Search Results - "gene therapies"

Evidence That a Peptide-Drug/p53 Gene Complex Promotes Cognate Gene Expression and Inhibits the Viability of Glioblastoma Cells by Ana Neves, Tânia Albuquerque, Rúben Faria, Cecília R. A. Santos, Eric Vivès, Prisca Boisguérin, Diana Carneiro, Daniel F. Bruno, Maria D. Pavlaki, Susana Loureiro, Ângela Sousa, Diana Costa

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Case report: First case report of an Emirati child with a novel gene variant causing aromatic L-amino acid decarboxylase deficiency by Mohamed O. E. Babiker, Manju A. Kurian, Manju A. Kurian, Jehan Suleiman, Jehan Suleiman

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In vivo genome editing using 244-cis LNPs and low-dose AAV achieves therapeutic threshold in hemophilia A mice by Jeong Pil Han, Yeji Lee, Jeong Hyeon Lee, Hye Yoon Chung, Geon Seong Lee, Yu Ri Nam, Myeongjin Choi, Kyoung-Sik Moon, Haeshin Lee, Hyukjin Lee, Su Cheong Yeom

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Elastin-Derived VGVAPG Fragment Decorated Cell-Penetrating Peptide with Improved Gene Delivery Efficacy by Wen-Juan Shen, Duo-Mei Tian, Le Fu, Biao Jin, Yu Liu, Yun-Sheng Xu, Yong-Bin Ye, Xiao-Bo Wang, Xiao-Jun Xu, Chun Tang, Fang-Ping Li, Chun-Fei Wang, Gang Wu, Le-Ping Yan

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Pompe Disease: Early Diagnosis and Early Treatment Make a Difference by Yin-Hsiu Chien, Wuh-Liang Hwu, Ni-Chung Lee

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Clonal Dominance With Retroviral Vector Insertions Near the ANGPT1 and ANGPT2 Genes in a Human Xenotransplant Mouse Model by Reinhard Haemmerle, Ruhi Phaltane, Michael Rothe, Simon Schröder, Axel Schambach, Thomas Moritz, Ute Modlich

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Effect On Alpha Hemoglobin Chain Expression In K562 Cell Line by Sh Alizadeh, S Kaviani, M Soleimani, AA Pourfathollah, N Amirizadeh, F Kouhkan, S Abroun, M Noruzinia

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The Use of a Barley-Based Well to Define Cationic Betaglucan to Study Mammalian Cell Toxicity Associated with Interactions with Biological Structures by Malgorzata Tymecka, Katarzyna Hac-Wydro, Magdalena Obloza, Piotr Bonarek, Kamil Kaminski

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Transfection by Electroporation of Cancer and Primary Cells Using Nanosecond and Microsecond Electric Fields by Eivina Radzevičiūtė, Veronika Malyško-Ptašinskė, Jurij Novickij, Vitalij Novickij, Irutė Girkontaitė

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Long-term muscle-specific overexpression of DOK7 in mice using AAV9-tMCK-DOK7 by Yu-Ting Huang, Hannah R. Crick, Helena Chaytow, Dinja van der Hoorn, Abrar Alhindi, Ross A. Jones, Ralph D. Hector, Stuart R. Cobb, Thomas H. Gillingwater

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Comprehensive evaluation of the child with global developmental delays or intellectual disability by Abdullah Nasser Aldosari, T. Saeed Aldosari

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Generation of Hutat2:Fc Knockin Primary Human Monocytes Using CRISPR/Cas9 by Bowen Wang, Jiahui Zuo, Wenzhen Kang, Qianqi Wei, Jianhui Li, Chunfu Wang, Zhihui Liu, Yuanan Lu, Yan Zhuang, Bianli Dang, Qing Liu, Wen Kang, Yongtao Sun

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In Vivo Knockout of the Vegfa Gene by Lentiviral Delivery of CRISPR/Cas9 in Mouse Retinal Pigment Epithelium Cells by Andreas Holmgaard, Anne Louise Askou, Josephine Natalia Esther Benckendorff, Emil Aagaard Thomsen, Yujia Cai, Toke Bek, Jacob Giehm Mikkelsen, Thomas J. Corydon

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Suppression of IGF1R in Melanoma Cells by an Adenovirus-Mediated One-Step Knockdown System by Haoran Xin, Mingxing Lei, Zhihui Zhang, Jie Li, Hao Zhang, Xinwei Luo, Aoyun Wang, Fang Deng

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A Tumor-specific MicroRNA Recognition System Facilitates the Accurate Targeting to Tumor Cells by Magnetic Nanoparticles by Yingting Yu, Yi Yao, Hao Yan, Rui Wang, Zhenming Zhang, Xiaodan Sun, Lingyun Zhao, Xiang Ao, Zhen Xie, Qiong Wu

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Design, Development, and Testing of a Device for Gene Electrotransfer to Skin Cells In Vivo by Aleksandra Cvetkoska, Janja Dermol-Černe, Damijan Miklavčič, Simona Kranjc Brezar, Boštjan Markelc, Gregor Serša, Matej Reberšek

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Management of ß-thalassemia - Consensus and controversies! by Mamta V. Manglani, Pranoti S. Kini

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siRNA-Loaded Hydroxyapatite Nanoparticles for <i>KRAS</i> Gene Silencing in Anti-Pancreatic Cancer Therapy by Dandan Luo, Xiaochun Xu, M. Zubair Iqbal, Qingwei Zhao, Ruibo Zhao, Jabeen Farheen, Quan Zhang, Peiliang Zhang, Xiangdong Kong

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Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma^®) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Report... by Rosanna Ruggiero, Nunzia Balzano, Maria Maddalena Nicoletti, Gabriella di Mauro, Federica Fraenza, Maria Rosaria Campitiello, Francesco Rossi, Annalisa Capuano

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RNA Combined with Nanoformulation to Advance Therapeutic Technologies by Eduarda Santos Lima, Déborah dos Santos, Atena Liriel Souza, Maria Eduarda Macedo, Mariana Evangelista Bandeira, Sérgio Santos Silva Junior, Bianca Sampaio Dotto Fiuza, Vinicius Pinto Costa Rocha, Larissa Moraes dos Santos Fonseca, Danielle Devequi Gomes Nunes, Katharine Valéria Saraiva Hodel, Bruna Aparecida Souza Machado

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