach to prevent dilated intercellular space, a hallmark of gastroesophageal reflux disease一种预防食管裂孔疝的基因治疗策略，这是一种胃食管反流病的标志性特征
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IF4.5
delete2026-08-06
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deleteGillian M. Schiralli Lester; Jing Huang; Michael Barravecchia; Lananh N. Whigham; Jada M. Hooker; Karen L. de Mesy Bentley; Jennifer L. Young; Zhongren Zhou; David A. Dean
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Precision correction of the GJB2 c.235delC mutation by prime editing in vitro在体外利用碱基编辑技术对GJB2 c.235delC突变进行精确修复
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IF4.5
delete2026-08-06
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PREAI
deleteJiang Jin; Xiujuan Lv; Yangyang Li; Yaoyao Xu; Lei Chen; Lili Xie; Kai Xu; Jineng Lv
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SACF and GILA for in vitro transformation assessment of CRISPR/Cas9-edited cell therapy candidates: a multi-site studySACF和GILA用于体外评估CRISPR/Cas9编辑的细胞治疗候选物的转化能力：一项多中心研究
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IF4.5
delete2026-07-29
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deleteLena Dorsheimer; Joana Rita Ferreira; Bojing Wang; Rebecca Bohnert; Rashade Haynes II; Sabine Kaiser; Andreas Neerincx; Corinna Smolarek; Louise Delsing; Benoit Fischer; Stefanie Könen; Andreas Schulz; Kyle L. Kolaja; Ridhirama Bhuwania; Connie L. Chen; Lucilia Pereira Mouriès; Mick Fellows; Silvana Libertini; Myriam Lemmens
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Bone- and muscle-targeted adeno-associated viral vectors enable tissue-selective vitamin D receptor knockdown in mice骨和肌肉靶向腺相关病毒载体使小鼠实现组织选择性维生素D受体敲低
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IF4.5
delete2026-07-29
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deleteAlexandra K. O’Donohue; Julian Chu; Nicholas Norris; Hsien-Yin Kao; Josephine Yu; Lucinda R. Lee; Dirk Grimm; Jenny E. Gunton; Aaron Schindeler
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rAAV production cost analysis: Indication-specific cost per dose and reduction strategiesrAAV生产成本分析：基于适应症的每剂成本及降低策略
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IF4.5
delete2026-07-21
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deleteMin Tae Park; Dan Matuszek; Angela Andaluz; Seyed Pouria Motevalian
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