公开(公告)号：US06518413B1

公开(公告)日：2003-02-11

申请号：US09091501

申请日：1998-07-14

Applicant: Jonathon M. Tinsley ,  Kay E. Davies

Inventor： Jonathon M. Tinsley ,  Kay E. Davies

IPC: C07H2102

CPC classification number: C07K14/4708 ,  A01K2217/05 ,  A61K38/00 ,  A61K48/00

Abstract: Nucleic acid from which a polypeptide with utrophin function can be expressed, especially mini-genes and chimaeric constructs. Expression significantly decreases the severity of the dystrophic muscle phenotype in an animal model, indicating usefulness in treatment of muscular dystrophy. The nucleic acid and encoded polypeptides are also useful in screening for substances to modulate utrophin binding to actin and/or the dystrophin protein complex.

Abstract translation: 具有utrophin功能的多肽可以从其中表达的核酸，特别是微型基因和嵌合体构建体。 表达显着降低动物模型中营养不良肌肉表型的严重程度，表明在治疗肌营养不良症中的有用性。 核酸和编码的多肽也可用于筛选调节utrophin结合肌动蛋白和/或肌营养不良蛋白复合物的物质。