Split intein-mediated protein trans-splicing to express large dystrophins  

作　　者：Hichem Tasfaout 

机构地区：[1]不详

出　　处：《四川生理科学杂志》2024年第7期1627-1627,共1页

摘　　要：Gene replacement using adeno-associated virus(AAV)vectors is a promising therapeutic approach for many diseases1,2.However,this therapeutic modality is challenged by the packaging capacity of AAVs(approximately 4.7 kilobases)3,limiting its application for disorders involving large coding sequences,such as Duchenne muscular dystrophy,with a 14 kilobase messenger RNA.

关 键 词：SPLIT BASES LIMITING 

分 类 号：R338[医药卫生—人体生理学]