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Compositions and methods for treating glycogen storage disease type 1A
Compositions and methods for treating glycogen storage disease type 1A
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机译:治疗糖原储存疾病1A的组合物和方法
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摘要
The present invention provides a composition comprising a novel adenosine base editor (eg, ABE8) with increased efficiency and a base editor comprising adenosine deaminase variants for altering mutations associated with glycogen storage disease type 1a (GSD1a). provides a way to use it.
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