Search Results - "gene therapies"

Engineered packaging cell line for the enhanced production of baboon-enveloped retroviral vectors by Denise Klatt, Lucia Sereni, Boya Liu, Pietro Genovese, Axel Schambach, Els Verhoeyen, David A. Williams, Christian Brendel

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Development of versatile allele-specific siRNAs able to silence all the dominant dynamin 2 mutations by Swati Dudhal, Lylia Mekzine, Bernard Prudhon, Karishma Soocheta, Bruno Cadot, Kamel Mamchaoui, Delphine Trochet, Marc Bitoun

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Stimuli-responsive drug delivery systems for head and neck cancer therapy by Jingou Liang, Bina Yang, Xuedong Zhou, Qi Han, Jing Zou, Lei Cheng

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Evidence Based Medicine and Herbal Medicine by S Akhondzadeh

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An Evalution of the Demographic and Clinical Characterictics of Patients with GM2 Gangliosidosis by Esra Er, Ebru Canda, Havva Yazıcı, Cenk Eraslan, Eser Sözmen, Sema Kalkan Uçar, Mahmut Çoker

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CRISPR-Cas9-Mediated Genome Editing Increases Lifespan and Improves Motor Deficits in a Huntington's Disease Mouse Model by Freja K. Ekman, David S. Ojala, Maroof M. Adil, Paola A. Lopez, David V. Schaffer, Thomas Gaj

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Anti-Inflammatory Therapy for Temporomandibular Joint Osteoarthritis Using mRNA Medicine Encoding Interleukin-1 Receptor Antagonist by Jia Deng, Yuta Fukushima, Kosuke Nozaki, Hideyuki Nakanishi, Erica Yada, Yuki Terai, Kenji Fueki, Keiji Itaka

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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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Expression of TGF-β variants in the airway epithelial cells exposed to Sulfur Mustard by F. Ghane zadeh, A.A. Imani Fooladi, M. Mirzaman Bafghi, M. Mirbagheri, Mohammad Reza Nourani

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Effective Prevention of Liver Fibrosis by Liver-targeted Hydrodynamic Gene Delivery of Matrix Metalloproteinase-13 in a Rat Liver Fibrosis Model by Hiroyuki Abe, Kenya Kamimura, Yuji Kobayashi, Masato Ohtsuka, Hiromi Miura, Riuko Ohashi, Takeshi Yokoo, Tsutomu Kanefuji, Takeshi Suda, Masanori Tsuchida, Yutaka Aoyagi, Guisheng Zhang, Dexi Liu, Shuji Terai

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Cell-based medicinal products approved in the European Union: current evidence and perspectives by Stefania Bellino, Anna La Salvia, Maria Francesca Cometa, Rosanna Botta

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Oncolytic adenovirus expressing interleukin-18 improves antitumor activity of dacarbazine for malignant melanoma by Yang C, Cao H, Liu N, Xu K, Ding M, Mao LJ

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Efficient Modification of the CCR5 Locus in Primary Human T Cells With megaTAL Nuclease Establishes HIV-1 Resistance by Guillermo S Romano Ibarra, Biswajit Paul, Blythe D Sather, Patrick M Younan, Karen Sommer, John P Kowalski, Malika Hale, Barry Stoddard, Jordan Jarjour, Alexander Astrakhan, Hans-Peter Kiem, David J Rawlings

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Early onset retinal dystrophies: clinical clues to diagnosis for pediatricians by Agnese Suppiej, Silvia Marino, Maria Eleonora Reffo, Veronica Maritan, Giovanna Vitaliti, Janette Mailo, Raffaele Falsaperla

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Current and upcoming methods of pharmacotherapy of obesity by Jan Bieniasz, Konrad Dendys, Marcel Kazzi, Michał Puła, Marta Wychota

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10th European immunogenicity platform open symposium on immunogenicity of biopharmaceuticals by S. Tourdot, A. Abdolzade-Bavil, J. Bessa, P. Broët, A. Fogdell-Hahn, M. Giorgi, V. Jawa, K. Kuranda, N. Legrand, S. Pattijn, J. A. Pedras-Vasconcelos, A. Rudy, P. Salmikangas, D. W. Scott, V. Snoeck, N. Smith, S. Spindeldreher, D. Kramer

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Autosomal dominant optic atrophy: A novel treatment for OPA1 splice defects using U1 snRNA adaption by Christoph Jüschke, Thomas Klopstock, Claudia B. Catarino, Marta Owczarek-Lipska, Bernd Wissinger, John Neidhardt

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Case report: Altered pre-mRNA splicing caused by intronic variant c.1499 + 1G > A in the SLC4A4 gene by Yan Liu, Wenchao Sheng, Wenchao Sheng, Wenchao Sheng, Jinying Wu, Jinying Wu, Jie Zheng, Jie Zheng, Xiufang Zhi, Xiufang Zhi, Xiufang Zhi, Shuyue Zhang, Shuyue Zhang, Shuyue Zhang, Chunyu Gu, Chunyu Gu, Chunyu Gu, Detong Guo, Detong Guo, Detong Guo, Wenhong Wang

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Microfluidic Formulation of DNA-Loaded Multicomponent Lipid Nanoparticles for Gene Delivery by Erica Quagliarini, Serena Renzi, Luca Digiacomo, Francesca Giulimondi, Barbara Sartori, Heinz Amenitsch, Valentina Tassinari, Laura Masuelli, Roberto Bei, Lishan Cui, Junbiao Wang, Augusto Amici, Cristina Marchini, Daniela Pozzi, Giulio Caracciolo

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Gauging the role and impact of drug interactions and repurposing in neurodegenerative disorders by Dharmendra Kumar Khatri, Amey Kadbhane, Monica Patel, Shweta Nene, Srividya Atmakuri, Saurabh Srivastava, Shashi Bala Singh

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