Erythropoietic porphyrias: Animal models and update in gene-based therapies

Current Gene Therapy

Volumn 8, Issue 3, 2008, Pages 176-186

  Richard, Emmanuel ^a,b,c   Robert Richard, Elodie ^a,b   Ged, Cécile ^a,b,c   Moreau Gaudry, François ^a,b,c   de Verneuil, Hubert ^a,b,c  

^a INSERM   (France)

^b UNIVERSITÉ DE BORDEAUX   (France)

^c HÔPITAL PELLEGRIN   (France)

Author keywords

Congenital erythropoietic porphyria; Hematopoietic stem cell; Hepatoerythropoietic porphyria; Lentriviral vector; MGMT; Mouse model; Protoporphyria; Red blood cells; Retroviral vector

Indexed keywords

5 AMINOLEVULINATE SYNTHASE; BUSULFAN; CARMUSTINE; FERROCHELATASE; HEME; LENTIVIRUS VECTOR; PORPHOBILINOGEN SYNTHASE; RETROVIRUS VECTOR; UROPORPHYRINOGEN DECARBOXYLASE; UROPORPHYRINOGEN III SYNTHASE;

ANIMAL MODEL; BONE MARROW TRANSPLANTATION; CD34 SELECTION; CELL TYPE; CLINICAL FEATURE; CLINICAL TRIAL; CONGENITAL ERYTHROPOIETIC PORPHYRIA; DISEASE CLASSIFICATION; DISEASE MODEL; ENZYME ACTIVITY; ENZYME DEFICIENCY; EX VIVO GENE TRANSFER; GENE THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEME SYNTHESIS; HUMAN; LIVER DISEASE; LIVER DYSFUNCTION; NONHUMAN; PATHOPHYSIOLOGY; PORPHYRIA CUTANEA TARDA; REVIEW; SKIN MANIFESTATION; SOMATIC GENE THERAPY; STEM CELL GENE THERAPY; STEM CELL MOBILIZATION; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY;

ANIMALIA; MURINAE; MUS;

EID: 48249103159     PISSN: 15665232     EISSN: None     Source Type: Journal    
DOI: 10.2174/156652308784746477     Document Type: Review

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