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Methods of gene therapy using nucleic acid sequences for ATP-binding cassette transporter

机译:使用用于ATP结合盒转运蛋白的核酸序列进行基因治疗的方法

摘要

The present invention provides nucleic acid and amino acid sequences of an ATP binding cassette transporter and mutated sequences thereof associated with macular degeneration. Methods of detecting agents that modify ATP-binding cassette transporter comprising combining purified ATP binding cassette transporter and at least one agent suspected of modifying the ATP binding cassette transporter an observing a change in at least one characteristic associated with ATP binding cassette transporter. Methods of detecting macular degeneration is also embodied by the present invention.
机译:本发明提供了与黄斑变性相关的ATP结合盒转运蛋白的核酸和氨基酸序列及其突变序列。检测修饰ATP结合盒转运蛋白的试剂的方法包括结合纯化的ATP结合盒转运蛋白和至少一种怀疑修饰ATP结合盒转运蛋白的试剂,观察与ATP结合盒转运蛋白相关的至少一种特征的变化。本发明也体现了检测黄斑变性的方法。

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