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Amyloid
The Journal of Protein Folding Disorders
Volume 23, 2016 - Issue 2
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Original Article

Preclinical evaluation of RNAi as a treatment for transthyretin-mediated amyloidosis

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Pages 109-118 | Received 12 Nov 2015, Accepted 29 Feb 2016, Published online: 31 Mar 2016

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Gita A. Pathak, Antonella De Lillo, Frank R. Wendt, Flavio De Angelis, Dora Koller, Brenda Cabrera Mendoza, Daniel Jacoby, Edward J. Miller, Joel N. Buxbaum & Renato Polimanti. (2022) The integration of genetically-regulated transcriptomics and electronic health records highlights a pattern of medical outcomes related to increased hepatic transthyretin expression. Amyloid 29:2, pages 110-119.
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R. Spoladore, G. Falasconi, M. Marcatti, S. Di Maio, G. Fiore, M. Slavich, A. Margonato, A. Turco & G. Fragasso. (2021) Advances in pharmacotherapy for cardiac amyloidosis. Expert Opinion on Pharmacotherapy 22:4, pages 469-481.
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Marco Luigetti, Angela Romano, Andrea Di Paolantonio, Giulia Bisogni & Mario Sabatelli. (2020) Diagnosis and Treatment of Hereditary Transthyretin Amyloidosis (hATTR) Polyneuropathy: Current Perspectives on Improving Patient Care. Therapeutics and Clinical Risk Management 16, pages 109-123.
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Xiaohua Guo, Zhaowen Liu, Yizhou Zheng, Yamei Li, Linfu Li, Hai Liu, Zhixi Chen & Longhuo Wu. (2020) Review on the Structures and Activities of Transthyretin Amyloidogenesis Inhibitors. Drug Design, Development and Therapy 14, pages 1057-1081.
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Kamran M. Miah, Stephen C. Hyde & Deborah R. Gill. (2019) Emerging gene therapies for cystic fibrosis. Expert Review of Respiratory Medicine 13:8, pages 709-725.
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Prakash Harikrishnan, Srikanth Yandrapalli, Wilbert S. Aronow, Gregg M. Lanier & Diwakar Jain. (2019) Novel drug therapies for cardiac amyloidosis. Expert Opinion on Investigational Drugs 28:6, pages 497-499.
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Junyi Yang. (2019) Patisiran for the treatment of hereditary transthyretin-mediated amyloidosis. Expert Review of Clinical Pharmacology 12:2, pages 95-99.
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Richard M. Giadone, Jessica D. Rosarda, Prithvi Reddy Akepati, Arianne C. Thomas, Batbold Boldbaatar, Marianne F. James, Andrew A. Wilson, Vaishali Sanchorawala, Lawreen H. Connors, John L. Berk, R. Luke Wiseman & George J. Murphy. (2018) A library of ATTR amyloidosis patient-specific induced pluripotent stem cells for disease modelling and in vitro testing of novel therapeutics. Amyloid 25:3, pages 148-155.
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Timofei S Zatsepin, Yuri V Kotelevtsev & Victor Koteliansky. (2016) Lipid nanoparticles for targeted siRNA delivery – going from bench to bedside. International Journal of Nanomedicine 11, pages 3077-3086.
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Ying K. Tam, Thomas D. Madden & Michael J. Hope. (2016) Pieter Cullis’ quest for a lipid-based, fusogenic delivery system for nucleic acid therapeutics: success with siRNA so what about mRNA?. Journal of Drug Targeting 24:9, pages 774-779.
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Paula Gonçalves, Helena Martins, Susete Costelha, Luis F. Maia & Maria Joao Saraiva. (2016) Efficiency of silencing RNA for removal of transthyretin V30M in a TTR leptomeningeal animal model. Amyloid 23:4, pages 249-253.
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