Search Results - "Genome editing"

Editing of homologous globin genes by nickase-deficient base editor mitigates large intergenic deletions in HSPCs by Anila George, Poornasree Sadanandan, Nithin Sam Ravi, B. Vaishnavi, Srujan Marepally, Saravanbhavan Thangavel, Shaji R. Velayudhan, Alok Srivastava, Kumarasamypet M Mohankumar

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CRISPR/Cas9-Mediated Three Nucleotide Insertion Corrects a Deletion Mutation in MRP1/ABCC1 and Restores Its Proper Folding and Function by Qinqin Xu, Yue-xian Hou, Xiu-bao Chang

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Repositioning of Lansoprazole as a Protective Agent Against Cisplatin-Induced Ototoxicity by Eri Wakai, Kenji Ikemura, Toshiro Mizuno, Kazuhiko Takeuchi, Satoshi Tamaru, Masahiro Okuda, Yuhei Nishimura

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Efficient Generation of Orthologous Point Mutations in Pigs via CRISPR-assisted ssODN-mediated Homology-directed Repair by Kankan Wang, Xiaochun Tang, Yan Liu, Zicong Xie, Xiaodong Zou, Mengjing Li, Hongming Yuan, Hongsheng Ouyang, Huping Jiao, Daxin Pang

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Preclinical evaluation of CRISPR-based therapies for Noonan syndrome caused by deep-intronic LZTR1 variants by Carolin Knauer, Henrike Haltern, Eric Schoger, Sebastian Kügler, Lennart Roos, Laura C. Zelarayán, Gerd Hasenfuss, Wolfram-Hubertus Zimmermann, Bernd Wollnik, Lukas Cyganek

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Cytosine Deaminase Base Editing to Restore COL7A1 in Dystrophic Epidermolysis Bullosa Human: Murine Skin Model by Gaetano Naso, Soragia Athina Gkazi, Christos Georgiadis, Vignesh Jayarajan, Joanna Jacków, Roland Fleck, Leanne Allison, Olumide Kayode Ogunbiyi, John Alexander McGrath, Dusko Ilic, Wei-Li Di, Anastasia Petrova, Waseem Qasim

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Molecular Marvels: Small Molecules Paving the Way for Enhanced Gene Therapy by Sebastian Hasselbeck, Xinlai Cheng

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Genome-wide screening identified SEC61A1 as an essential factor for mycolactone-dependent apoptosis in human premonocytic THP-1 cells. by Akira Kawashima, Mitsuo Kiriya, Junichiro En, Kazunari Tanigawa, Yasuhiro Nakamura, Yoko Fujiwara, Yuqian Luo, Keiji Maruyama, Shigekazu Watanabe, Masamichi Goto, Koichi Suzuki

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Tumor reversion: a dream or a reality by Avantika Tripathi, Anjali Kashyap, Greesham Tripathi, Joni Yadav, Rakhi Bibban, Nikita Aggarwal, Kulbhushan Thakur, Arun Chhokar, Mohit Jadli, Ashok Kumar Sah, Yeshvandra Verma, Hatem Zayed, Amjad Husain, Alok Chandra Bharti, Manoj Kumar Kashyap

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Compensatory mechanisms for methylglyoxal detoxification in experimental & clinical diabetes by Dagmar Schumacher, Jakob Morgenstern, Yoko Oguchi, Nadine Volk, Stefan Kopf, Jan Benedikt Groener, Peter Paul Nawroth, Thomas Fleming, Marc Freichel

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RNA-Seq Analysis of an Antisense Sequence Optimized for Exon Skipping in Duchenne Patients Reveals No Off-Target Effect by Claire Domenger, Marine Allais, Virginie François, Adrien Léger, Emilie Lecomte, Marie Montus, Laurent Servais, Thomas Voit, Philippe Moullier, Yann Audic, Caroline Le Guiner

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Allele-Specific CRISPR/Cas9 Correction of a Heterozygous DNM2 Mutation Rescues Centronuclear Myopathy Cell Phenotypes by Aymen Rabai, Léa Reisser, Bernardo Reina-, Kamel Mamchaoui, Belinda S. Cowling, Anne-Sophie Nicot, Jocelyn Laporte

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Context base editing for splice correction of IVSI-110 β-thalassemia by Basma Naiisseh, Panayiota L. Papasavva, Nikoletta Y. Papaioannou, Marios Tomazou, Lola Koniali, Xenia Felekis, Constantina G. Constantinou, Maria Sitarou, Soteroula Christou, Marina Kleanthous, Carsten W. Lederer, Petros Patsali

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mRNA-mediated delivery of gene editing tools to human primary muscle stem cells by Christian Stadelmann, Silvia Di Francescantonio, Andreas Marg, Stefanie Müthel, Simone Spuler, Helena Escobar

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Priming Human Repopulating Hematopoietic Stem and Progenitor Cells for Cas9/sgRNA Gene Targeting by Carsten T. Charlesworth, Joab Camarena, M. Kyle Cromer, Sriram Vaidyanathan, Rasmus O. Bak, Jason M. Carte, Jason Potter, Daniel P. Dever, Matthew H. Porteus

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Combined refinements to somatic cell nuclear transfer methods improve porcine embryo development by Thanh Quang DANG-NGUYEN, David WELLS, Seiki HARAGUCHI, Nguyen Thi MEN, Hiep Thi NGUYEN, Junko NOGUCHI, Hiroyuki KANEKO, Kazuhiro KIKUCHI

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Environmentally applied nucleic acids and proteins for purposes of engineering changes to genes and other genetic material by Jack A. Heinemann, Sophie Walker

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Engineering an adenine base editor in human embryonic stem cells with minimal DNA and RNA off-target activities by Zhenwu Zhang, Wanyu Tao, Shisheng Huang, Wenjun Sun, Yue Wang, Wen Jiang, Xingxu Huang, Chao-Po Lin

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Understanding Gene Involvement in Hepatocellular Carcinoma: Implications for Gene Therapy and Personalized Medicine by Younis MA, Harashima H

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AAV-based gene editing of type 1 collagen mutation to treat osteogenesis imperfecta by Yeon-Suk Yang, Tadatoshi Sato, Sachin Chaugule, Hong Ma, Jun Xie, Guangping Gao, Jae-Hyuck Shim

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