Molecular Therapy
Volume 28, Issue 2, 5 February 2020, Pages 677-689
Original ArticleA Pharmacological Chaperone Therapy for Acute Intermittent Porphyria
Under a Creative Commons license
open access
Graphical Abstract
Keywords
acute intermittent porphyria
hydroxymethylbilane synthase
protein stabilization
pharmacological chaperones
autosomal dominant disease
heme synthesis
inborn error of metabolism
mouse model of AIP
Cited by (0)
© 2019 The Authors.