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

Published 2016 View Full Article

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Title
Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria
Authors
Keywords
Gene therapy, Acute intermittent porphyria, AAV/PBGD, Adeno-associated virus
Journal
JOURNAL OF HEPATOLOGY
Volume 65, Issue 4, Pages 776-783
Publisher
Elsevier BV
Online
2016-05-18
DOI
10.1016/j.jhep.2016.05.012

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