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Gene Therapy cover

Gene Therapy

IF4.5
Papers227
Citations6730
Journal Papers 212
Publication Date
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Correction: CRISPR/Cas9-mediated gene correction of Wilson disease H1069Q point mutation in patient-specific induced pluripotent stem cells
deleteGene Therapy
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
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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
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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
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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
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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
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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
deleteGene Therapy
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
deleteGene Therapy
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
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
deleteGene Therapy
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
deleteGene Therapy
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
deleteGene Therapy
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
deleteGene Therapy
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
deleteGene Therapy
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
deleteGene Therapy
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
deleteGene Therapy
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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