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Composition and method for TTR gene editing and treatment of ATTR amyloidosis
Composition and method for TTR gene editing and treatment of ATTR amyloidosis
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机译:TTR基因编辑和治疗ATTR淀粉样变性的组合物和方法
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摘要
Compositions and methods for editing are provided, such as introducing double-strand breaks in the TTR gene. Compositions and methods are provided for treating a subject with amyloidosis (ATTR) associated with transthyretin.
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