首页>
外国专利>
Materials and methods for treatment of usher syndrome type 2A
Materials and methods for treatment of usher syndrome type 2A
展开▼
机译:用于治疗迎膜综合征2A的材料和方法
展开▼
页面导航
摘要
著录项
相似文献
摘要
The present application provides materials and methods for treating a patient with Usher Syndrome Type 2A, both ex vivo and in vivo; materials and methods for editing a USH2A gene in a human cell; materials and methods for editing an USH2A gene containing an IVS40 mutation; materials and methods for treating a patient with an USH2A gene containing an IVS40 mutation; and a method for deleting a sequence comprising an IVS40 mutation within a USH2A gene of a cell. The present application also provides one or more gRNAs or sgRNAs for editing an USH2A gene containing an IVS40 mutation. The present application provides a therapeutic for treating a patient with Usher Syndrome Type 2A. The present application also provides a kit for treating a patient with Usher Syndrome Type 2A.
展开▼