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

✍ Scribed by F. Moreau-Gaudry; C. Barbot; F. Mazurier; F. X. Mahon; J. Reiffers; C. Ged; H. de Verneuil

Book ID: 105995218
Publisher: Springer
Year: 1996
Tongue: English
Weight: 129 KB
Volume: 38
Category: Article
ISSN: 1269-3286
DOI: 10.1007/s00282-996-0217-3

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Background Congenital erythropoietic porphyria (CEP) is an inherited disease caused by a de®ciency of uroporphyrinogen III synthase, the fourth enzyme of the haem biosynthesis pathway. It is characterized by accumulation of uroporphyrin I in the bone marrow, peripheral blood and other organs. The pr

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