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Title:
【発明の名称】rhPBGDの製造及び急性間欠性ポルフィリン症(AIP)及び他のポルフィリン症の患者を処置するための新規な治療方法
Document Type and Number:
Japanese Patent JP2003505057
Kind Code:
A
Abstract:
A method for production of rhPBGD in high scale and use of rhPBGD in a method for treatment or prophylaxis of disease caused by deficiency, in a subject, of an enzyme belonging to the heme biosynthetic pathway. The method comprising administering, to the subject, an effective amount of one or more catalyst which is said enzyme or an enzymatically equivalent part or analogue thereof prefreable in combination with a gene therapy of a mutation related to the catalyst. The disease is selected from the group consisting of acute intermittent porphyria (AIP), ALA deficiency porphyria (ADP), Porphyria cutanea tarda (PCT), Hereditary coproporphyria (HCP), Harderoporphyria (HDP), Variegata porphyria (VP), Congenital erythropoietic porphyria (CEP), Erythropoietic protoporphyria (EPP), and Hepatoerythropoietic porphyria (HEP). The catalyst is one or more enzymes selected from the group consisting of delta-aminolevulininic acid synthetase, delta-aminovulinic acid dehydratase (ALAD), porphobilinogen deaminase (PBGD), uroporphyrinogen III cosythetase, uroporphyrinogen decarboxylase, coproporphyrinogen oxidase, protoporphyrinogen oxidase, and ferrochelatase, or an enzymatically equivalent part or analogue therof. In addition the invention relates to the use of rhPBGD. The invention also relates to an expression plasmid pExp1-M2-BB (seq. ID No.1) and to use of a DNA fragment, the EcoR I - Hind III linear fragment (seq. ID No. 2), used for transformation in the hemC disruption strategy for production of rhPBGD expressed in E. coli..

Inventors:
Gelafore, Par
Fog, jens
Application Number:
JP2001511948A
Publication Date:
February 12, 2003
Filing Date:
July 27, 2000
Export Citation:
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Assignee:
Hem-Biotech A/S
International Classes:
C12N15/09; A61K9/127; A61K9/19; A61K9/50; A61K9/56; A61K33/24; A61K35/12; A61K35/76; A61K38/43; A61K38/44; A61K38/51; A61K47/04; A61K47/18; A61K47/24; A61K47/26; A61K48/00; A61P7/00; A61P43/00; C12N1/15; C12N1/19; C12N1/21; C12N5/10; C12N9/88; (IPC1-7): C12N15/09; A61K9/127; A61K9/19; A61K9/50; A61K9/56; A61K33/24; A61K35/12; A61K35/76; A61K38/43; A61K38/44; A61K47/04; A61K47/18; A61K47/24; A61K47/26; A61K48/00; A61P7/00; A61P43/00; C12N1/15; C12N1/19; C12N1/21; C12N5/10; C12N9/88
Attorney, Agent or Firm:
Shintaro Nogawa