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Gene Therapy cover

Gene Therapy

IF4.5
Papers219
Citations6730
Journal Papers 204
Publication Date
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An engineered helper plasmid generates differential E4orf6 and L4-22/33K gene expression increasing AAV vector production
deleteGene Therapy
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
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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
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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 study
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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
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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 strategies
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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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Gene augmentation therapy successfully treats mice with complete congenital stationary night blindness (cCSNB), improving retinal function and visual acuity
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IF4.5
delete2026-07-18
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deleteNazarul Hasan; Cecilia A. Attaway; Mattia Di Paolo; Maureen A. McCall; Ronald G. Gregg
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Reversing cancer cell behavior using AI-guided CRISPR and quantum nanobiology: a systems-based approach to epigenetic reprogramming
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IF4.5
delete2026-07-16
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PREAI
deleteBakr Ahmed Taha; Ali J. Addie; Adawiya J. Haider; Khalid Ibnaouf; Norhana Arsad
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FMR1 gene therapy restores translationally relevant phenotypes in a mouse model for fragile X syndrome
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IF4.5
delete2026-07-10
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deleteRichard K. Lacher; Kari Henson; Lindsay N. Wathen; Caitlin Jones; Tiffany Arnold; McKenzie R. Rice; Heather M. Carles; Angela R. White; Elizabeth Ramsuchit; Darren Murrey; Rebecca Raig; Austen Fisher; Kaitlin Bucher; Grace C. Westerkamp; Adam L. Fritz; Brooke M. Gollaway; Sebastian Piloto; David Dismuke; J Elliott Robinson; Michael T. Williams; Charles V. Vorhees; Erandi K. De Silva; Durgesh Tiwari; Craig A. Erickson; Ernest V. Pedapati; Christina Gross
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Improving the precision of AAV lung gene therapy for SP-B deficiency using computationally derived lung-specific promoters
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IF4.5
delete2026-06-30
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PREAI
deleteNicole Zielinska; Erin. L. Howard; Brenna A. Y. Stevens; Melanie M. Goens; Elena S. B. Campbell; Madison E. Hughes; Yanlong Pei; Liqun Xu; Adithya Achuthan; Arul Vadivel; Arnold Apostol; Xiaolin He; Dinghai Zheng; D. Benjamin Gordon; Raja R. Srinivas; Alec A. K. Nielsen; Jeff L. Caswell; Luis G. Arroyo; Darren A. Yuen; Bernard Thebaud; Sarah K. Wootton
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Recent advancements in improving cross-species applicability of bioengineered AAV capsids
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IF4.5
delete2026-06-27
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PREAI
deleteHaolai Pan; Nianci Li; Jieyu Qi; Renjie Chai
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Assessment of F/HN-pseudotyped lentiviral vector following intravenous delivery to mice
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IF4.5
delete2026-06-18
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deleteRobyn V. Bell; Nikhil B. Faulkner; Anthony Sinadinos; Cuixiang Meng; Emily Castells; Mariana A. Viegas; Stephen C. Hyde; Deborah R. Gill; Eric WFW Alton; Uta Griesenbach
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Applications of genome editing technologies in the treatment of human diseases
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IF4.5
delete2026-06-15
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PREAI
deleteJamal Alshorman; Mohammad Javad Mehran; Kingsley Miyanda Tembo; Niusha mostafavi; Mansoor Bolideei; Yongping Wang
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High resolution ES-DMA quantifies AAV capsid DNA content by electrical mobility to mass correlation
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IF4.5
delete2026-06-04
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deletePreston Dennett; Lohra M. Young; Benjamin E. Draper; Jerome Schmitt; Martin Jarrold; Luis Javier Perez-Lorenzo
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AAV8-mediated mouse/human PROC expression rescues thrombophilia in hereditary protein C-deficient mice
deleteGene Therapy
IF4.5
delete2026-06-01
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deleteTingting Wu; Yanyi Tao; Hui Lu; Yaohua Cai; Yunqing Xia; Tingting Liu; Li Wang; Zhipeng Cheng; Yu Hu; Javier Corral; Liang V. Tang
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Environmental and developmental factors shape anti-AAV immunity in pigs
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IF4.5
delete2026-05-29
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deleteGodwin I. Iroanya; Ilangovan Raju; Chandra Boosani; Pradeep N. Subramanyam; Addison K. Byrne; Kevin D. Wells; Jonathan A. Green
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Characterization of CAR-T cell factors that contribute to myeloid cell activation
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IF4.5
delete2026-05-26
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deleteSupreet Khanal; Md Kamal Hossain; Joseph Fischer; Santosh Panthi; Alan Baer; Nirjal Bhattarai
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DOCTER: a genetically encoded switchable protein module for ERα-mediated transcriptional regulation
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IF4.5
delete2026-05-26
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PREAI
deleteJinmin Wang; Jingfang Liu; Dandan Peng; Yuhao Ji; Ke Huang; Junfen Fu; Yingke Xu
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Research progress and development strategies of antibody-oligonucleotide conjugates
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IF4.5
delete2026-05-25
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deleteWenshuang Fan; Weijing Luan; Wenjun Yu; Yan Li; Chao Wei; Xinfeng Liu; Ling Ni; Haiqiang Cao; Chunyan Fang; Yuanlei Fu
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rAAV8 encapsidated HMR-001/z enables high efficiency hepatic transduction and restores hemostasis in hemophilic mice
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IF4.5
delete2026-05-16
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PREAI
deleteXiaomo Wu; Jinying Zhong; Xiaorong He; Ming Wu; Yu He; Jiguang Wu; Chunping Zhang; Jinyan Zhang; Meijun Guo; Xiaochang Jiang; Junyong Wong
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