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Depletion of Human-enzyme Mediated Homocysteine for the Treatment of Patients with Hyperhomocysteinemia and Homocystinuria

机译:人类酶介导的同型半胱氨酸的消耗治疗高同型半胱氨酸血症和同型半胱氨酸尿症的患者

摘要

Depletion of Human-enzyme Mediated Homocysteine for the Treatment of Patients with Hyperhomocysteinemia and Homocystinuria Disclosed are methods and compositions for the manipulation of improved proteins having homocysty (te) nase enzyme activity. For example, modified cystathionine- [gamma] -lyase (CGL) enzymes are disclosed that include one or more amino acid substitutions and are capable of degrading homocystine (taine). Also provided are compositions and methods for treating homocystin (taine) depleted homocystinuria or hyperhomocysteinemia using the disclosed enzymes or nucleic acids.
机译:耗竭人类酶介导的同型半胱氨酸治疗高同型半胱氨酸血症和同型半胱氨酸尿症的患者公开了用于处理具有同型半胱氨酸酶活性的改良蛋白的方法和组合物。例如,公开了包括一个或多个氨基酸取代并且能够降解高胱氨酸(甜菜碱)的修饰的胱硫醚-γ-裂合酶(CGL)酶。还提供了使用所公开的酶或核酸来治疗高半胱氨酸(taine)耗尽的高半胱氨酸尿症或高同型半胱氨酸血症的组合物和方法。

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