Search Results - "gene therapies"

Transferrin Non-Viral Gene Therapy for Treatment of Retinal Degeneration by Karine Bigot, Pauline Gondouin, Romain Bénard, Pierrick Montagne, Jenny Youale, Marie Piazza, Emilie Picard, Thierry Bordet, Francine Behar-Cohen

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Optimization of Ultrasound-mediated Anti-angiogenic Cancer Gene Therapy by Hiroko Fujii, Pratiek Matkar, Christine Liao, Dmitriy Rudenko, Paul JH Lee, Michael A Kuliszewski, Gerald J Prud'homme, Howard Leong-Poi

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Current State of Human Gene Therapy: Approved Products and Vectors by Aladdin Y. Shchaslyvyi, Svitlana V. Antonenko, Maksym G. Tesliuk, Gennadiy D. Telegeev

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Delivery of RNAs to Specific Organs by Lipid Nanoparticles for Gene Therapy by Kelly Godbout, Jacques P. Tremblay

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Gene Therapy, Early Promises, Subsequent Problems, and Recent Breakthroughs by Saeideh Razi Soofiyani, Behzad Baradaran, Farzaneh Lotfipour, Tohid Kazemi, Leila Mohammadnejad

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A route to engineer a genome editor for gene therapy by Wenxia Yu, Yunbo Qiao

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Market Access Challenges and Solutions in Cell and Gene Therapy in The Netherlands by Rimma Velikanova, Sharon Wolters, Hinko S. Hofstra, Maarten J. Postma, Cornelis Boersma

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Introduction to Special Issue: A New Paradigm of Gene Therapy by Keiji Itaka

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Gene therapy: a promising approach for neuroprotection in Parkinson's disease? by Pamela Valdés, Bernard Laurent Schneider

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Opportunities and challenges for applying model‐informed drug development approaches to gene therapies by Artur Belov, Kimberly Schultz, Richard Forshee, Million A. Tegenge

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An overview of health technology assessments of gene therapies with the focus on cost-effectiveness models by Michał Pochopień, Ewelina Paterak, Emilie Clay, Justyna Janik, Samuel Aballea, Małgorzata Biernikiewicz, Mondher Toumi

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In Vivo and Ex Vivo Gene Therapy for Inherited and Non-Inherited Disorders

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Advances in ultrasound-targeted microbubble-mediated gene therapy for liver fibrosis by Cuiyuan Huang, Hong Zhang, Ruidan Bai

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Multicomponent nanoparticles as nonviral vectors for the treatment of Fabry disease by gene therapy by Ruiz de Garibay AP, Delgado D, del Pozo-Rodríguez A, Solinís MA, Gascón AR

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Non-Viral Delivery of RNA Gene Therapy to the Central Nervous System by Ellen S. Hauck, James G. Hecker

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Targeting transgenic proteins to alpha granules for platelet-directed gene therapy by Vanessa M.A. Woods, Lisette J. Latorre-Rey, Franziska Schenk, Marcel G.E. Rommel, Thomas Moritz, Ute Modlich

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A systems pharmacology model for gene therapy in sickle cell disease by Bo Zheng, Lucia Wille, Karsten Peppel, David Hagen, Andrew Matteson, Jeffrey Ahlers, James Schaff, Fei Hua, Theresa Yuraszeck, Enoch Cobbina, Joshua F. Apgar, John M. Burke, John Roberts, Raibatak Das

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Gene Therapy for Systemic or Organ Specific Delivery of Manganese Superoxide Dismutase by Joel S. Greenberger, Amitava Mukherjee, Michael W. Epperly

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Predicting determinants of long-term HIV control with gene therapy strategies by A.L. Hill, L. Hu, L.E. Hogan, M.A. Nowak, T.J. Henrich

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

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