发明名称 |
ALANINE-GLYOXYLATE AMINOTRANSFERASE THERAPEUTICS |
摘要 |
The present invention relates to an alanine glyoxylate aminotransferase (AGXT) I340M therapeutic for use as a medicament or in a method of treatment, for example in the treatment of an AGXT-responsive condition. The AGXT I340M therapeutic is an AGXT I340M protein comprising an amino acid sequence, which, when optimally aligned with SEQ ID NO: 2, comprises a methionine at a position corresponding to position 340 in SEQ ID NO: 2, a nucleic acid molecule encoding such an AGXT I340M protein, or a virion of a viral gene therapy vector comprising such a nucleic acid molecule. The AGXT I340M therapeutic has a higher specific activity as compared to other AGXT alleles and may therefore be advantageously used in the treatment of primary hyperoxaluria type I.
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申请公布号 |
US2012027745(A1) |
申请公布日期 |
2012.02.02 |
申请号 |
US201013146869 |
申请日期 |
2010.02.01 |
申请人 |
RODRIGUEZ PENA MARIA SOL;PETRY HARALD;TWISK JAAP;VAN DEVENTER SANDER JAN HENDRIK;SALIDO RUIZ EDUARDO CARLOS;TORRES RAMIREZ ARMANDO;AMSTERDAM MOLCULAR THERAPEUTICS (AMT) IP B.V. |
发明人 |
RODRIGUEZ PENA MARIA SOL;PETRY HARALD;TWISK JAAP;VAN DEVENTER SANDER JAN HENDRIK;SALIDO RUIZ EDUARDO CARLOS;TORRES RAMIREZ ARMANDO |
分类号 |
A61K38/45;A61K48/00;A61P3/00;C12N15/85;C12N15/86;C12N15/864 |
主分类号 |
A61K38/45 |
代理机构 |
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地址 |
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