LEAPER TECHNOLOGY BASED METHOD FOR TREATING MPS IH AND COMPOSITION

摘要

A LEAPER technology based method for target editing of RNA, comprising: using an LEAPER technology to safely and effectively carry out in-vivo editing from adenosine to hypoxanthine bases on the RNA, accurately repairing pathogenic mutation sites and realizing the purpose of treating all diseases caused by G>A mutation, such as MPS IH.