Original paper
Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria
Abstract
Background & AimsAcute intermittent porphyria (AIP) results from porphobilinogen deaminase (PBGD) haploinsufficiency, which leads to hepatic over-production of the neurotoxic heme precursors porphobilinogen (PBG) and delta-aminolevulinic acid (ALA) and the occurrence of neurovisceral attacks. Severe AIP is a devastating disease that can only be corrected by liver transplantation. Gene therapy represents a promising curative option. The objective...
Paper Details
Title
Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria
Published Date
Oct 1, 2016
Journal
Volume
65
Issue
4
Pages
776 - 783
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Notes
History