Congenital erythropoietic porphyria: Prolonged high-level expression and correction of the heme biosynthetic defect by retroviral-mediated gene transfer into porphyric and erythroid cells

Molecular Genetics and Metabolism

Volumn 65, Issue 1, 1998, Pages 10-17

  Kauppinen, R ^a   Glass, I A ^a   Aizencang, G ^a   Astrin, K H ^a   Atweh, G F ^a   Desnick, R J ^a  

^a MOUNT SINAI SCHOOL OF MEDICINE   (United States)

Author keywords

Congenital erythropoietic porphyria (CEP); Gene therapy; Porphyria; Retroviral gene transfer; Uroporphyrinogen III synthase (UROS)

Indexed keywords

UROPORPHYRINOGEN III SYNTHASE;

ARTICLE; AUTOSOMAL RECESSIVE DISORDER; BONE MARROW TRANSPLANTATION; CELL STIMULATION; CONGENITAL ERYTHROPOIETIC PORPHYRIA; ERYTHROID CELL; ERYTHROLEUKEMIA CELL; FIBROBLAST; GENE THERAPY; GENE TRANSFER; HEME SYNTHESIS; HUMAN; HUMAN CELL; PRIORITY JOURNAL; PROTEIN EXPRESSION; RETROVIRUS; SIGNAL TRANSDUCTION; SKIN MANIFESTATION;

UNIDENTIFIED RETROVIRUS;

EID: 0031769899     PISSN: 10967192     EISSN: None     Source Type: Journal    
DOI: 10.1006/mgme.1998.2739     Document Type: Article

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