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Results: 1 to 20 of 108

1.

Metabolic correction of congenital erythropoietic porphyria with iPSCs free of reprogramming factors.

Bedel A, Taillepierre M, Guyonnet-Duperat V, Lippert E, Dubus P, Dabernat S, Mautuit T, Cardinaud B, Pain C, Rousseau B, Lalanne M, Ged C, Duchartre Y, Richard E, de Verneuil H, Moreau-Gaudry F.

Am J Hum Genet. 2012 Jul 13;91(1):109-21. doi: 10.1016/j.ajhg.2012.05.026.

PMID:
22795135
[PubMed - indexed for MEDLINE]
Free PMC Article
2.

Lentivirus-mediated gene transfer of uroporphyrinogen III synthase fully corrects the porphyric phenotype in human cells.

Géronimi F, Richard E, Lamrissi-Garcia I, Lalanne M, Ged C, Redonnet-Vernhet I, Moreau-Gaudry F, de Verneuil H.

J Mol Med (Berl). 2003 May;81(5):310-20. Epub 2003 Apr 30.

PMID:
12721665
[PubMed - indexed for MEDLINE]
3.

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

Kauppinen R, Glass IA, Aizencang G, Astrin KH, Atweh GF, Desnick RJ.

Mol Genet Metab. 1998 Sep;65(1):10-7.

PMID:
9787090
[PubMed - indexed for MEDLINE]
4.

Effective gene therapy of mice with congenital erythropoietic porphyria is facilitated by a survival advantage of corrected erythroid cells.

Robert-Richard E, Moreau-Gaudry F, Lalanne M, Lamrissi-Garcia I, Cario-André M, Guyonnet-Dupérat V, Taine L, Ged C, de Verneuil H.

Am J Hum Genet. 2008 Jan;82(1):113-24. doi: 10.1016/j.ajhg.2007.09.007.

PMID:
18179890
[PubMed - indexed for MEDLINE]
Free PMC Article
5.

Correction of deficient CD34+ cells from peripheral blood after mobilization in a patient with congenital erythropoietic porphyria.

Mazurier F, Géronimi F, Lamrissi-Garcia I, Morel C, Richard E, Ged C, Fontanellas A, Moreau-Gaudry F, Morey M, de Verneuil H.

Mol Ther. 2001 Mar;3(3):411-7.

PMID:
11273784
[PubMed - indexed for MEDLINE]
Free Article
6.

Modeling of congenital erythropoietic porphyria by RNA interference: a new tool for preclinical gene therapy evaluation.

Robert-Richard E, Lalanne M, Lamrissi-Garcia I, Guyonnet-Duperat V, Richard E, Pitard V, Mazurier F, Moreau-Gaudry F, Ged C, de Verneuil H.

J Gene Med. 2010 Aug;12(8):637-46. doi: 10.1002/jgm.1478.

PMID:
20586119
[PubMed - indexed for MEDLINE]
7.

Gene transfer of the uroporphyrinogen III synthase cDNA into haematopoietic progenitor cells in view of a future gene therapy in congenital erythropoietic porphyria.

Mazurier F, Moreau-Gaudry F, Salesse S, Barbot C, Ged C, Reiffers J, de Verneuil H.

J Inherit Metab Dis. 1997 Jun;20(2):247-57.

PMID:
9211197
[PubMed - indexed for MEDLINE]
8.

[Successful gene therapy of mice with congenital erythropoietic porphyria].

de Verneuil H, Robert-Richard E, Ged C, Mazurier F, Richard E, Moreau-Gaudry F.

Med Sci (Paris). 2008 Jun-Jul;24(6-7):615-20. doi: 10.1051/medsci/20082467615. French.

PMID:
18601879
[PubMed - indexed for MEDLINE]
Free Article
9.

Successful treatment of congenital erythropoietic porphyria using matched unrelated hematopoietic stem cell transplantation.

Martinez Peinado C, Díaz de Heredia C, To-Figueras J, Arias-Santiago S, Nogueras P, Elorza I, Olivé T, Bádenas C, Moreno MJ, Tercedor J, Herrero C.

Pediatr Dermatol. 2013 Jul-Aug;30(4):484-9. doi: 10.1111/pde.12117. Epub 2013 Apr 5.

PMID:
23557135
[PubMed - indexed for MEDLINE]
10.

A knock-in mouse model of congenital erythropoietic porphyria.

Ged C, Mendez M, Robert E, Lalanne M, Lamrissi-Garcia I, Costet P, Daniel JY, Dubus P, Mazurier F, Moreau-Gaudry F, de Verneuil H.

Genomics. 2006 Jan;87(1):84-92. Epub 2005 Nov 28.

PMID:
16314073
[PubMed - indexed for MEDLINE]
Free Article
11.

Successful match-unrelated donor bone marrow transplantation for congenital erythropoietic porphyria (Günther disease).

Dupuis-Girod S, Akkari V, Ged C, Galambrun C, Kebaïli K, Deybach JC, Claudy A, Geburher L, Philippe N, de Verneuil H, Bertrand Y.

Eur J Pediatr. 2005 Feb;164(2):104-7. Epub 2004 Nov 20.

PMID:
15703981
[PubMed - indexed for MEDLINE]
12.

Therapeutic potential of proteasome inhibitors in congenital erythropoietic porphyria.

Blouin JM, Duchartre Y, Costet P, Lalanne M, Ged C, Lain A, Millet O, de Verneuil H, Richard E.

Proc Natl Acad Sci U S A. 2013 Nov 5;110(45):18238-43. doi: 10.1073/pnas.1314177110. Epub 2013 Oct 21.

PMID:
24145442
[PubMed - indexed for MEDLINE]
Free PMC Article
13.

Correction of the enzyme defect in cultured congenital erythropoietic porphyria disease cells by retrovirus-mediated gene transfer.

Moreau-Gaudry F, Ged C, Barbot C, Mazurier F, Boiron JM, Bensidhoum M, Reiffers J, de Verneuil H.

Hum Gene Ther. 1995 Jan;6(1):13-20.

PMID:
7703283
[PubMed - indexed for MEDLINE]
14.

Generation of human induced pluripotent stem cells bearing an anti-HIV transgene by a lentiviral vector carrying an internal murine leukemia virus promoter.

Kamata M, Liu S, Liang M, Nagaoka Y, Chen IS.

Hum Gene Ther. 2010 Nov;21(11):1555-67. doi: 10.1089/hum.2010.050. Epub 2010 Oct 6.

PMID:
20524893
[PubMed - indexed for MEDLINE]
Free PMC Article
15.

Molecular genetics of congenital erythropoietic porphyria.

Desnick RJ, Glass IA, Xu W, Solis C, Astrin KH.

Semin Liver Dis. 1998;18(1):77-84. Review.

PMID:
9516681
[PubMed - indexed for MEDLINE]
16.

[Congenital erythropoeietic porphyria treated by haematopoietic stem cell allograft].

Lebreuilly-Sohyer I, Morice A, Acher A, Dompmartin A, Clement C, de Verneuil H, Ged C, Leroy D, Verneuil L.

Ann Dermatol Venereol. 2010 Oct;137(10):635-9. doi: 10.1016/j.annder.2010.06.018. Epub 2010 Aug 13. French.

PMID:
20932444
[PubMed - indexed for MEDLINE]
17.

Metabolic correction of congenital erythropoietic porphyria by retrovirus-mediated gene transfer into Epstein-Barr virus-transformed B-cell lines.

Moreau-Gaudry F, Mazurier F, Bensidhoum M, Ged C, de Verneuil H.

Blood. 1995 Mar 15;85(6):1449-53.

PMID:
7888667
[PubMed - indexed for MEDLINE]
Free Article
18.

ALAS2 acts as a modifier gene in patients with congenital erythropoietic porphyria.

To-Figueras J, Ducamp S, Clayton J, Badenas C, Delaby C, Ged C, Lyoumi S, Gouya L, de Verneuil H, Beaumont C, Ferreira GC, Deybach JC, Herrero C, Puy H.

Blood. 2011 Aug 11;118(6):1443-51. doi: 10.1182/blood-2011-03-342873. Epub 2011 Jun 7.

PMID:
21653323
[PubMed - indexed for MEDLINE]
Free Article
19.

[A model of congenital erythropoietic porphyria for gene transfer in hematopoietic cells].

de Verneuil H, Moreau-Gaudry F, Ged C.

Transfus Clin Biol. 1997;4(3):263-6. French.

PMID:
9264783
[PubMed - indexed for MEDLINE]
20.

A molecular study of congenital erythropoietic porphyria in cattle.

Agerholm JS, Thulstrup PW, Bjerrum MJ, Bendixen C, Jørgensen CB, Fredholm M.

Anim Genet. 2012 Apr;43(2):210-5. doi: 10.1111/j.1365-2052.2011.02228.x. Epub 2011 Jul 21.

PMID:
22404357
[PubMed - indexed for MEDLINE]

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