Aminoglycoside antibiotics are candidate drugs for a large number of such genetic diseases; however, aminoglycosides are toxic, lack specificity and show low efficacy in this application.

 
  • 氨基糖苷类抗生素是治疗大部分此类遗传病的手段;但是,目前使用的氨基糖苷具有毒性,缺乏特异性并且使用效率低。
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