Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria

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Bibliographic Details
Published in	Journal of hepatology Vol. 65; no. 4; pp. 776 - 783
Main Authors	D’Avola, Delia, López-Franco, Esperanza, Sangro, Bruno, Pañeda, Astrid, Grossios, Nadina, Gil-Farina, Irene, Benito, Alberto, Twisk, Jaap, Paz, María, Ruiz, Juan, Schmidt, Manfred, Petry, Harald, Harper, Pauline, de Salamanca, Rafael Enríquez, Fontanellas, Antonio, Prieto, Jesús, González-Aseguinolaza, Gloria
Format	Journal Article
Language	English
Published	Netherlands Elsevier B.V 01.10.2016
Subjects	AAV/PBGD Acute intermittent porphyria Adeno-associated virus Aminolevulinic Acid Animals Gastroenterology and Hepatology Gene therapy Genetic Therapy Humans Hydroxymethylbilane Synthase Medicin och hälsovetenskap Mice Porphyria, Acute Intermittent ALAS 36-Item short form health survey AIP PBGD adeno-associated virus BAI Beck anxiety inventory SF-36 Beck depression inventory II PBG rAAV2/5- PBGD acute intermittent porphyria AAV/PBGD ALA AAV porphobilinogen deaminase aminolevulinic acid BDI-II 5-aminolevulinic acid synthase Gene therapy PCR polymerase chain reaction porphobilinogen recombinant adeno-associated vector expressing PBGD Adeno-associated virus rAAV2/5-PBGD Acute intermittent porphyria
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