Antisense oligonucleotide-based therapy in human erythropoietic protoporphyria

American Journal of Human Genetics

Volumn 94, Issue 4, 2014, Pages 611-617

  Oustric, Vincent ^a   Manceau, Hana ^a   Ducamp, Sarah ^a   Soaid, Rima ^a   Karim, Zoubida ^a,b   Schmitt, Caroline ^a,b,c   Mirmiran, Arienne ^a   Peoc'H, Katell ^a   Grandchamp, Bernard ^a,b,d   Beaumont, Carole ^a,b   Lyoumi, Said ^a,e   Moreau Gaudry, François ^a,f   Guyonnet Dupérat, Véronique ^a,f   De Verneuil, Hubert ^a,f   Marie, Joëlle ^g   Puy, Herve ^a,b,c,d   Deybach, Jean Charles ^a,b,c,d   Gouya, Laurent ^a,c,e,h,i  

^a INSERM   (France)

^b UNIVERSITÉ PARIS DESCARTES   (France)

^c HÔPITAL LOUIS MOURIER   (France)

^d BICHAT HOSPITAL   (France)

^e LABORATOIRE DES SCIENCES DU CLIMAT ET DE L ENVIRONNEMENT   (France)

^f UNIVERSITÉ DE BORDEAUX   (France)

^g CNRS   (France)

^h HÔPITAL AMBROISE PARÉ   (France)

ⁱ Laboratory of Excellence GR Ex   (France)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; CIS ACTING ELEMENT; MESSENGER RNA; PROTOPORPHYRIN;

ARTICLE; BINDING SITE; CONTROLLED STUDY; ERYTHROBLAST; ERYTHROPOIETIC PROTOPORPHYRIA; EXON; FECH GENE; GENE; GENE REPRESSION; GENE SEQUENCE; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; INTRON; LYMPHOBLASTOID CELL LINE; PRIORITY JOURNAL; RNA SPLICING;

CELL LINE; FEMALE; FERROCHELATASE; HUMANS; MALE; OLIGONUCLEOTIDES, ANTISENSE; PEDIGREE; POLYMORPHISM, GENETIC; PROTOPORPHYRIA, ERYTHROPOIETIC; PROTOPORPHYRINS; RNA SPLICING; RNA, MESSENGER;

EID: 84897999586     PISSN: 00029297     EISSN: 15376605     Source Type: Journal    
DOI: 10.1016/j.ajhg.2014.02.010     Document Type: Article

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