Erythroid-Progenitor-Targeted Gene Therapy Using Bifunctional TFR1 Ligand-Peptides in Human Erythropoietic Protoporphyria

Erythropoietic protoporphyria (EPP) is a hereditary disease characterized by a deficiency in ferrochelatase (FECH) activity. FECH activity is responsible for the accumulation of protoporphyrin IX (PPIX). Without etiopathogenic treatment, EPP manifests as severe photosensitivity. 95% of affected indi...

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Veröffentlicht in:Am J Hum Genet
Hauptverfasser: Mirmiran, Arienne, Schmitt, Caroline, Lefebvre, Thibaud, Manceau, Hana, Daher, Raêd, Oustric, Vincent, Poli, Antoine, Lacapère, Jean-Jacques, Moulouel, Boualem, Puy, Hervé, Karim, Zoubida, Peoc'h, Katell, Lenglet, Hugo, Simonin, Sylvie, Deybach, Jean-Charles, Nicolas, Gaël, Gouya, Laurent
Format: Artigo
Sprache:Inglês
Veröffentlicht: Elsevier 2019
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Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6369449/
https://ncbi.nlm.nih.gov/pubmed/30712775
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ajhg.2018.12.021
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