通过逆转录病毒基因转移纠正先天性红细胞生成性卟啉病中骨髓细胞的酶缺陷。 - Suppr

Correction of the enzyme deficit of bone marrow cells in congenital erythropoietic porphyria by retroviral gene transfer.

作者信息

Moreau-Gaudry F, Barbot C, Mazurier F, Mahon F X, Reiffers J, Ged C, de Verneuil H

机构信息

Laboratoire de Biochimie Médicale et Biologie Moléculaire, UPR-ES, Pathologie Moléculaire et Thérapie Génique, Bordeaux, France.

出版信息

Hematol Cell Ther. 1996 Apr;38(2):217-20. doi: 10.1007/s00282-996-0217-3.

DOI:10.1007/s00282-996-0217-3

PMID:8932010

Abstract

摘要

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Correction of the enzyme deficit of bone marrow cells in congenital erythropoietic porphyria by retroviral gene transfer.通过逆转录病毒基因转移纠正先天性红细胞生成性卟啉病中骨髓细胞的酶缺陷。

Hematol Cell Ther. 1996 Apr;38(2):217-20. doi: 10.1007/s00282-996-0217-3.

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

Transfus Clin Biol. 1997;4(3):263-6. doi: 10.1016/s1246-7820(97)80050-x.

Correction of deficient CD34+ cells from peripheral blood after mobilization in a patient with congenital erythropoietic porphyria.先天性红细胞生成性卟啉病患者动员后外周血中CD34+细胞缺陷的纠正

Mol Ther. 2001 Mar;3(3):411-7. doi: 10.1006/mthe.2001.0270.

Correction of the enzyme defect in cultured congenital erythropoietic porphyria disease cells by retrovirus-mediated gene transfer.通过逆转录病毒介导的基因转移纠正培养的先天性红细胞生成性卟啉病细胞中的酶缺陷。

Hum Gene Ther. 1995 Jan;6(1):13-20. doi: 10.1089/hum.1995.6.1-13.

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

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

Compound heterozygosity for a premature termination codon and missense mutation in the exon 10 of the uroporphyrinogen III cosynthase gene causes a severe phenotype of congenital erythropoietic porphyria.尿卟啉原III同合酶基因第10外显子中一个过早终止密码子和错义突变的复合杂合性导致先天性红细胞生成性卟啉症的严重表型。

J Eur Acad Dermatol Venereol. 2009 Apr;23(4):470-1. doi: 10.1111/j.1468-3083.2008.02905.x. Epub 2008 Jul 16.

Congenital erythropoietic porphyria: prolonged high-level expression and correction of the heme biosynthetic defect by retroviral-mediated gene transfer into porphyric and erythroid cells.先天性红细胞生成性卟啉病：通过逆转录病毒介导的基因转移至卟啉病和红系细胞中实现血红素生物合成缺陷的长期高水平表达及纠正

Mol Genet Metab. 1998 Sep;65(1):10-7. doi: 10.1006/mgme.1998.2739.

[Congenital erythropoietic porphyria. Apropos of a fatal case in the neonatal period due to acute hemolysis with hepatic failure].

Arch Pediatr. 1995 Aug;2(8):755-61. doi: 10.1016/0929-693x(96)81246-2.

Identification of mutations in the uroporphyrinogen III synthase gene in a Thai girl patient with congenital erythropoietic porphyria.一名患有先天性红细胞生成性卟啉症的泰国女童患者尿卟啉原III合酶基因突变的鉴定。

Int J Lab Hematol. 2015 Apr;37(2):e44-7. doi: 10.1111/ijlh.12282. Epub 2014 Aug 5.

A knock-in mouse model of congenital erythropoietic porphyria.先天性红细胞生成性卟啉病的基因敲入小鼠模型。

Genomics. 2006 Jan;87(1):84-92. doi: 10.1016/j.ygeno.2005.08.018. Epub 2005 Nov 28.

引用本文的文献

Lentivirus-mediated gene transfer of uroporphyrinogen III synthase fully corrects the porphyric phenotype in human cells.慢病毒介导的尿卟啉原III合酶基因转移可完全纠正人类细胞中的卟啉症表型。

J Mol Med (Berl). 2003 May;81(5):310-20. doi: 10.1007/s00109-003-0438-7. Epub 2003 Apr 30.

Gene transfer of the uroporphyrinogen III synthase cDNA into haematopoietic progenitor cells in view of a future gene therapy in congenital erythropoietic porphyria.考虑到未来对先天性红细胞生成性卟啉病进行基因治疗，将尿卟啉原III合酶cDNA基因转移至造血祖细胞。

J Inherit Metab Dis. 1997 Jun;20(2):247-57. doi: 10.1023/a:1005365008147.

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Correction of the enzyme deficit of bone marrow cells in congenital erythropoietic porphyria by retroviral gene transfer.

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