Delivery of oligonucleotides to bone marrow to modulate ferrochelatase splicing in a mouse model of erythropoietic protoporphyria

Erythropoietic protoporphyria (EPP) is a rare genetic disease in which patients experience acute phototoxic reactions after sunlight exposure. It is caused by a deficiency in ferrochelatase (FECH) in the heme biosynthesis pathway. Most patients exhibit a loss-of-function mutation in trans to an alle...

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Detalhes bibliográficos
Publicado no:Nucleic Acids Res
Main Authors: Halloy, François, Iyer, Pavithra S, Ćwiek, Paulina, Ghidini, Alice, Barman-Aksözen, Jasmin, Wildner-Verhey van Wijk, Nicole, Theocharides, Alexandre P A, Minder, Elisabeth I, Schneider-Yin, Xiaoye, Schümperli, Daniel, Hall, Jonathan
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2020
Assuntos:
Chemical Biology and Nucleic Acid Chemistry
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7229840/
https://ncbi.nlm.nih.gov/pubmed/32313951
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkaa229
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