Current and innovative emerging therapies for porphyrias with hepatic involvement.

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第一作者： Antonio,Fontanellas

第一单位： Hepatology Program, Center for Applied Medical Research (CIMA), University of Navarra, Spain; Instituto de Investigación Sanitaria de Navarra (IdiSNA), Pamplona, Spain; Centro de Investigación Biomédica en Red de Enfermedades Hepáticas y Digestivas (CIBEREHD), Spain. Instituto de Salud Carlos III, Spain. Electronic address: afontanellas@unav.es.

作者： Antonio,Fontanellas ^[1] ; Matías A,Ávila ^[2] ; Karl E,Anderson ^[3] ; Jean-Charles,Deybach ^[4]

作者单位： Hepatology Program, Center for Applied Medical Research (CIMA), University of Navarra, Spain; Instituto de Investigación Sanitaria de Navarra (IdiSNA), Pamplona, Spain; Centro de Investigación Biomédica en Red de Enfermedades Hepáticas y Digestivas (CIBEREHD), Spain. Instituto de Salud Carlos III, Spain. Electronic address: afontanellas@unav.es. ^[1] Hepatology Program, Center for Applied Medical Research (CIMA), University of Navarra, Spain; Instituto de Investigación Sanitaria de Navarra (IdiSNA), Pamplona, Spain; Centro de Investigación Biomédica en Red de Enfermedades Hepáticas y Digestivas (CIBEREHD), Spain. Instituto de Salud Carlos III, Spain. ^[2] Porphyria Laboratory & Center, Departments of Preventive Medicine and Community Health, and Internal Medicine (Division of Gastroenterology), University of Texas Medical Branch, Galveston, TX, USA. ^[3] CRMR Porphyries France, Assistance Publique-Hôpitaux de Paris (AP-HP), University Denis Diderot Paris 7, France; European Porphyria Network (EPNET). ^[4]

关键词 Antisense oligonucleotide therapy Direct-acting antiviral agents and PCT Hematopoietic-stem-cell-based gene therapy Heme synthesis Lentiviral vectors Liver gene therapy Melanogenesis stimulator Porphyrias RNA interference technology mRNA therapy